ATE217874T1 - Texaphyrin metallkomplexe mit verbesserten funktionellen gruppen - Google Patents

Texaphyrin metallkomplexe mit verbesserten funktionellen gruppen

Info

Publication number
ATE217874T1
ATE217874T1 AT95911776T AT95911776T ATE217874T1 AT E217874 T1 ATE217874 T1 AT E217874T1 AT 95911776 T AT95911776 T AT 95911776T AT 95911776 T AT95911776 T AT 95911776T AT E217874 T1 ATE217874 T1 AT E217874T1
Authority
AT
Austria
Prior art keywords
macrocycle
electron
positions
addition
metal complexes
Prior art date
Application number
AT95911776T
Other languages
English (en)
Inventor
Jonathan L Sessler
Tarak D Mody
Gregory W Hemmi
Original Assignee
Univ Texas
Pharmacyclics Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Univ Texas, Pharmacyclics Inc filed Critical Univ Texas
Application granted granted Critical
Publication of ATE217874T1 publication Critical patent/ATE217874T1/de

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D487/00Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00
    • C07D487/22Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00 in which the condensed system contains four or more hetero rings
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K41/00Medicinal preparations obtained by treating materials with wave energy or particle radiation ; Therapies using these preparations
    • A61K41/0038Radiosensitizing, i.e. administration of pharmaceutical agents that enhance the effect of radiotherapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K41/00Medicinal preparations obtained by treating materials with wave energy or particle radiation ; Therapies using these preparations
    • A61K41/0057Photodynamic therapy with a photosensitizer, i.e. agent able to produce reactive oxygen species upon exposure to light or radiation, e.g. UV or visible light; photocleavage of nucleic acids with an agent
    • A61K41/0076PDT with expanded (metallo)porphyrins, i.e. having more than 20 ring atoms, e.g. texaphyrins, sapphyrins, hexaphyrins, pentaphyrins, porphocyanines
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K41/00Medicinal preparations obtained by treating materials with wave energy or particle radiation ; Therapies using these preparations
    • A61K41/10Inactivation or decontamination of a medicinal preparation prior to administration to an animal or a person
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K49/00Preparations for testing in vivo
    • A61K49/001Preparation for luminescence or biological staining
    • A61K49/0013Luminescence
    • A61K49/0017Fluorescence in vivo
    • A61K49/0019Fluorescence in vivo characterised by the fluorescent group, e.g. oligomeric, polymeric or dendritic molecules
    • A61K49/0021Fluorescence in vivo characterised by the fluorescent group, e.g. oligomeric, polymeric or dendritic molecules the fluorescent group being a small organic molecule
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K49/00Preparations for testing in vivo
    • A61K49/06Nuclear magnetic resonance [NMR] contrast preparations; Magnetic resonance imaging [MRI] contrast preparations
    • A61K49/08Nuclear magnetic resonance [NMR] contrast preparations; Magnetic resonance imaging [MRI] contrast preparations characterised by the carrier
    • A61K49/10Organic compounds
    • A61K49/101Organic compounds the carrier being a complex-forming compound able to form MRI-active complexes with paramagnetic metals
    • A61K49/106Organic compounds the carrier being a complex-forming compound able to form MRI-active complexes with paramagnetic metals the complex-forming compound being cyclic, e.g. DOTA
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K51/00Preparations containing radioactive substances for use in therapy or testing in vivo
    • A61K51/02Preparations containing radioactive substances for use in therapy or testing in vivo characterised by the carrier, i.e. characterised by the agent or material covalently linked or complexing the radioactive nucleus
    • A61K51/04Organic compounds
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K51/00Preparations containing radioactive substances for use in therapy or testing in vivo
    • A61K51/02Preparations containing radioactive substances for use in therapy or testing in vivo characterised by the carrier, i.e. characterised by the agent or material covalently linked or complexing the radioactive nucleus
    • A61K51/04Organic compounds
    • A61K51/0474Organic compounds complexes or complex-forming compounds, i.e. wherein a radioactive metal (e.g. 111In3+) is complexed or chelated by, e.g. a N2S2, N3S, NS3, N4 chelating group
    • A61K51/0485Porphyrins, texaphyrins wherein the nitrogen atoms forming the central ring system complex the radioactive metal
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K51/00Preparations containing radioactive substances for use in therapy or testing in vivo
    • A61K51/02Preparations containing radioactive substances for use in therapy or testing in vivo characterised by the carrier, i.e. characterised by the agent or material covalently linked or complexing the radioactive nucleus
    • A61K51/04Organic compounds
    • A61K51/08Peptides, e.g. proteins, carriers being peptides, polyamino acids, proteins
    • A61K51/10Antibodies or immunoglobulins; Fragments thereof, the carrier being an antibody, an immunoglobulin or a fragment thereof, e.g. a camelised human single domain antibody or the Fc fragment of an antibody
    • A61K51/1093Antibodies or immunoglobulins; Fragments thereof, the carrier being an antibody, an immunoglobulin or a fragment thereof, e.g. a camelised human single domain antibody or the Fc fragment of an antibody conjugates with carriers being antibodies
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61LMETHODS OR APPARATUS FOR STERILISING MATERIALS OR OBJECTS IN GENERAL; DISINFECTION, STERILISATION OR DEODORISATION OF AIR; CHEMICAL ASPECTS OF BANDAGES, DRESSINGS, ABSORBENT PADS OR SURGICAL ARTICLES; MATERIALS FOR BANDAGES, DRESSINGS, ABSORBENT PADS OR SURGICAL ARTICLES
    • A61L2/00Methods or apparatus for disinfecting or sterilising materials or objects other than foodstuffs or contact lenses; Accessories therefor
    • A61L2/0005Methods or apparatus for disinfecting or sterilising materials or objects other than foodstuffs or contact lenses; Accessories therefor for pharmaceuticals, biologicals or living parts
    • A61L2/0011Methods or apparatus for disinfecting or sterilising materials or objects other than foodstuffs or contact lenses; Accessories therefor for pharmaceuticals, biologicals or living parts using physical methods
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H17/00Compounds containing heterocyclic radicals directly attached to hetero atoms of saccharide radicals
    • C07H17/02Heterocyclic radicals containing only nitrogen as ring hetero atoms
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2121/00Preparations for use in therapy
AT95911776T 1994-02-15 1995-02-15 Texaphyrin metallkomplexe mit verbesserten funktionellen gruppen ATE217874T1 (de)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US08/196,964 US5599923A (en) 1989-03-06 1994-02-15 Texaphyrin metal complexes having improved functionalization
PCT/US1995/001996 WO1995021845A1 (en) 1994-02-15 1995-02-15 Texaphyrin metal complexes having improved functionalization

Publications (1)

Publication Number Publication Date
ATE217874T1 true ATE217874T1 (de) 2002-06-15

Family

ID=22727479

Family Applications (1)

Application Number Title Priority Date Filing Date
AT95911776T ATE217874T1 (de) 1994-02-15 1995-02-15 Texaphyrin metallkomplexe mit verbesserten funktionellen gruppen

Country Status (14)

Country Link
US (2) US5599923A (de)
EP (1) EP0745085B1 (de)
JP (1) JPH10500659A (de)
KR (1) KR970701193A (de)
AT (1) ATE217874T1 (de)
AU (1) AU688008B2 (de)
CA (1) CA2182960A1 (de)
DE (1) DE69526782T2 (de)
DK (1) DK0745085T3 (de)
ES (1) ES2173951T3 (de)
NO (1) NO308473B1 (de)
NZ (1) NZ282185A (de)
PT (1) PT745085E (de)
WO (1) WO1995021845A1 (de)

Families Citing this family (611)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5252720A (en) * 1989-03-06 1993-10-12 Board Of Regents, The University Of Texas System Metal complexes of water soluble texaphyrins
US5567687A (en) * 1989-03-06 1996-10-22 University Of Texas Texaphyrins and uses thereof
US5457183A (en) * 1989-03-06 1995-10-10 Board Of Regents, The University Of Texas System Hydroxylated texaphyrins
US6335434B1 (en) 1998-06-16 2002-01-01 Isis Pharmaceuticals, Inc., Nucleosidic and non-nucleosidic folate conjugates
US8153602B1 (en) 1991-11-19 2012-04-10 Isis Pharmaceuticals, Inc. Composition and methods for the pulmonary delivery of nucleic acids
US5607924A (en) * 1992-01-21 1997-03-04 Pharmacyclics, Inc. DNA photocleavage using texaphyrins
US5888997A (en) * 1994-04-14 1999-03-30 Pharmacyclics, Inc. Radiation sensitization using texaphyrins
US5565552A (en) * 1992-01-21 1996-10-15 Pharmacyclics, Inc. Method of expanded porphyrin-oligonucleotide conjugate synthesis
US5595726A (en) * 1992-01-21 1997-01-21 Pharmacyclics, Inc. Chromophore probe for detection of nucleic acid
US5523389A (en) * 1992-09-29 1996-06-04 Isis Pharmaceuticals, Inc. Inhibitors of human immunodeficiency virus
US5798491A (en) * 1993-06-09 1998-08-25 Board Of Regents, The University Of Texas System Multi-mechanistic chemical cleavage using certain metal complexes
ATE247128T1 (de) 1993-09-03 2003-08-15 Isis Pharmaceuticals Inc Aminoderivatisierte nukleoside und oligonukleoside
US5969111A (en) * 1994-04-14 1999-10-19 Board Of Regents, The University Of Texas System Texaphyrins substituted with imidazole are provided
US5837866A (en) * 1994-09-21 1998-11-17 Board Of Regents, The University Of Texas Phosphoramidite derivatives of macrocycles
US5633354A (en) * 1994-09-21 1997-05-27 Pharmacyclics, Inc. Phosphoramidite derivatives of texaphyrins
US6176842B1 (en) 1995-03-08 2001-01-23 Ekos Corporation Ultrasound assembly for use with light activated drugs
US6210356B1 (en) * 1998-08-05 2001-04-03 Ekos Corporation Ultrasound assembly for use with a catheter
US6420549B1 (en) 1995-06-06 2002-07-16 Isis Pharmaceuticals, Inc. Oligonucleotide analogs having modified dimers
US5714328A (en) * 1995-06-07 1998-02-03 Board Of Regents, The University Of Texas System RNA photocleavage using texaphyrins
RU2106146C1 (ru) 1995-07-17 1998-03-10 Институт элементоорганических соединений РАН Средство для подавления опухолевого роста
US5776925A (en) * 1996-01-25 1998-07-07 Pharmacyclics, Inc. Methods for cancer chemosensitization
NZ331977A (en) * 1996-03-26 2000-07-28 Pharmacyclics Inc Texaphyrin in photodynamic therapy of pigment-related lesions
DE69727833T2 (de) * 1996-06-04 2005-01-13 Board of Regents, The University of Texas System, Austin Membraneinbau von texaphyrinen
US6375930B2 (en) 1996-06-04 2002-04-23 Board Of Regents, The University Of Texas System Membrane incorporation of texaphyrins
US20040266706A1 (en) * 2002-11-05 2004-12-30 Muthiah Manoharan Cross-linked oligomeric compounds and their use in gene modulation
US9096636B2 (en) 1996-06-06 2015-08-04 Isis Pharmaceuticals, Inc. Chimeric oligomeric compounds and their use in gene modulation
US20040147022A1 (en) * 1996-06-06 2004-07-29 Baker Brenda F. 2'-methoxy substituted oligomeric compounds and compositions for use in gene modulations
US20030044941A1 (en) 1996-06-06 2003-03-06 Crooke Stanley T. Human RNase III and compositions and uses thereof
US7812149B2 (en) * 1996-06-06 2010-10-12 Isis Pharmaceuticals, Inc. 2′-Fluoro substituted oligomeric compounds and compositions for use in gene modulations
US20050119470A1 (en) * 1996-06-06 2005-06-02 Muthiah Manoharan Conjugated oligomeric compounds and their use in gene modulation
US7875733B2 (en) 2003-09-18 2011-01-25 Isis Pharmaceuticals, Inc. Oligomeric compounds comprising 4′-thionucleosides for use in gene modulation
US20040171031A1 (en) * 1996-06-06 2004-09-02 Baker Brenda F. Sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
US20040203024A1 (en) * 1996-06-06 2004-10-14 Baker Brenda F. Modified oligonucleotides for use in RNA interference
US20050053976A1 (en) * 1996-06-06 2005-03-10 Baker Brenda F. Chimeric oligomeric compounds and their use in gene modulation
US5898031A (en) * 1996-06-06 1999-04-27 Isis Pharmaceuticals, Inc. Oligoribonucleotides for cleaving RNA
US6022959A (en) * 1996-08-20 2000-02-08 Pharmacyclics, Inc. Nucleic acids internally-derivatized with a texaphyrin metal complex and uses thereof
US6331286B1 (en) 1998-12-21 2001-12-18 Photogen, Inc. Methods for high energy phototherapeutics
US6270749B1 (en) * 1996-12-11 2001-08-07 Pharmacyclics, Inc. Use of Texaphyrin in ocular diagnosis and therapy
EP1091742A4 (de) 1997-02-14 2007-05-30 Miravant Pharm Inc Indium photosensibilisatoren zur pdt
JPH10279502A (ja) * 1997-03-31 1998-10-20 Katsuro Tachibana 光感受性物質の励起方法
US6716625B1 (en) 1997-04-16 2004-04-06 Claude Selitrennikoff Histidine kinases of Aspergillus and other fungal species, related compositions, and methods of use
WO1998047539A1 (en) * 1997-04-22 1998-10-29 Nycomed Imaging As Nmr image compounds
US6676626B1 (en) * 1998-05-01 2004-01-13 Ekos Corporation Ultrasound assembly with increased efficacy
US6582392B1 (en) 1998-05-01 2003-06-24 Ekos Corporation Ultrasound assembly for use with a catheter
EP1012331B1 (de) 1997-07-01 2006-03-29 Isis Pharmaceuticals, Inc. Zusammensetzungen und verfahren zur verabreichung von oligonukleotiden über die speiseröhre
US5886173A (en) * 1997-07-30 1999-03-23 Pharmacyclics, Inc. Metallation of macrocycles with 2,4-dicarbonyl-metal complexes
US6022526A (en) * 1997-07-30 2000-02-08 Pharmacyclics, Inc. Use of texaphyrins in detection of melanin and melanin metabolites diagnostic of melanotic melanoma
US6495118B1 (en) 1997-09-26 2002-12-17 Schering Aktiengesellschaft Lipophilic metal complexes for necrosis and infarction imaging
US8974363B2 (en) * 1997-12-11 2015-03-10 Provectus Pharmatech, Inc. Topical medicaments and methods for photodynamic treatment of disease
US20040186071A1 (en) * 1998-04-13 2004-09-23 Bennett C. Frank Antisense modulation of CD40 expression
US7321828B2 (en) * 1998-04-13 2008-01-22 Isis Pharmaceuticals, Inc. System of components for preparing oligonucleotides
US6841539B1 (en) * 1998-05-21 2005-01-11 Isis Pharmaceuticals, Inc. Compositions and methods for topical delivery of oligonucleotides
WO1999060012A1 (en) * 1998-05-21 1999-11-25 Isis Pharmaceuticals, Inc. Compositions and methods for non-parenteral delivery of oligonucleotides
DE19831217A1 (de) * 1998-07-03 2000-01-05 Schering Ag Neue Porphyrinderivate, diese enthaltende pharmazeutische Mittel und ihre Verwendung in der photodynamischen Therapie und MRI-Diagnostik
US8557298B2 (en) * 1998-08-06 2013-10-15 Provectus Pharmatech, Inc. Medicaments for chemotherapeutic treatment of disease
US20090117199A1 (en) * 1998-08-06 2009-05-07 Scott Timothy C Method of treatment of cancer
US6225293B1 (en) 1998-09-02 2001-05-01 Isis Pharmaceuticals, Inc. Methods and compounds for tracking the biodistribution of macromolecule-carrier combinations
US6077709A (en) 1998-09-29 2000-06-20 Isis Pharmaceuticals Inc. Antisense modulation of Survivin expression
US20020001567A1 (en) * 1998-12-21 2002-01-03 Photogen, Inc. Intracorporeal medicaments for high energy phototherapeutic treatment of disease
US8470296B2 (en) * 1998-12-21 2013-06-25 Provectus Pharmatech, Inc. Intracorporeal medicaments for high energy phototherapeutic treatment of disease
US7384623B1 (en) 1998-12-21 2008-06-10 Provectus Pharmatech, Inc. High energy phototherapeutic agents
US6300320B1 (en) 1999-01-05 2001-10-09 Isis Pharmaceuticals, Inc. Modulation of c-jun using inhibitors of protein kinase C
US6127124A (en) 1999-01-20 2000-10-03 Isis Pharmaceuticals, Inc. Fluorescence based nuclease assay
US6821738B2 (en) * 1999-01-20 2004-11-23 The Board Of Regents For Oklahoma State University Broad spectrum bio-detection of nerve agents, organophosphates, and other chemical warfare agents
US7098192B2 (en) 1999-04-08 2006-08-29 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of STAT3 expression
US6838292B1 (en) 1999-04-19 2005-01-04 Alion Science And Technology Corporation Detection of biological warfare agents
US6656730B1 (en) 1999-06-15 2003-12-02 Isis Pharmaceuticals, Inc. Oligonucleotides conjugated to protein-binding drugs
EP1206288B1 (de) * 1999-08-02 2007-01-10 The Regents Of The University Of Michigan Zielgerichtete fiberlose radiative effektoren
US7579338B2 (en) * 1999-10-29 2009-08-25 Pharmacyclics, Inc. Methods and compositions for treating atheroma, tumors and other neoplastic tissues
WO2001032210A2 (en) * 1999-10-29 2001-05-10 Pharmacyclics, Inc. Compositions for treating atheroma and neoplastic tissue
US7897140B2 (en) * 1999-12-23 2011-03-01 Health Research, Inc. Multi DTPA conjugated tetrapyrollic compounds for phototherapeutic contrast agents
US20020055479A1 (en) 2000-01-18 2002-05-09 Cowsert Lex M. Antisense modulation of PTP1B expression
US6261840B1 (en) 2000-01-18 2001-07-17 Isis Pharmaceuticals, Inc. Antisense modulation of PTP1B expression
US20030176385A1 (en) * 2000-02-15 2003-09-18 Jingfang Ju Antisense modulation of protein expression
US6680172B1 (en) 2000-05-16 2004-01-20 Regents Of The University Of Michigan Treatments and markers for cancers of the central nervous system
US8568766B2 (en) * 2000-08-24 2013-10-29 Gattadahalli M. Anantharamaiah Peptides and peptide mimetics to treat pathologies associated with eye disease
US6638924B2 (en) 2000-08-30 2003-10-28 Pharmacyclics, Inc. Metallotexaphyrin derivatives
US7112671B2 (en) * 2000-08-30 2006-09-26 Pharmacyclics, Inc. Non-symmetric tripyrranes in the synthesis of novel macrocycles
US7449454B2 (en) * 2000-08-30 2008-11-11 Pharmacyclics, Inc. Metallotexaphyrin derivatives
JP5305553B2 (ja) 2000-10-12 2013-10-02 ユニバーシティー オブ ロチェスター 癌細胞の増殖を阻害する組成物
CA2429411A1 (en) * 2000-11-17 2002-05-23 Pharmacyclics, Inc. Texaphyrin coordination compounds and uses thereof
US7767802B2 (en) 2001-01-09 2010-08-03 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of anti-apoptotic genes
US20050107595A1 (en) * 2001-06-20 2005-05-19 Genentech, Inc. Compositions and methods for the diagnosis and treatment of tumor
ATE522623T1 (de) 2001-06-20 2011-09-15 Genentech Inc Zusammensetzungen und verfahren zur behandlung und diagnose von lungentumoren
US7803915B2 (en) * 2001-06-20 2010-09-28 Genentech, Inc. Antibody compositions for the diagnosis and treatment of tumor
CA2451643C (en) 2001-06-21 2012-11-13 Isis Pharmaceuticals, Inc. Antisense modulation of superoxide dismutase 1, soluble expression
US6964950B2 (en) 2001-07-25 2005-11-15 Isis Pharmaceuticals, Inc. Antisense modulation of C-reactive protein expression
US7425545B2 (en) 2001-07-25 2008-09-16 Isis Pharmaceuticals, Inc. Modulation of C-reactive protein expression
US20030096772A1 (en) 2001-07-30 2003-05-22 Crooke Rosanne M. Antisense modulation of acyl CoA cholesterol acyltransferase-2 expression
US7407943B2 (en) 2001-08-01 2008-08-05 Isis Pharmaceuticals, Inc. Antisense modulation of apolipoprotein B expression
US7227014B2 (en) 2001-08-07 2007-06-05 Isis Pharmaceuticals, Inc. Antisense modulation of apolipoprotein (a) expression
ES2390531T3 (es) 2001-09-18 2012-11-13 Genentech, Inc. Composiciones y procedimientos para el diagnósitco y tratamiento de tumor
US6750019B2 (en) 2001-10-09 2004-06-15 Isis Pharmaceuticals, Inc. Antisense modulation of insulin-like growth factor binding protein 5 expression
KR100437547B1 (ko) * 2001-10-09 2004-06-30 김환규 희토류 이온을 포함하는 착염형 텍사피린 덴드리머 화합물및 그 제조방법
EP2270231A3 (de) 2001-10-09 2011-04-27 ISIS Pharmaceuticals, Inc. Antisense Modulation der Expression von insulin-like growth factor binding protein 5
EP1647232B1 (de) 2001-12-03 2011-08-17 Ekos Corporation Katheter mit mehreren Ultraschall-abstrahlenden Teilen
US6965025B2 (en) 2001-12-10 2005-11-15 Isis Pharmaceuticals, Inc. Antisense modulation of connective tissue growth factor expression
AU2002367318B2 (en) 2002-01-02 2007-07-12 Genentech, Inc. Compositions and methods for the diagnosis and treatment of tumor
WO2003072581A1 (en) 2002-02-26 2003-09-04 Board Of Regents, The University Of Texas System Cyclo[n]pyrroles and methods thereto
US6723750B2 (en) 2002-03-15 2004-04-20 Allergan, Inc. Photodynamic therapy for pre-melanomas
US20030180712A1 (en) 2002-03-20 2003-09-25 Biostratum Ab Inhibition of the beta3 subunit of L-type Ca2+ channels
US8226629B1 (en) 2002-04-01 2012-07-24 Ekos Corporation Ultrasonic catheter power control
KR20040101502A (ko) 2002-04-16 2004-12-02 제넨테크, 인크. 종양의 진단 및 치료 방법 및 이를 위한 조성물
US7199107B2 (en) 2002-05-23 2007-04-03 Isis Pharmaceuticals, Inc. Antisense modulation of kinesin-like 1 expression
JP4529344B2 (ja) * 2002-06-03 2010-08-25 ニプロ株式会社 腫瘍組織の酸素濃度を上昇させるためのポルフィリン酸素輸液製剤
US20040092470A1 (en) * 2002-06-18 2004-05-13 Leonard Sherry A. Dry powder oligonucleotide formualtion, preparation and its uses
DE60328496D1 (en) 2002-06-27 2009-09-03 Health Research Inc Fluorinierte chlorin und bacteriochlorin photosensitizer für fotodynamische therapie
EP1606291A2 (de) * 2002-07-02 2005-12-21 Health Research, Inc. Effiziente synthese von pyropheophorbid-a und seiner derivate
US20040019000A1 (en) * 2002-07-19 2004-01-29 Muthiah Manoharan Polyalkyleneamine-containing oligomers
CA2495478A1 (en) 2002-08-05 2004-02-12 University Of Rochester Protein transducing domain/deaminase chimeric proteins, related compounds, and uses thereof
US6878805B2 (en) * 2002-08-16 2005-04-12 Isis Pharmaceuticals, Inc. Peptide-conjugated oligomeric compounds
EA008940B1 (ru) 2002-09-13 2007-10-26 Репликор, Инк. Антивирусные олигонуклеотиды, не связанные с комплементарностью последовательностей
WO2004031350A2 (en) 2002-09-26 2004-04-15 Amgen, Inc. Modulation of forkhead box o1a expression
US6921371B2 (en) * 2002-10-14 2005-07-26 Ekos Corporation Ultrasound radiating members for catheter
WO2004110345A2 (en) * 2002-10-29 2004-12-23 Pharmacia Corporation Differentially expressed genes involved in cancer, the polypeptides encoded thereby, and methods of using the same
US9150606B2 (en) * 2002-11-05 2015-10-06 Isis Pharmaceuticals, Inc. Compositions comprising alternating 2'-modified nucleosides for use in gene modulation
US9150605B2 (en) 2002-11-05 2015-10-06 Isis Pharmaceuticals, Inc. Compositions comprising alternating 2′-modified nucleosides for use in gene modulation
WO2004044138A2 (en) 2002-11-05 2004-05-27 Isis Pharmaceuticals, Inc. Chimeric oligomeric compounds and their use in gene modulation
CA2504929C (en) * 2002-11-05 2014-07-22 Charles Allerson Compositions comprising alternating 2'-modified nucleosides for use in gene modulation
PT1569695E (pt) 2002-11-13 2013-07-16 Genzyme Corp Modulação anti-sentido da expressão de apolipoproteína
JP4986109B2 (ja) 2002-11-13 2012-07-25 ジェンザイム・コーポレーション アポリポタンパク質b発現のアンチセンス調節
WO2004046330A2 (en) * 2002-11-15 2004-06-03 Morphotek, Inc. Methods of generating high-production of antibodies from hybridomas created by in vitro immunization
WO2004045520A2 (en) 2002-11-15 2004-06-03 Musc Foundation For Research Development Complement receptor 2 targeted complement modulators
AU2003294462C1 (en) 2002-11-21 2011-06-30 University Of Utah Research Foundation Purinergic modulation of smell
US7144999B2 (en) 2002-11-23 2006-12-05 Isis Pharmaceuticals, Inc. Modulation of hypoxia-inducible factor 1 alpha expression
US7468356B2 (en) 2003-02-11 2008-12-23 Antisense Therapeutics Ltd. Modulation of insulin like growth factor I receptor expression
US7803781B2 (en) 2003-02-28 2010-09-28 Isis Pharmaceuticals, Inc. Modulation of growth hormone receptor expression and insulin-like growth factor expression
US20040185559A1 (en) 2003-03-21 2004-09-23 Isis Pharmaceuticals Inc. Modulation of diacylglycerol acyltransferase 1 expression
US7598227B2 (en) 2003-04-16 2009-10-06 Isis Pharmaceuticals Inc. Modulation of apolipoprotein C-III expression
US7399853B2 (en) 2003-04-28 2008-07-15 Isis Pharmaceuticals Modulation of glucagon receptor expression
EP1660512A4 (de) * 2003-06-02 2009-12-23 Isis Pharmaceuticals Inc Oligonucleotid-synthese mit alternativen lösungsmitteln
JP4579911B2 (ja) 2003-06-03 2010-11-10 アイシス・ファーマシューティカルズ・インコーポレイテッド スルビビン発現の調節
EP3604537B1 (de) 2003-06-13 2021-12-08 Alnylam Europe AG Doppelsträngige ribonukleinsäure mit erhöhter wirksamkeit in einem organismus
EP1644475A4 (de) * 2003-06-20 2009-06-03 Isis Pharmaceuticals Inc Doppelstrang-zusammensetzungen mit einem 3'-endo-modifizierten strang zur verwendung bei der genmodulation
EP1636342A4 (de) * 2003-06-20 2008-10-08 Isis Pharmaceuticals Inc Oligomere verbindungen zur verwendung bei der modulation von genen
US7057100B2 (en) * 2003-06-26 2006-06-06 The J.C. Robinson Seed Co. Inbred corn line W23129
US7025840B1 (en) 2003-07-15 2006-04-11 Lockheed Martin Corporation Explosive/energetic fullerenes
WO2005013901A2 (en) 2003-07-31 2005-02-17 Isis Pharmaceuticals, Inc. Oligomeric compounds and compositions for use in modulation of small non-coding rnas
US7825235B2 (en) 2003-08-18 2010-11-02 Isis Pharmaceuticals, Inc. Modulation of diacylglycerol acyltransferase 2 expression
US20050053981A1 (en) * 2003-09-09 2005-03-10 Swayze Eric E. Gapped oligomeric compounds having linked bicyclic sugar moieties at the termini
US20070123480A1 (en) * 2003-09-11 2007-05-31 Replicor Inc. Oligonucleotides targeting prion diseases
EA009670B1 (ru) 2003-09-18 2008-02-28 Ай Эс Ай Эс ФАРМАСЬЮТИКАЛЗ, ИНК. МОДУЛИРОВАНИЕ ЭКСПРЕССИИ eIF4E
WO2005035548A1 (en) 2003-10-10 2005-04-21 Meditech Research Limited The moduilation of hyaluronan synthesis and degradation in the treatment of disease
US20050191653A1 (en) 2003-11-03 2005-09-01 Freier Susan M. Modulation of SGLT2 expression
CA2747871C (en) 2003-11-17 2018-04-10 Genentech, Inc. Compositions and methods for the treatment of tumor of hematopoietic origin
WO2005071080A2 (en) 2004-01-20 2005-08-04 Isis Pharmaceuticals, Inc. Modulation of glucocorticoid receptor expression
US7468431B2 (en) * 2004-01-22 2008-12-23 Isis Pharmaceuticals, Inc. Modulation of eIF4E-BP2 expression
US8569474B2 (en) * 2004-03-09 2013-10-29 Isis Pharmaceuticals, Inc. Double stranded constructs comprising one or more short strands hybridized to a longer strand
EP2700720A3 (de) 2004-03-15 2015-01-28 Isis Pharmaceuticals, Inc. Zusammensetzungen und Verfahren zur Optimierung der Spaltung von RNA durch RNase H
US20050267300A1 (en) 2004-04-05 2005-12-01 Muthiah Manoharan Processes and reagents for oligonucleotide synthesis and purification
US20050244869A1 (en) * 2004-04-05 2005-11-03 Brown-Driver Vickie L Modulation of transthyretin expression
US20050260755A1 (en) * 2004-04-06 2005-11-24 Isis Pharmaceuticals, Inc. Sequential delivery of oligomeric compounds
US20050260652A1 (en) * 2004-04-15 2005-11-24 The General Hospital Corporation Compositions and methods that modulate RNA interference
EP1750776A2 (de) 2004-04-30 2007-02-14 Alnylam Pharmaceuticals Inc. Oligonukleotide mit c5-modifiziertem pyrimidin
US20080063605A1 (en) * 2004-05-20 2008-03-13 Wai-Fung Cheong Method of Enhancing Visualization of Atherosclerotic Plaque
AU2005326817B2 (en) 2004-05-21 2012-04-19 Benaroya Research Institute Variable lymphocyte receptors, related polypeptides and nucleic acids, and uses thereof
EP1766052A4 (de) * 2004-06-03 2009-12-16 Isis Pharmaceuticals Inc Chimärische oligomere zusammensetzungen mit gap
US8394947B2 (en) * 2004-06-03 2013-03-12 Isis Pharmaceuticals, Inc. Positionally modified siRNA constructs
WO2005121372A2 (en) * 2004-06-03 2005-12-22 Isis Pharmaceuticals, Inc. Double strand compositions comprising differentially modified strands for use in gene modulation
WO2006023880A2 (en) * 2004-08-23 2006-03-02 Isis Pharmaceuticals, Inc. Compounds and methods for the characterization of oligonucleotides
US7884086B2 (en) * 2004-09-08 2011-02-08 Isis Pharmaceuticals, Inc. Conjugates for use in hepatocyte free uptake assays
PL1809303T3 (pl) 2004-09-23 2019-11-29 Arc Medical Devices Inc Kompozycje farmaceutyczne i sposoby dotyczące hamowania zrostów włóknistych lub stanów zapalnych przy użyciu fukanów o niskiej zawartości siarczanów
US20060099712A1 (en) * 2004-11-08 2006-05-11 Eastman Kodak Company Correlation of anti-cancer activity of dyes with redox potentials
JP2006224318A (ja) * 2005-02-15 2006-08-31 Brother Ind Ltd インクジェット記録装置
WO2006098998A2 (en) 2005-03-10 2006-09-21 Genentech, Inc. Methods and compositions for modulating vascular integrity
US7476733B2 (en) * 2005-03-25 2009-01-13 The United States Of America As Represented By The Department Of Health And Human Services Development of a real-time PCR assay for detection of pneumococcal DNA and diagnosis of pneumococccal disease
US20100028439A1 (en) * 2005-05-23 2010-02-04 Elan Pharma International Limited Nanoparticulate stabilized anti-hypertensive compositions
WO2007008300A2 (en) 2005-05-31 2007-01-18 ECOLE POLYTECHNIQUE FéDéRALE DE LAUSANNE Triblock copolymers for cytoplasmic delivery of gene-based drugs
WO2006138145A1 (en) 2005-06-14 2006-12-28 Northwestern University Nucleic acid functionalized nanoparticles for therapeutic applications
US20070072838A1 (en) * 2005-09-26 2007-03-29 Pharmacyclics, Inc. High-purity texaphyrin metal complexes
US20070078119A1 (en) * 2005-09-30 2007-04-05 Pharmacyclics, Inc. Storage system for texaphyrin pharmaceutical formulations
US8080534B2 (en) * 2005-10-14 2011-12-20 Phigenix, Inc Targeting PAX2 for the treatment of breast cancer
EP2402435A3 (de) 2005-10-14 2012-03-28 MUSC Foundation For Research Development Targeting von PAX2 zur Induktion der DEFB1-vermittelten Tumorimmunität und Krebstherapie
JP5111385B2 (ja) 2005-10-28 2013-01-09 アルナイラム ファーマシューティカルズ, インコーポレイテッド ハンチンチン遺伝子の発現を抑制するための組成物および方法
EP1945270B1 (de) 2005-11-09 2011-05-25 Alnylam Pharmaceuticals Inc. Zusammensetzungen und verfahren zur hemmung der expression von faktor v leiden mutationsgen
WO2007062380A2 (en) 2005-11-21 2007-05-31 Isis Pharmaceuticals, Inc. Modulation of eif4e-bp2 expression
WO2007114843A2 (en) * 2006-03-31 2007-10-11 North Carolina State University Light activated antiviral materials and devices and methods for decontaminating virus infected environments
CA2647728C (en) 2006-03-31 2014-12-09 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of eg5 gene
BRPI0711182A2 (pt) * 2006-05-03 2011-08-23 Baltic Technology Dev agentes anti-sentido combinando base fortemente ligada-oligonucleotìdeo modificado e nuclease artificial
EP2392583A1 (de) 2006-05-19 2011-12-07 Alnylam Europe AG. RNAi-Modulation von AHA und therapeutische Verwendungen davon
WO2007137220A2 (en) 2006-05-22 2007-11-29 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of ikk-b gene
WO2007137301A2 (en) * 2006-05-23 2007-11-29 Isis Pharmaceuticals, Inc. Modulation of chrebp expression
US20090280188A1 (en) * 2006-06-23 2009-11-12 Northwestern University Asymmetric functionalizated nanoparticles and methods of use
WO2008011473A2 (en) 2006-07-19 2008-01-24 Isis Pharmaceuticals, Inc. Compositions and their uses directed to hbxip
US8133474B2 (en) * 2006-09-15 2012-03-13 Massachusetts Institute Of Technology Sensors for fluorescence and magnetic resonance imaging
WO2008136852A2 (en) 2006-11-01 2008-11-13 University Of Rochester Methods and compositions related to the structure and function of apobec3g
KR20090089462A (ko) * 2006-12-11 2009-08-21 유니버시티 오브 유타 리써치 파운데이션 병적 혈관형성 및 혈관 투과성을 치료하기 위한 조성물 및 방법
US10182833B2 (en) 2007-01-08 2019-01-22 Ekos Corporation Power parameters for ultrasonic catheter
MX2009008470A (es) * 2007-02-09 2009-11-26 Univ Northwestern Particulas para detectar objetivos intracelulares.
AP2014007971A0 (en) 2007-03-29 2014-09-30 Alnylam Pharmaceuticals Inc Compositions and methods for inhibiting expressionof a gene from the ebola
EP2160464B1 (de) 2007-05-30 2014-05-21 Northwestern University Nukleinsäure-funktionalisierte nanopartikel für therapeutische anwendungen
ES2471118T3 (es) 2007-06-22 2014-06-25 Ekos Corporation Método y aparato para el tratamiento de hemorragias intracraneales
US8580845B2 (en) * 2007-06-29 2013-11-12 Board Of Regents, The University Of Texas System Functionalized expanded porphyrins
DK2682400T5 (da) * 2007-08-28 2017-11-27 Uab Research Foundation Syntetiske apolipoprotein E-efterlignende polypeptider og fremgangsmåder til anvendelse
WO2009032702A2 (en) 2007-08-28 2009-03-12 Uab Research Foundation Synthetic apolipoprotein e mimicking polypeptides and methods of use
WO2009039466A1 (en) 2007-09-20 2009-03-26 Vanderbilt University Free solution measurement of molecular interactions by backscattering interferometry
US7951785B2 (en) * 2007-09-21 2011-05-31 California Institute Of Technology NFIA in glial fate determination, glioma therapy and astrocytoma treatment
MX2010003465A (es) 2007-10-02 2010-07-05 Amgen Inc Incremento de eritropoyetina utilizando acidos nucleicos que hibridan para micro-arn y precursores de los mismos.
CN101983241A (zh) * 2007-11-05 2011-03-02 波罗的科技发展有限公司 带有修饰碱基的寡核苷酸在核酸杂交中的应用
US8916531B2 (en) * 2007-11-20 2014-12-23 Isis Pharmaceuticals, Inc. Modulation of CD40 expression
US7871985B2 (en) 2007-12-10 2011-01-18 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of factor VII gene
US20090187137A1 (en) * 2007-12-14 2009-07-23 Kim Volz Ultrasound pulse shaping
EP2712926A2 (de) 2008-03-05 2014-04-02 Alnylam Pharmaceuticals, Inc. Zusammensetzungen und Verfahren zur Hemmung der Expression des Eg5- und des VEGF-Gens
EP2105145A1 (de) * 2008-03-27 2009-09-30 ETH Zürich Verfahren zur muskelspezifischen Abgabe lipidkonjugierter Oligonukleotide
WO2009124295A2 (en) * 2008-04-04 2009-10-08 Isis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleosides and having reduced toxicity
AU2009234266B2 (en) 2008-04-11 2015-08-06 Tekmira Pharmaceuticals Corporation Site-specific delivery of nucleic acids by combining targeting ligands with endosomolytic components
US8324366B2 (en) 2008-04-29 2012-12-04 Alnylam Pharmaceuticals, Inc. Compositions and methods for delivering RNAI using lipoproteins
US20110237646A1 (en) * 2008-08-07 2011-09-29 Isis Pharmaceuticals, Inc. Modulation of transthyretin expression for the treatment of cns related disorders
CN103429270B (zh) 2008-08-25 2016-11-23 埃克斯雷德制药有限公司 阻止结缔组织生长因子的反义核苷酸及其用途
EP2690175B1 (de) 2008-09-02 2016-12-28 Alnylam Pharmaceuticals, Inc. Zusammensetzungen und Verfahren zur kombinierten Hemmung der Expression eines mutierenden EGFR-Gens und IL-6
AU2009298802A1 (en) 2008-09-23 2010-04-08 Alnylam Pharmaceuticals, Inc. Chemical modifications of monomers and oligonucleotides with cycloaddition
WO2010036962A1 (en) 2008-09-25 2010-04-01 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of serum amyloid a gene
HUE037875T2 (hu) 2008-10-20 2018-09-28 Alnylam Pharmaceuticals Inc Transztiretin-expresszió gátlására szolgáló készítmények és eljárások
EP2447274B1 (de) 2008-10-24 2017-10-04 Ionis Pharmaceuticals, Inc. Oligomerverbindungen und Verfahren
US8999351B2 (en) 2008-11-10 2015-04-07 Tekmira Pharmaceuticals Corporation Lipids and compositions for the delivery of therapeutics
EP3335705A1 (de) 2008-11-24 2018-06-20 Northwestern University Polyvalente rna-nanopartikelzusammensetzungen
US8802074B2 (en) 2008-11-26 2014-08-12 Board Of Regents, The University Of Texas System Polymers functionalized with ion-specific recognition elements
WO2010065792A2 (en) 2008-12-04 2010-06-10 Curna, Inc. Treatment of erythropoietin (epo) related diseases by inhibition of natural antisense transcript to epo
MX358603B (es) 2008-12-04 2018-08-28 Curna Inc Tratamiento de enfermedades relacionadas con un gen supresor de tumor mediante inhibicion del transcrito antisentido natural para el gen.
KR101749352B1 (ko) 2008-12-04 2017-06-20 큐알엔에이, 인크. Sirt1에 대한 천연 안티센스 전사체의 억제에 의해 sirt1 관련된 질환의 치료
NZ593618A (en) 2008-12-10 2013-02-22 Alnylam Pharmaceuticals Inc Gnaq targeted dsrna compositions and methods for inhibiting expression
CA2749536A1 (en) * 2009-01-08 2010-07-15 Northwestern University Inhibition of bacterial protein production by polyvalent oligonucleotide modified nanoparticle conjugates
US20100233270A1 (en) 2009-01-08 2010-09-16 Northwestern University Delivery of Oligonucleotide-Functionalized Nanoparticles
EP2393825A2 (de) 2009-02-06 2011-12-14 Isis Pharmaceuticals, Inc. Oligomere verbindungen und verfahren
DK2396038T3 (en) 2009-02-12 2016-02-01 Curna Inc TREATMENT OF BRAIN-DERIVATED NEUROTROPHIC FACTOR- (BDNF) RELATED DISEASES BY INHIBITION OF NATURAL ANTISENCE TRANSCRIPTION TO BDNF
KR101805199B1 (ko) 2009-02-12 2017-12-05 큐알엔에이, 인크. 신경교세포 유래된 신경영양성 인자 (gdnf)에 대한 자연 안티센스 전사체의 저해에 의한 신경교세포 유래된 신경영양성 인자 (gdnf) 관련된 질환의 치료
US20120041051A1 (en) 2009-02-26 2012-02-16 Kevin Fitzgerald Compositions And Methods For Inhibiting Expression Of MIG-12 Gene
EP3424939A1 (de) 2009-03-02 2019-01-09 Alnylam Pharmaceuticals Inc. Chemische nukleinsäuremodifikationen
ES2845644T3 (es) 2009-03-04 2021-07-27 Curna Inc Tratamiento de enfermedades relacionadas con sirtuina1 (SIRT1) mediante inhibición del transcrito del antisentido natural a sirtuina 1
CA2754043A1 (en) 2009-03-12 2010-09-16 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of eg5 and vegf genes
MX2011009751A (es) 2009-03-16 2011-09-29 Opko Curna Llc Tratamiento de enfermedades relacionadas con factor nuclear (eritroide derivado 2) tipo 2 (nrf2) por inhibicion del transcrito antisentido natural a nrf2.
MX2011009752A (es) 2009-03-17 2011-09-29 Opko Curna Llc Tratamiento de enfermedades relacionadas a homologo tipo delta 1(dlk1) por inhibicion de transcrito antisentido natural a homologo tipo delta (dlk1).
EP3524275A1 (de) 2009-04-22 2019-08-14 Massachusetts Institute Of Technology Angeborene immununterdrückung ermöglicht die wiederholte verabreichung von langen rna-molekülen
WO2010127195A2 (en) 2009-05-01 2010-11-04 Curna, Inc. Antisense oligonucleotides of hemoglobins
ES2609655T3 (es) 2009-05-06 2017-04-21 Curna, Inc. Tratamiento de enfermedades relacionadas con tristetraprolina (TTP) mediante inhibición de transcrito antisentido natural para TTP
JP5883782B2 (ja) 2009-05-06 2016-03-15 クルナ・インコーポレーテッド 脂質輸送代謝遺伝子に対する天然アンチセンス転写物の抑制による脂質輸送代謝遺伝子関連疾患の治療
CN102575251B (zh) 2009-05-18 2018-12-04 库尔纳公司 通过抑制针对重编程因子的天然反义转录物来治疗重编程因子相关的疾病
WO2010135695A2 (en) 2009-05-22 2010-11-25 Curna, Inc. TREATMENT OF TRANSCRIPTION FACTOR E3 (TFE3) and INSULIN RECEPTOR SUBSTRATE 2 (IRS2) RELATED DISEASES BY INHIBITION OF NATURAL ANTISENSE TRANSCRIPT TO TFE3
JP5960049B2 (ja) 2009-05-28 2016-08-02 クルナ・インコーポレーテッド 抗ウイルス遺伝子に対する天然アンチセンス転写物の抑制による抗ウイルス遺伝子関連疾患の治療
MX2011013320A (es) 2009-06-10 2012-02-28 Alnylam Pharmaceuticals Inc Formulacion mejorada de lipido.
ES2620960T3 (es) 2009-06-16 2017-06-30 Curna, Inc. Tratamiento de enfermedades relacionadas con un gen de colágeno mediante la inhibición de un transcrito antisentido natural a un gen de colágeno
US8951981B2 (en) 2009-06-16 2015-02-10 Curna, Inc. Treatment of paraoxonase 1 (PON1) related diseases by inhibition of natural antisense transcript to PON1
EP2442816A4 (de) 2009-06-17 2013-10-02 Isis Pharmaceuticals Inc Zusammensetzungen und verfahren zur modulation der smn2-aufspaltung im körper eines patienten
KR101807323B1 (ko) 2009-06-24 2017-12-08 큐알엔에이, 인크. Tnfr2에 대한 천연 안티센스 전사체의 억제에 의한 종양 괴사 인자 수용체 2(tnfr2) 관련된 질환의 치료
CN102482672B (zh) 2009-06-26 2016-11-09 库尔纳公司 通过抑制唐氏综合征基因的天然反义转录物治疗唐氏综合征基因相关疾病
US9234199B2 (en) 2009-08-05 2016-01-12 Curna, Inc. Treatment of insulin gene (INS) related diseases by inhibition of natural antisense transcript to an insulin gene (INS)
AP2015008874A0 (en) 2009-08-14 2015-11-30 Alnylam Pharmaceuticals Inc Lipid formulated compositions and methods for inhibiting expression of a gene from the ebola virus
CN102482671B (zh) 2009-08-25 2017-12-01 库尔纳公司 通过抑制‘含有iq模体的gtp酶激活蛋白’(iqgap)的天然反义转录物而治疗iqgap相关疾病
US9321823B2 (en) 2009-09-02 2016-04-26 Genentech, Inc. Mutant smoothened and methods of using the same
BR112012009409A2 (pt) 2009-10-22 2017-02-21 Genentech Inc método de identificação de uma substância inibidora, molécula antagonista, ácido nucleico isolado, vetor, célula hospedeira, método para fabricar a molécula, composição, artigo de fabricação, método de inibição de uma atividade biológica, método de tratamento de uma condição patológica, método para detectar msp em uma amostra e método para detectar hepsina em uma amostra
EP2494075B1 (de) 2009-10-30 2018-04-04 Northwestern University Templatgesteuerte nanokonjugate
RU2015132142A (ru) 2009-11-30 2015-12-20 Дженентек, Инк. Композиции и способы для диагностики и лечения опухоли
US9687550B2 (en) 2009-12-07 2017-06-27 Arbutus Biopharma Corporation Compositions for nucleic acid delivery
WO2011084455A2 (en) 2009-12-16 2011-07-14 Opko Curna, Llc. Treatment of membrane bound transcription factor peptidase, site 1 (mbtps1) related diseases by inhibition of natural antisense transcript to mbtps1
ES2749426T3 (es) 2009-12-18 2020-03-20 Univ British Columbia Métodos y composiciones para administración de ácidos nucleicos
CN102869776B (zh) 2009-12-23 2017-06-23 库尔纳公司 通过抑制肝细胞生长因子(hgf)的天然反义转录物而治疗hgf相关疾病
KR101793753B1 (ko) 2009-12-23 2017-11-03 큐알엔에이, 인크. 커플링방지 단백질 2(ucp2)에 대한 천연 안티센스 전사체의 저해에 의한 ucp2 관련 질환의 치료
RU2615450C2 (ru) 2009-12-29 2017-04-04 Курна, Инк. Лечение заболеваний, связанных с ядерным респираторным фактором 1(nrf1), путем ингибирования природного антисмыслового транскрипта к nrf1
KR101853508B1 (ko) 2009-12-29 2018-06-20 큐알엔에이, 인크. 종양 단백질 63 (p63)에 대한 천연 안티센스 전사체의 억제에 의한 p63에 관련된 질환의 치료
ES2664605T3 (es) 2010-01-04 2018-04-20 Curna, Inc. Tratamiento de enfermedades relacionadas con el factor 8 regulador del interferón (irf8) mediante inhibición del transcrito antisentido natural al gen irf8
WO2011085066A2 (en) 2010-01-06 2011-07-14 Curna, Inc. Treatment of pancreatic developmental gene related diseases by inhibition of natural antisense transcript to a pancreatic developmental gene
CN102803493B (zh) 2010-01-11 2018-07-31 库尔纳公司 通过抑制性激素结合球蛋白(shbg)的天然反义转录物而治疗shbg相关疾病
WO2011091390A2 (en) 2010-01-25 2011-07-28 Opko Curna, Llc Treatment of rnase h1 related diseases by inhibition of natural antisense transcript to rnase h1
WO2011100131A2 (en) 2010-01-28 2011-08-18 Alnylam Pharmacuticals, Inc. Monomers and oligonucleotides comprising cycloaddition adduct(s)
WO2011094580A2 (en) 2010-01-28 2011-08-04 Alnylam Pharmaceuticals, Inc. Chelated copper for use in the preparation of conjugated oligonucleotides
WO2011097407A1 (en) 2010-02-04 2011-08-11 Ico Therapeutics Inc. Dosing regimens for treating and preventing ocular disorders using c-raf antisense
KR101838308B1 (ko) 2010-02-22 2018-03-13 큐알엔에이, 인크. 피롤린-5-카르복실레이트 환원효소 1(pycr1)에 대한 천연 안티센스 전사체의 억제에 의한 pycr1과 관련된 질환의 치료
WO2011105900A2 (en) 2010-02-23 2011-09-01 Academisch Ziekenhuis Bij De Universiteit Van Amsterdam Antagonists of complement component 8-alpha (c8-alpha) and uses thereof
EP2538981B1 (de) 2010-02-23 2017-12-20 F. Hoffmann-La Roche AG Zusammensetzungen und methoden zur diagnose und behandlung von tumoren
WO2011105902A2 (en) 2010-02-23 2011-09-01 Academisch Ziekenhuis Bij De Universiteit Van Amsterdam Antagonists of complement component 8-beta (c8-beta) and uses thereof
WO2011105901A2 (en) 2010-02-23 2011-09-01 Academisch Ziekenhuis Bij De Universiteit Van Amsterdam Antagonists of complement component 9 (c9) and uses thereof
WO2011112516A1 (en) 2010-03-08 2011-09-15 Ico Therapeutics Inc. Treating and preventing hepatitis c virus infection using c-raf kinase antisense oligonucleotides
WO2011112732A2 (en) 2010-03-12 2011-09-15 The Brigham And Women's Hospital, Inc. Methods of treating vascular inflammatory disorders
CA2792291A1 (en) 2010-03-29 2011-10-06 Kumamoto University Sirna therapy for transthyretin (ttr) related ocular amyloidosis
WO2011123621A2 (en) 2010-04-01 2011-10-06 Alnylam Pharmaceuticals Inc. 2' and 5' modified monomers and oligonucleotides
JP5978203B2 (ja) 2010-04-09 2016-08-24 カッパーアールエヌエー,インコーポレイテッド Fgf21に対する天然アンチセンス転写物の阻害による線維芽細胞増殖因子(fgf21)線維芽細胞増殖因子fgf21)関連疾患の治療
WO2011133876A2 (en) 2010-04-22 2011-10-27 Alnylam Pharmaceuticals, Inc. Oligonucleotides comprising acyclic and abasic nucleosides and analogs
US20130260460A1 (en) 2010-04-22 2013-10-03 Isis Pharmaceuticals Inc Conformationally restricted dinucleotide monomers and oligonucleotides
US9725479B2 (en) 2010-04-22 2017-08-08 Ionis Pharmaceuticals, Inc. 5′-end derivatives
US8993738B2 (en) 2010-04-28 2015-03-31 Isis Pharmaceuticals, Inc. Modified nucleosides, analogs thereof and oligomeric compounds prepared therefrom
EP2606057B1 (de) 2010-04-28 2016-06-15 Ionis Pharmaceuticals, Inc. Modifizierte 5' diphosphat-nukleoside und daraus hergestellte oligomere verbindungen
WO2011139699A2 (en) 2010-04-28 2011-11-10 Isis Pharmaceuticals, Inc. 5' modified nucleosides and oligomeric compounds prepared therefrom
CN103038345B (zh) 2010-04-29 2017-07-21 Ionis制药公司 转甲状腺素蛋白表达的调节
WO2011139911A2 (en) 2010-04-29 2011-11-10 Isis Pharmaceuticals, Inc. Lipid formulated single stranded rna
RU2018110641A (ru) 2010-05-03 2019-02-27 Курна, Инк. Лечение заболеваний, связанных с сиртуином (sirt), путем ингибирования природного антисмыслового транскрипта к сиртуину (sirt)
KR20130079384A (ko) 2010-05-03 2013-07-10 제넨테크, 인크. 종양의 진단 및 치료를 위한 조성물 및 방법
TWI586356B (zh) 2010-05-14 2017-06-11 可娜公司 藉由抑制par4天然反股轉錄本治療par4相關疾病
US8895528B2 (en) 2010-05-26 2014-11-25 Curna, Inc. Treatment of atonal homolog 1 (ATOH1) related diseases by inhibition of natural antisense transcript to ATOH1
US9944671B2 (en) 2010-06-02 2018-04-17 Alnylam Pharmaceuticals, Inc. Compositions and methods directed to treating liver fibrosis
WO2011156713A1 (en) 2010-06-11 2011-12-15 Vanderbilt University Multiplexed interferometric detection system and method
US20130236968A1 (en) 2010-06-21 2013-09-12 Alnylam Pharmaceuticals, Inc. Multifunctional copolymers for nucleic acid delivery
ES2663598T3 (es) 2010-07-14 2018-04-16 Curna, Inc. Tratamiento de enfermedades relacionadas con el homólogo de discos grandes (dlg) mediante la inhibición del transcrito antisentido natural a dlg
US20130202652A1 (en) 2010-07-30 2013-08-08 Alnylam Pharmaceuticals, Inc. Methods and compositions for delivery of active agents
US20130323269A1 (en) 2010-07-30 2013-12-05 Muthiah Manoharan Methods and compositions for delivery of active agents
SG188666A1 (en) 2010-09-30 2013-05-31 Agency Science Tech & Res Methods and reagents for detection and treatment of esophageal metaplasia
ES2588981T3 (es) 2010-10-05 2016-11-08 Genentech, Inc. Smoothened mutante y métodos de uso de la misma
ES2640755T3 (es) 2010-10-06 2017-11-06 Curna, Inc. Tratamiento de enfermedades relacionadas con la Sialidasa 4 (neu4) mediante inhibición del transcrito antisentido natural al gen neu4
US20140031250A1 (en) 2010-10-07 2014-01-30 David Tsai Ting Biomarkers of Cancer
CA2815212A1 (en) 2010-10-22 2012-04-26 Curna, Inc. Treatment of alpha-l-iduronidase (idua) related diseases by inhibition of natural antisense transcript to idua
JP6073795B2 (ja) 2010-10-27 2017-02-01 カッパーアールエヌエー,インコーポレイテッド インターフェロン関連発生制御因子1(ifrd1)への天然アンチセンス転写物の阻害によるifrd1関連疾患の治療
US9339513B2 (en) 2010-11-09 2016-05-17 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of Eg5 and VEGF genes
AU2011325956B2 (en) 2010-11-12 2016-07-14 The General Hospital Corporation Polycomb-associated non-coding RNAs
CA2818824A1 (en) 2010-11-23 2012-05-31 Joseph Collard Treatment of nanog related diseases by inhibition of natural antisense transcript to nanog
EP2648763A4 (de) 2010-12-10 2014-05-14 Alnylam Pharmaceuticals Inc Zusammensetzungen und verfahren zur expressionshemmung der gene klf-1 und bcl11a
WO2012078967A2 (en) 2010-12-10 2012-06-14 Alnylam Pharmaceuticals, Inc. Compositions and methods for increasing erythropoietin (epo) production
AU2012207606B2 (en) 2011-01-11 2017-02-23 Alnylam Pharmaceuticals, Inc. Pegylated lipids and their use for drug delivery
US9045749B2 (en) 2011-01-14 2015-06-02 The General Hospital Corporation Methods targeting miR-128 for regulating cholesterol/lipid metabolism
WO2012106508A1 (en) 2011-02-02 2012-08-09 Pfizer Inc. Method of treating keloids or hypertrophic scars using antisense compounds targeting connective tissue growth factor (ctgf)
EP2678026B1 (de) 2011-02-21 2016-05-18 The University of Zurich Ankyrin-g zur verwendung in der behandlung von neurodegenerativer erkrankungen
US9562853B2 (en) 2011-02-22 2017-02-07 Vanderbilt University Nonaqueous backscattering interferometric methods
MX360349B (es) 2011-03-29 2018-10-30 Alnylam Pharmaceuticals Inc Composiciones y metodos para inhibir la expresion del gen de proteasa transmembrana, serina 6 (tmprss6).
ES2653247T3 (es) 2011-06-09 2018-02-06 Curna, Inc. Tratamiento de enfermedades relacionadas con la frataxina (FXN) mediante inhibición del transcrito antisentido natural al gen FXN
EP3444348A1 (de) 2011-06-21 2019-02-20 Alnylam Pharmaceuticals, Inc. Angiopoietin-3 (angptl3)-irna-zusammensetzungen und verfahren zur verwendung davon
EP3388068A1 (de) 2011-06-21 2018-10-17 Alnylam Pharmaceuticals, Inc. Zusammensetzung und verfahren zur hemmung der expression von protein-c (proc)-genen
WO2012177947A2 (en) 2011-06-21 2012-12-27 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibition of expression of apolipoprotein c-iii (apoc3) genes
EP3597750B1 (de) 2011-06-23 2022-05-04 Alnylam Pharmaceuticals, Inc. Serpina1-sirnas: materialzusammensetzungen und behandlungsverfahren
JP2014526887A (ja) 2011-08-01 2014-10-09 アルナイラム ファーマシューティカルズ, インコーポレイテッド 造血幹細胞移植の成功率を改善する方法
AU2012308302A1 (en) 2011-09-14 2014-03-20 Northwestern University Nanoconjugates able to cross the blood-brain barrier
EP2756080B1 (de) 2011-09-14 2019-02-20 Translate Bio MA, Inc. Multimere oligonukleotidverbindungen
EP3456317A1 (de) 2011-09-27 2019-03-20 Alnylam Pharmaceuticals, Inc. Substituierte di-aliphatic pegylierte lipide
JP6234370B2 (ja) 2011-10-11 2017-11-22 ザ ブリガム アンド ウィメンズ ホスピタル インコーポレイテッドThe Brigham and Women’s Hospital, Inc. 神経変性障害におけるマイクロrna
EP2790736B1 (de) 2011-12-12 2018-01-31 Oncoimmunin, Inc. In-vivo-verabreichung von oligonucleotiden
CN110201187A (zh) 2011-12-16 2019-09-06 现代泰克斯公司 经修饰的核苷、核苷酸和核酸组合物
CN110438125A (zh) 2012-03-15 2019-11-12 科纳公司 通过抑制脑源神经营养因子(bdnf)的天然反义转录物治疗bdnf相关疾病
AU2013243952A1 (en) 2012-04-02 2014-10-30 Modernatx, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
CA2868391A1 (en) 2012-04-02 2013-10-10 Stephane Bancel Polynucleotides comprising n1-methyl-pseudouridine and methods for preparing the same
US9133461B2 (en) 2012-04-10 2015-09-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the ALAS1 gene
CN107083385A (zh) 2012-04-20 2017-08-22 艾珀特玛治疗公司 产热的miRNA调节剂
US9127274B2 (en) 2012-04-26 2015-09-08 Alnylam Pharmaceuticals, Inc. Serpinc1 iRNA compositions and methods of use thereof
WO2013163628A2 (en) 2012-04-27 2013-10-31 Duke University Genetic correction of mutated genes
WO2013169640A1 (en) * 2012-05-05 2013-11-14 Academia Sinica Diaminobenzoic or ortho-phenylene diamine conjugated peptides as functional saccharide tagging reagents
US9273949B2 (en) 2012-05-11 2016-03-01 Vanderbilt University Backscattering interferometric methods
AU2013262649A1 (en) 2012-05-16 2015-01-22 Rana Therapeutics, Inc. Compositions and methods for modulating smn gene family expression
KR102028784B1 (ko) 2012-05-16 2019-10-04 트랜슬레이트 바이오 인코포레이티드 유전자 발현을 조절하기 위한 조성물 및 방법
US20150216892A1 (en) 2012-08-03 2015-08-06 Aptamir Therapeutics, Inc. Cell-specific delivery of mirna modulators for the treatment of obesity and related disorders
PT2922554T (pt) 2012-11-26 2022-06-28 Modernatx Inc Arn modificado nas porções terminais
EP3434774A1 (de) 2013-01-17 2019-01-30 ModernaTX, Inc. Signal-sensor-polynukleotide zur veränderung zellulärer phänotypen
US20150366890A1 (en) 2013-02-25 2015-12-24 Trustees Of Boston University Compositions and methods for treating fungal infections
US20160024181A1 (en) 2013-03-13 2016-01-28 Moderna Therapeutics, Inc. Long-lived polynucleotide molecules
US10258698B2 (en) 2013-03-14 2019-04-16 Modernatx, Inc. Formulation and delivery of modified nucleoside, nucleotide, and nucleic acid compositions
KR20230162998A (ko) 2013-03-14 2023-11-29 알닐람 파마슈티칼스 인코포레이티드 보체 성분 C5 iRNA 조성물 및 그 이용 방법
US8980864B2 (en) 2013-03-15 2015-03-17 Moderna Therapeutics, Inc. Compositions and methods of altering cholesterol levels
US9937231B2 (en) 2013-03-27 2018-04-10 The General Hospital Corporation Methods and agents for treating Alzheimer's disease
US10590412B2 (en) 2013-04-19 2020-03-17 Ionis Pharmaceuticals, Inc. Compositions and methods for modulation nucleic acids through nonsense mediated decay
PL2999785T3 (pl) 2013-05-22 2018-09-28 Alnylam Pharmaceuticals, Inc. Kompozycje iRNA Serpina1 i sposoby ich zastosowania
US20160129089A1 (en) 2013-06-13 2016-05-12 Antisense Therapeutics Ltd Combination therapy
PT3019619T (pt) 2013-07-11 2021-11-11 Modernatx Inc Composições que compreendem polinucleótidos sintéticos que codificam proteínas relacionadas com crispr e sgarn sintéticos e métodos de utilização
SI3041854T1 (sl) 2013-08-08 2020-03-31 The Scripps Research Institute Postopek za mestnospecifično encimsko označevanje nukleinskih kislin in vitro z vključevanjem nenaravnih nukleotidov
US20160194368A1 (en) 2013-09-03 2016-07-07 Moderna Therapeutics, Inc. Circular polynucleotides
AU2014315287A1 (en) 2013-09-03 2015-03-12 Moderna Therapeutics, Inc. Chimeric polynucleotides
TWI669393B (zh) 2013-10-02 2019-08-21 艾爾妮蘭製藥公司 抑制lect2基因表現之組合物及方法
US10323076B2 (en) 2013-10-03 2019-06-18 Modernatx, Inc. Polynucleotides encoding low density lipoprotein receptor
EA036477B1 (ru) 2013-10-04 2020-11-16 Элнилэм Фармасьютикалз, Инк. Композиции и способы ингибирования экспрессии гена alas1
CN107075515B (zh) 2013-11-22 2020-10-30 米纳治疗有限公司 C/EBPα组合物和使用方法
CA2844640A1 (en) 2013-12-06 2015-06-06 The University Of British Columbia Method for treatment of castration-resistant prostate cancer
BR112016013148B1 (pt) 2013-12-12 2024-02-27 Alnylam Pharmaceuticals, Inc ÁCIDO RIBONUCLEICO DE FITA DUPLA (dsRNA) PARA INIBIÇÃO DA EXPRESSÃO DO FATOR B DO COMPLEMENTO (CFB) EM UMA CÉLULA, COMPOSIÇÃO FARMACÊUTICA PARA INIBIÇÃO DA EXPRESSÃO DE UM GENE DO FATOR B DO COMPLEMENTO, SEU USO, E MÉTODO IN VITRO DE INIBIÇÃO DA EXPRESSÃO DO FATOR B DO COMPLEMENTO (CFB) EM UMA CÉLULA
CA2937539A1 (en) 2014-02-04 2015-08-13 Genentech, Inc. Mutant smoothened and methods of using the same
EP3960860A3 (de) 2014-02-11 2022-06-08 Alnylam Pharmaceuticals, Inc. Ketohexokinase (khk)-irna-zusammensetzungen und verfahren zur verwendung davon
DK3129493T3 (da) 2014-04-09 2021-09-27 Scripps Research Inst Import af unaturlige eller modificerede nukleosidtriphosphater ind i celler via nukleinsyre-triphosphat-transportører
EP4162940A1 (de) 2014-04-17 2023-04-12 Biogen MA Inc. Zusammensetzungen und verfahren zur modulation der smn2-aufspaltung in einem patienten
WO2015171918A2 (en) 2014-05-07 2015-11-12 Louisiana State University And Agricultural And Mechanical College Compositions and uses for treatment thereof
WO2015175510A1 (en) 2014-05-12 2015-11-19 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating a serpinc1-associated disorder
SG10202104570TA (en) 2014-05-22 2021-06-29 Alnylam Pharmaceuticals Inc Angiotensinogen (agt) irna compositions and methods of use thereof
ES2908962T3 (es) 2014-06-10 2022-05-04 Univ Erasmus Med Ct Rotterdam Oligonucleótidos antisentido útiles en el tratamiento de la enfermedad de Pompe
CN116178456A (zh) 2014-06-11 2023-05-30 德克萨斯州大学系统董事会 用于克服铂耐受性的德克萨卟啉-pt(iv)缀合物及组合物
WO2015200697A1 (en) 2014-06-25 2015-12-30 The General Hospital Corporation Targeting human satellite ii (hsatii)
US20170204152A1 (en) 2014-07-16 2017-07-20 Moderna Therapeutics, Inc. Chimeric polynucleotides
WO2016014846A1 (en) 2014-07-23 2016-01-28 Moderna Therapeutics, Inc. Modified polynucleotides for the production of intrabodies
AU2015298263B2 (en) 2014-07-31 2020-05-14 Anji Pharmaceuticals, Inc. ApoE mimetic peptides and higher potency to clear plasma cholesterol
EP3185910A4 (de) 2014-08-29 2018-01-10 Children's Medical Center Corporation Verfahren und zusammensetzungen zur krebsbehandlung
WO2016033424A1 (en) 2014-08-29 2016-03-03 Genzyme Corporation Methods for the prevention and treatment of major adverse cardiovascular events using compounds that modulate apolipoprotein b
EP3191591A1 (de) 2014-09-12 2017-07-19 Alnylam Pharmaceuticals, Inc. Gegen komplementkomponente c5 gerichtete polynukleotidwirkstoffe und verfahren zur verwendung davon
US10556020B2 (en) 2014-09-26 2020-02-11 University Of Massachusetts RNA-modulating agents
WO2016061487A1 (en) 2014-10-17 2016-04-21 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting aminolevulinic acid synthase-1 (alas1) and uses thereof
EP3904519A1 (de) 2014-10-30 2021-11-03 Genzyme Corporation Polynukleotidwirkstoffe mit abzielung auf serpinc1 (at3) und verfahren zur verwendung davon
JOP20200092A1 (ar) 2014-11-10 2017-06-16 Alnylam Pharmaceuticals Inc تركيبات iRNA لفيروس الكبد B (HBV) وطرق لاستخدامها
EP3221451A1 (de) 2014-11-17 2017-09-27 Alnylam Pharmaceuticals, Inc. Apolipoprotein-c3 (apoc3)-irna-zusammensetzungen und verfahren zur verwendung davon
US11213593B2 (en) 2014-11-21 2022-01-04 Northwestern University Sequence-specific cellular uptake of spherical nucleic acid nanoparticle conjugates
EP3229842B1 (de) 2014-12-08 2022-07-06 The Board of Regents of The University of Texas System Lipokationische polymere und verwendungen davon
WO2016100716A1 (en) 2014-12-18 2016-06-23 Vasant Jadhav Reversirtm compounds
EP3247988A4 (de) 2015-01-23 2018-12-19 Vanderbilt University Robustes interferometer und verfahren zur verwendung davon
EP3256487A4 (de) 2015-02-09 2018-07-18 Duke University Zusammensetzungen und verfahren zur epigenombearbeitung
CA2976445A1 (en) 2015-02-13 2016-08-18 Alnylam Pharmaceuticals, Inc. Patatin-like phospholipase domain containing 3 (pnpla3) irna compositions and methods of use thereof
EP3262173A2 (de) 2015-02-23 2018-01-03 Crispr Therapeutics AG Materialien und verfahren zur behandlung von humangenetischen erkrankungen einschliesslich hämoglobinopathien
WO2016164463A1 (en) 2015-04-07 2016-10-13 The General Hospital Corporation Methods for reactivating genes on the inactive x chromosome
US10745702B2 (en) 2015-04-08 2020-08-18 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the LECT2 gene
CA2982853C (en) 2015-04-17 2022-06-07 University Health Network Texaphyrin-phospholipid conjugates and methods of preparing same
WO2016201136A1 (en) 2015-06-10 2016-12-15 Ekos Corporation Ultrasound catheter
WO2016201301A1 (en) 2015-06-12 2016-12-15 Alnylam Pharmaceuticals, Inc. Complement component c5 irna compositions and methods of use thereof
WO2016205323A1 (en) 2015-06-18 2016-12-22 Alnylam Pharmaceuticals, Inc. Polynucleotde agents targeting hydroxyacid oxidase (glycolate oxidase, hao1) and methods of use thereof
WO2016209862A1 (en) 2015-06-23 2016-12-29 Alnylam Pharmaceuticals, Inc. Glucokinase (gck) irna compositions and methods of use thereof
JP2018524588A (ja) 2015-06-26 2018-08-30 ベス・イスラエル・ディーコネス・メディカル・センター,インコーポレイテッド 骨髄由来サプレッサー細胞中のテトラスパニン33(Tspan33)を標的化するがん療法
US10590425B2 (en) 2015-06-29 2020-03-17 Caris Science, Inc. Therapeutic oligonucleotides
WO2017011286A1 (en) 2015-07-10 2017-01-19 Alnylam Pharmaceuticals, Inc. Insulin-like growth factor binding protein, acid labile subunit (igfals) and insulin-like growth factor 1 (igf-1) irna compositions and methods of use thereof
EP3324980B1 (de) 2015-07-17 2021-11-10 Alnylam Pharmaceuticals, Inc. Mehrfach gerichtete einheitlichen konjugate
WO2017019918A1 (en) 2015-07-28 2017-02-02 Caris Science, Inc. Targeted oligonucleotides
KR20180051550A (ko) 2015-09-02 2018-05-16 알닐람 파마슈티칼스 인코포레이티드 프로그램된 세포사 1 리간드 1 (PD-L1) iRNA 조성물 및 그의 사용 방법
KR102641298B1 (ko) 2015-09-14 2024-03-04 더 보드 오브 리젠츠 오브 더 유니버시티 오브 텍사스 시스템 지질양이온성 덴드리머 및 이의 용도
WO2017064546A1 (en) 2015-09-24 2017-04-20 Crispr Therapeutics Ag Novel family of rna-programmable endonucleases and their uses in genome editing and other applications
EP3362571A4 (de) 2015-10-13 2019-07-10 Duke University Genom-engineering mit typ-i-crispr-systemen in eukaryotischen zellen
CA3000931A1 (en) 2015-10-28 2017-05-04 Crispr Therapeutics Ag Materials and methods for treatment of duchenne muscular dystrophy
ES2938883T3 (es) 2015-11-05 2023-04-17 Los Angeles Childrens Hospital Oligo antisentido para su uso en el tratamiento de la leucemia mieloide aguda
CA2999649A1 (en) 2015-11-06 2017-05-11 Crispr Therapeutics Ag Materials and methods for treatment of glycogen storage disease type 1a
US11001622B2 (en) 2015-11-19 2021-05-11 The Brigham And Women's Hospital, Inc. Method of treating autoimmune disease with lymphocyte antigen CD5-like (CD5L) protein
WO2017093804A2 (en) 2015-12-01 2017-06-08 Crispr Therapeutics Ag Materials and methods for treatment of alpha-1 antitrypsin deficiency
WO2017099579A1 (en) 2015-12-07 2017-06-15 Erasmus University Medical Center Rotterdam Enzymatic replacement therapy and antisense therapy for pompe disease
US11761007B2 (en) 2015-12-18 2023-09-19 The Scripps Research Institute Production of unnatural nucleotides using a CRISPR/Cas9 system
MX2018007840A (es) 2015-12-23 2019-05-02 Crispr Therapeutics Ag Materiales y metodos para el tratamiento de la esclerosis lateral amiotrofica y/o la degeneracion lobar frontotemporal.
WO2017132483A1 (en) 2016-01-29 2017-08-03 Vanderbilt University Free-solution response function interferometry
US20190038771A1 (en) 2016-02-02 2019-02-07 Crispr Therapeutics Ag Materials and methods for treatment of severe combined immunodeficiency (scid) or omenn syndrome
CN109071625A (zh) 2016-02-04 2018-12-21 柯瑞斯公司 平滑化突变体和其使用方法
WO2017141109A1 (en) 2016-02-18 2017-08-24 Crispr Therapeutics Ag Materials and methods for treatment of severe combined immunodeficiency (scid) or omenn syndrome
JP7033072B2 (ja) 2016-02-25 2022-03-09 ザ ブリガム アンド ウィメンズ ホスピタル インコーポレイテッド Smoc2を標的化する線維症のための治療方法
WO2017158422A1 (en) 2016-03-16 2017-09-21 Crispr Therapeutics Ag Materials and methods for treatment of hereditary haemochromatosis
JP2019516393A (ja) 2016-03-18 2019-06-20 カリス サイエンス インコーポレイテッド オリゴヌクレオチドプローブおよびその使用
MX2018012729A (es) 2016-04-18 2019-07-04 Crispr Therapeutics Ag Materiales y metodos para el tratamiento de hemoglobinopatias.
WO2017191503A1 (en) 2016-05-05 2017-11-09 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
CA3024129A1 (en) 2016-05-16 2017-11-23 The Board Of Regents Of The University Of Texas System Cationic sulfonamide amino lipids and amphiphilic zwitterionic amino lipids
CA3025486A1 (en) 2016-05-25 2017-11-30 Caris Science, Inc. Oligonucleotide probes and uses thereof
US20190256845A1 (en) 2016-06-10 2019-08-22 Alnylam Pharmaceuticals, Inc. COMPLEMENT COMPONENT C5 iRNA COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
EP3475295B1 (de) 2016-06-24 2022-08-10 The Scripps Research Institute Neuartiger nukleosid-triphosphat-transporter und verwendungen davon
US11174469B2 (en) 2016-06-29 2021-11-16 Crispr Therapeutics Ag Materials and methods for treatment of Amyotrophic Lateral Sclerosis (ALS) and other related disorders
EP3478829A1 (de) 2016-06-29 2019-05-08 Crispr Therapeutics AG Materialien und verfahren zur behandlung von myotoner dystrophie typ 1 (dm1) und anderen erkrankungen
US11564997B2 (en) 2016-06-29 2023-01-31 Crispr Therapeutics Ag Materials and methods for treatment of friedreich ataxia and other related disorders
US11459587B2 (en) 2016-07-06 2022-10-04 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of pain related disorders
JP7305534B2 (ja) 2016-07-06 2023-07-10 バーテックス ファーマシューティカルズ インコーポレイテッド 疼痛関連障害を処置するための物質及び方法
WO2018007871A1 (en) 2016-07-08 2018-01-11 Crispr Therapeutics Ag Materials and methods for treatment of transthyretin amyloidosis
CA3030701A1 (en) 2016-07-11 2018-01-18 Translate Bio Ma, Inc. Nucleic acid conjugates and uses thereof
WO2018020323A2 (en) 2016-07-25 2018-02-01 Crispr Therapeutics Ag Materials and methods for treatment of fatty acid disorders
NL2017294B1 (en) 2016-08-05 2018-02-14 Univ Erasmus Med Ct Rotterdam Natural cryptic exon removal by pairs of antisense oligonucleotides.
NL2017295B1 (en) 2016-08-05 2018-02-14 Univ Erasmus Med Ct Rotterdam Antisense oligomeric compound for Pompe disease
US11459568B2 (en) 2016-10-31 2022-10-04 University Of Massachusetts Targeting microRNA-101-3p in cancer therapy
TW202313978A (zh) 2016-11-23 2023-04-01 美商阿尼拉製藥公司 絲胺酸蛋白酶抑制因子A1 iRNA組成物及其使用方法
CN110191955A (zh) 2016-12-13 2019-08-30 西雅图儿童医院(Dba西雅图儿童研究所) 在体外和体内对工程化的细胞中表达的化学物质诱导的信号传导复合物进行外源性药物激活的方法
KR20230166146A (ko) 2016-12-16 2023-12-06 알닐람 파마슈티칼스 인코포레이티드 트랜스티레틴(TTR) iRNA 조성물을 사용하여 TTR-관련 질병을 치료하거나 예방하는 방법
RU2021133626A (ru) 2017-01-23 2022-01-31 Регенерон Фармасьютикалз, Инк. Варианты hsd17b13 и их применения
WO2018154462A2 (en) 2017-02-22 2018-08-30 Crispr Therapeutics Ag Materials and methods for treatment of spinocerebellar ataxia type 2 (sca2) and other spinocerebellar ataxia type 2 protein (atxn2) gene related conditions or disorders
WO2018154387A1 (en) 2017-02-22 2018-08-30 Crispr Therapeutics Ag Compositions and methods for gene editing
WO2018154459A1 (en) 2017-02-22 2018-08-30 Crispr Therapeutics Ag Materials and methods for treatment of primary hyperoxaluria type 1 (ph1) and other alanine-glyoxylate aminotransferase (agxt) gene related conditions or disorders
WO2018154439A1 (en) 2017-02-22 2018-08-30 Crispr Therapeutics Ag Materials and methods for treatment of spinocerebellar ataxia type 1 (sca1) and other spinocerebellar ataxia type 1 protein (atxn1) gene related conditions or disorders
WO2018154418A1 (en) 2017-02-22 2018-08-30 Crispr Therapeutics Ag Materials and methods for treatment of early onset parkinson's disease (park1) and other synuclein, alpha (snca) gene related conditions or disorders
CA3059446A1 (en) 2017-04-18 2018-10-25 Alnylam Pharmaceuticals, Inc. Methods for the treatment of subjects having a hepatitis b virus (hbv) infection
US20200384115A1 (en) 2017-04-21 2020-12-10 The Broad Institute , Inc. Targeted delivery to beta cells
IL270415B1 (en) 2017-05-12 2024-04-01 Crispr Therapeutics Ag Materials and methods for cell engineering and their uses in immuno-oncology
EP3652316A4 (de) 2017-07-11 2021-04-07 Synthorx, Inc. Einbindung von unnatürlichen nukleotiden und verfahren dafür
AU2018301477A1 (en) 2017-07-13 2020-02-27 Alnylam Pharmaceuticals Inc. Lactate dehydrogenase a (LDHA) iRNA compositions and methods of use thereof
BR112020002271A2 (pt) 2017-08-03 2020-07-28 Synthorx, Inc. conjugados de citocina para o tratamento de doenças proliferativas e infecciosas
WO2019048631A1 (en) 2017-09-08 2019-03-14 Mina Therapeutics Limited SMALL HNF4A ACTIVATOR RNA COMPOSITIONS AND METHODS OF USE
EP4219715A3 (de) 2017-09-08 2023-09-06 MiNA Therapeutics Limited Stabilisierte cebpa-sarna-zusammensetzungen und verfahren zur verwendung
KR20200067190A (ko) 2017-10-17 2020-06-11 크리스퍼 테라퓨틱스 아게 A형 혈우병을 위한 유전자 편집용 조성물 및 방법
EP3701029A1 (de) 2017-10-26 2020-09-02 Vertex Pharmaceuticals Incorporated Materialien und verfahren zur behandlung von hämoglobinopathien
CA3078971A1 (en) 2017-11-01 2019-05-09 Alnylam Pharmaceuticals, Inc. Complement component c3 irna compositions and methods of use thereof
MA50578A (fr) 2017-11-09 2021-09-15 Vertex Pharma Systèmes crispr/cas pour le traitement de dmd
WO2019100039A1 (en) 2017-11-20 2019-05-23 Alnylam Pharmaceuticals, Inc. Serum amyloid p component (apcs) irna compositions and methods of use thereof
US10662425B2 (en) 2017-11-21 2020-05-26 Crispr Therapeutics Ag Materials and methods for treatment of autosomal dominant retinitis pigmentosa
US20200384033A1 (en) 2017-12-05 2020-12-10 Vertex Pharmaceuticals Incorporated Crispr-cas9 modified cd34+ human hematopoietic stem and progenitor cells and uses thereof
AU2018386301A1 (en) 2017-12-14 2020-06-18 Bayer Healthcare Llc Novel RNA-programmable endonuclease systems and their use in genome editing and other applications
EA202091520A1 (ru) 2017-12-18 2020-10-05 Элнилэм Фармасьютикалз, Инк. Композиции на основе irna против бокса-1 высокомобильной группы (hmgb1) и способы их применения
WO2019123430A1 (en) 2017-12-21 2019-06-27 Casebia Therapeutics Llp Materials and methods for treatment of usher syndrome type 2a and/or non-syndromic autosomal recessive retinitis pigmentosa (arrp)
CN111836892A (zh) 2017-12-21 2020-10-27 克里斯珀医疗股份公司 用于治疗2a型乌谢尔综合征的材料和方法
WO2019140330A1 (en) 2018-01-12 2019-07-18 Casebia Therapeutics Limited Liability Partnership Compositions and methods for gene editing by targeting transferrin
WO2019147743A1 (en) 2018-01-26 2019-08-01 Massachusetts Institute Of Technology Structure-guided chemical modification of guide rna and its applications
EP3749768A1 (de) 2018-02-05 2020-12-16 Vertex Pharmaceuticals Incorporated Materialien und verfahren zur behandlung von hämoglobinopathien
MA51787A (fr) 2018-02-05 2020-12-16 Vertex Pharma Substances et méthodes de traitement d'hémoglobinopathies
WO2019161310A1 (en) 2018-02-16 2019-08-22 Casebia Therapeutics Limited Liability Partnership Compositions and methods for gene editing by targeting fibrinogen-alpha
AU2019224159A1 (en) 2018-02-26 2020-10-01 Synthorx, Inc. IL-15 conjugates and uses thereof
US20210054353A1 (en) 2018-03-19 2021-02-25 Crispr Therapeutics Ag Novel rna-programmable endonuclease systems and uses thereof
WO2019186514A2 (en) 2018-03-30 2019-10-03 Rheinische Friedrich-Wilhelms-Universitat Bonn Aptamers for targeted activaton of t cell-mediated immunity
JP2021520781A (ja) 2018-04-06 2021-08-26 チルドレンズ メディカル センター コーポレーションChildren’S Medical Center Corporation 体細胞リプログラミングおよびインプリンティングのモジュレートのための組成物および方法
US11566246B2 (en) 2018-04-12 2023-01-31 Mina Therapeutics Limited SIRT1-saRNA compositions and methods of use
WO2019204668A1 (en) 2018-04-18 2019-10-24 Casebia Therapeutics Limited Liability Partnership Compositions and methods for knockdown of apo(a) by gene editing for treatment of cardiovascular disease
SG11202010910QA (en) 2018-05-07 2020-12-30 Alnylam Pharmaceuticals Inc Extrahepatic delivery
TW202016304A (zh) 2018-05-14 2020-05-01 美商阿尼拉製藥公司 血管收縮素原(AGT)iRNA組成物及其使用方法
AU2019321375A1 (en) 2018-08-13 2021-03-11 Alnylam Pharmaceuticals, Inc. Hepatitis B virus (HBV) dsRNA agent compositions and methods of use thereof
US20210348162A1 (en) 2018-08-16 2021-11-11 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the lect2 gene
WO2020047229A1 (en) 2018-08-29 2020-03-05 University Of Massachusetts Inhibition of protein kinases to treat friedreich ataxia
WO2020060986A1 (en) 2018-09-18 2020-03-26 Alnylam Pharmaceuticals, Inc. Ketohexokinase (khk) irna compositions and methods of use thereof
JP7470107B2 (ja) 2018-09-28 2024-04-17 アルナイラム ファーマシューティカルズ, インコーポレイテッド トランスサイレチン(TTR)iRNA組成物及びTTR関連眼疾患を治療又は予防するためのその使用方法
US20210348159A1 (en) 2018-10-17 2021-11-11 Crispr Therapeutics Ag Compositions and methods for delivering transgenes
US10913951B2 (en) 2018-10-31 2021-02-09 University of Pittsburgh—of the Commonwealth System of Higher Education Silencing of HNF4A-P2 isoforms with siRNA to improve hepatocyte function in liver failure
CA3123617A1 (en) 2018-12-20 2020-06-25 Praxis Precision Medicines, Inc. Compositions and methods for the treatment of kcnt1 related disorders
SI3897672T1 (sl) 2018-12-20 2024-02-29 Humabs Biomed Sa Kombinirana terapija HBV
CA3126933A1 (en) 2019-01-16 2020-07-23 Genzyme Corporation Serpinc1 irna compositions and methods of use thereof
WO2020163532A1 (en) 2019-02-06 2020-08-13 Synthorx, Inc. Il-2 conjugates and methods of use thereof
AU2020221340A1 (en) 2019-02-15 2021-09-16 Bayer Healthcare Llc Gene editing for hemophilia A with improved Factor VIII expression
WO2020171889A1 (en) 2019-02-19 2020-08-27 University Of Rochester Blocking lipid accumulation or inflammation in thyroid eye disease
EP3937963A2 (de) 2019-03-12 2022-01-19 CRISPR Therapeutics AG Neuartige rna-programmierbare high-fidelity-endonukleasesysteme und verwendungen davon
EP3953473A1 (de) 2019-04-12 2022-02-16 MiNA Therapeutics Limited Sirt1-sarna-zusammensetzungen und verfahren zur verwendung
BR112021022806A2 (pt) 2019-05-13 2022-01-25 Vir Biotechnology Inc Método para tratar a infecção crônica por hbv, sirna, usos de um sirna, e de um sirna e peg-ifna e de um sirna, peg-ifna e um nrti, método, composição para uso ou uso e kit
CA3138915A1 (en) 2019-05-17 2020-11-26 Alnylam Pharmaceuticals, Inc. Oral delivery of oligonucleotides
US11879145B2 (en) 2019-06-14 2024-01-23 The Scripps Research Institute Reagents and methods for replication, transcription, and translation in semi-synthetic organisms
EP4007811A2 (de) 2019-08-01 2022-06-08 Alnylam Pharmaceuticals, Inc. Carboxypeptidase-b2(cpb2)-irna-zusammensetzungen und verfahren zur verwendung davon
EP4007812A1 (de) 2019-08-01 2022-06-08 Alnylam Pharmaceuticals, Inc. Irna-zusammensetzungen der serpinfamilie f element 2 (serpinf2) und verfahren zu deren verwendung
WO2021030522A1 (en) 2019-08-13 2021-02-18 Alnylam Pharmaceuticals, Inc. SMALL RIBOSOMAL PROTEIN SUBUNIT 25 (RPS25) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
MX2022001776A (es) 2019-08-15 2022-03-17 Synthorx Inc Terapias de combinacion de inmuno oncologia con conjugados de il-2.
JP2022545101A (ja) 2019-08-19 2022-10-25 ミナ セラピューティクス リミテッド オリゴヌクレオチドコンジュゲート組成物および使用方法
WO2021041206A1 (en) 2019-08-23 2021-03-04 Synthorx, Inc. Il-15 conjugates and uses thereof
EP4025694A1 (de) 2019-09-03 2022-07-13 Alnylam Pharmaceuticals, Inc. Zusammensetzungen und verfahren zum hemmen der lect2-genexpression
MX2022002740A (es) 2019-09-10 2022-03-25 Synthorx Inc Conjugados de il-2 y metodos de uso para tratar enfermedades autoinmunes.
EP4038189A1 (de) 2019-10-04 2022-08-10 Alnylam Pharmaceuticals, Inc. Zusammensetzungen und verfahren zum ausschalten einer ugt1a1-genexpression
US20240141358A1 (en) 2019-10-18 2024-05-02 Alnylam Pharmaceuticals, Inc. Solute carrier family member irna compositions and methods of use thereof
MX2022004726A (es) 2019-10-22 2022-05-13 Alnylam Pharmaceuticals Inc Composiciones de acido ribonucleico de interferencia (arni) de componente c3 de complemento y metodos de uso de las mismas.
US20230040920A1 (en) 2019-11-01 2023-02-09 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing dnajb1-prkaca fusion gene expression
EP4051795A1 (de) 2019-11-01 2022-09-07 Alnylam Pharmaceuticals, Inc. Huntingtin (htt)-irna-wirkstoffzusammensetzungen und verfahren zu deren verwendung
BR112022006703A2 (pt) 2019-11-04 2022-07-12 Synthorx Inc Conjugados de interleucina 10 e usos dos mesmos
WO2021092145A1 (en) 2019-11-06 2021-05-14 Alnylam Pharmaceuticals, Inc. Transthyretin (ttr) irna composition and methods of use thereof for treating or preventing ttr-associated ocular diseases
JP2023500681A (ja) 2019-11-06 2023-01-10 アルニラム ファーマスーティカルズ インコーポレイテッド 肝臓外送達
WO2021096763A1 (en) 2019-11-13 2021-05-20 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating an angiotensinogen- (agt-) associated disorder
US20230056569A1 (en) 2019-11-22 2023-02-23 Alnylam Pharmaceuticals, Inc. Ataxin3 (atxn3) rnai agent compositions and methods of use thereof
WO2021119226A1 (en) 2019-12-13 2021-06-17 Alnylam Pharmaceuticals, Inc. Human chromosome 9 open reading frame 72 (c9orf72) irna agent compositions and methods of use thereof
TW202138559A (zh) 2019-12-16 2021-10-16 美商阿尼拉製藥公司 含類PATATIN磷脂酶結構域3(PNPLA3)iRNA組成物及其使用方法
EP4077674A1 (de) 2019-12-18 2022-10-26 Alia Therapeutics S.R.L. Zusammensetzungen und verfahren zur behandlung von retinitis pigmentosa
WO2021154941A1 (en) 2020-01-31 2021-08-05 Alnylam Pharmaceuticals, Inc. Complement component c5 irna compositions for use in the treatment of amyotrophic lateral sclerosis (als)
CN115427571A (zh) 2020-02-10 2022-12-02 阿尔尼拉姆医药品有限公司 沉默vegf-a表达的组合物和方法
EP4107265A1 (de) 2020-02-18 2022-12-28 Alnylam Pharmaceuticals, Inc. Apolipoprotein-c3 (apoc3)-irna-zusammensetzungen und verfahren zur verwendung davon
EP4114947A1 (de) 2020-03-05 2023-01-11 Alnylam Pharmaceuticals, Inc. Komplementkomponenten-c3-irna-zusammensetzungen und verfahren zur verwendung davon zur behandlung oder prävention von mit komplementkomponenten-c3-assoziierten erkrankungen
BR112022017822A2 (pt) 2020-03-06 2022-11-08 Alnylam Pharmaceuticals Inc Composições de irna de cetoexocinase (khk) e métodos de uso das mesmas
CN115461460A (zh) 2020-03-06 2022-12-09 阿尔尼拉姆医药品有限公司 用于抑制转甲状腺素蛋白(ttr)的表达的组合物和方法
EP4121534A1 (de) 2020-03-18 2023-01-25 Alnylam Pharmaceuticals, Inc. Zusammensetzungen und verfahren zur behandlung von patienten mit einer heterozygoten alaninglyoxylat-aminotransferase-genvariante (agxt)
EP4127168A1 (de) 2020-03-26 2023-02-08 Alnylam Pharmaceuticals, Inc. Coronavirus-irna-zusammensetzungen und verfahren zur verwendung davon
EP4127171A2 (de) 2020-03-30 2023-02-08 Alnylam Pharmaceuticals, Inc. Zusammensetzungen und verfahren zur ausschaltung der dnajc15-genexpression
BR112022020145A2 (pt) 2020-04-06 2023-01-03 Alnylam Pharmaceuticals Inc Composições e métodos para silenciamento da expressão de myoc
EP4133077A1 (de) 2020-04-07 2023-02-15 Alnylam Pharmaceuticals, Inc. Irna-zusammensetzungen mit transmembran-serinprotease 2 (tmprss2) und verfahren zur verwendung davon
EP4133076A1 (de) 2020-04-07 2023-02-15 Alnylam Pharmaceuticals, Inc. Irna-zusammensetzungen aus angiotensin-konvertierendem enzym 2 (ace2) und verfahren zur verwendung davon
JP2023521094A (ja) 2020-04-07 2023-05-23 アルナイラム ファーマシューティカルズ, インコーポレイテッド Scn9a発現をサイレンシングするための組成物および方法
CA3181400A1 (en) 2020-04-27 2021-11-04 Alnylam Pharmaceuticals, Inc. Apolipoprotein e (apoe) irna agent compositions and methods of use thereof
KR20230017789A (ko) 2020-04-30 2023-02-06 알닐람 파마슈티칼스 인코포레이티드 보체 인자 B (CFB) iRNA 조성물 및 이의 사용 방법
EP4150086A1 (de) 2020-05-15 2023-03-22 Korro Bio, Inc. Verfahren und zusammensetzungen zur adar-vermittelten bearbeitung von leucinreicher repeat-kinase 2 (lrrk2)
WO2021231692A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of otoferlin (otof)
EP4150088A1 (de) 2020-05-15 2023-03-22 Korro Bio, Inc. Verfahren und zusammensetzungen zur adar-vermittelten bearbeitung von argininosuccinat-synthetase (ass1)
EP4150087A1 (de) 2020-05-15 2023-03-22 Korro Bio, Inc. Verfahren und zusammensetzungen zur adar-vermittelten bearbeitung des gap-junction-proteins beta 2 (gjb2)
EP4150089A1 (de) 2020-05-15 2023-03-22 Korro Bio, Inc. Verfahren und zusammensetzungen zur adar-vermittelten bearbeitung von retinosin 1 (rs1)
EP4150077A1 (de) 2020-05-15 2023-03-22 Korro Bio, Inc. Verfahren und zusammensetzungen zur adar-vermittelten bearbeitung von transmembrankanalähnlichem protein 1 (tmc1)
WO2021231680A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of methyl-cpg binding protein 2 (mecp2)
EP4153746A1 (de) 2020-05-21 2023-03-29 Alnylam Pharmaceuticals, Inc. Zusammensetzungen und verfahren zur hemmung der marc1-genexpression
MX2022014606A (es) 2020-05-22 2023-03-08 Wave Life Sciences Ltd Composiciones de oligonucleótidos bicatenarios y métodos relacionados con las mismas.
US11408000B2 (en) 2020-06-03 2022-08-09 Triplet Therapeutics, Inc. Oligonucleotides for the treatment of nucleotide repeat expansion disorders associated with MSH3 activity
EP4162050A1 (de) 2020-06-09 2023-04-12 Alnylam Pharmaceuticals, Inc. Rnai-zusammensetzungen und verfahren zur verwendung davon zur verabreichung durch inhalation
AU2021292296A1 (en) 2020-06-18 2023-01-19 Alnylam Pharmaceuticals, Inc. Xanthine dehydrogenase (XDH) iRNA compositions and methods of use thereof
BR112022026316A2 (pt) 2020-06-24 2023-03-07 Vir Biotechnology Inc Anticorpos neutralizantes do vírus da hepatite b engenheirados e usos dos mesmos
MX2022016254A (es) 2020-06-25 2023-04-11 Synthorx Inc Terapia de combinación de inmunooncología con conjugados de il-2 y anticuerpos anti-egfr.
CA3177613A1 (en) 2020-07-10 2022-04-13 Inserm (Institut National De La Sante Et De La Recherche Medicale) Methods and compositions for treating epilepsy
US20230257745A1 (en) 2020-07-10 2023-08-17 Alnylam Pharmaceuticals, Inc. Circular siRNAs
EP4217489A1 (de) 2020-09-24 2023-08-02 Alnylam Pharmaceuticals, Inc. Dipeptidylpeptidase 4 (dpp4)-irna-zusammensetzungen und verfahren zur verwendung davon
EP4222264A1 (de) 2020-09-30 2023-08-09 CRISPR Therapeutics AG Materialien und verfahren zur behandlung von amyotropher lateralsklerose
WO2022076291A1 (en) 2020-10-05 2022-04-14 Alnylam Pharmaceuticals, Inc. G protein-coupled receptor 75 (gpr75) irna compositions and methods of use thereof
IL301612A (en) 2020-10-09 2023-05-01 Synthorx Inc Immuno-oncological treatments with IL-2 couples
KR20230084203A (ko) 2020-10-09 2023-06-12 신톡스, 인크. Il-2 접합체 및 펨브롤리주맙을 사용한 면역 종양 병용 요법
EP4232582A1 (de) 2020-10-23 2023-08-30 Alnylam Pharmaceuticals, Inc. Mucin-5b (muc5b)-irna-zusammensetzungen und verfahren zur verwendung davon
IL302709A (en) 2020-11-13 2023-07-01 Alnylam Pharmaceuticals Inc Coagulation factor iRNA compositions (F5) and methods of using them
WO2022125490A1 (en) 2020-12-08 2022-06-16 Alnylam Pharmaceuticals, Inc. Coagulation factor x (f10) irna compositions and methods of use thereof
JP2024501857A (ja) 2020-12-31 2024-01-16 アルナイラム ファーマシューティカルズ, インコーポレイテッド 環状ジスルフィド修飾リン酸ベースのオリゴヌクレオチドプロドラッグ
WO2022147223A2 (en) 2020-12-31 2022-07-07 Alnylam Pharmaceuticals, Inc. 2'-modified nucleoside based oligonucleotide prodrugs
EP4274896A1 (de) 2021-01-05 2023-11-15 Alnylam Pharmaceuticals, Inc. Komplementkomponente 9 (c9)-irna-zusammensetzungen und verfahren zur verwendung davon
EP4291243A1 (de) 2021-02-12 2023-12-20 Synthorx, Inc. Lungenkrebskombinationstherapie mit il-2-konjugaten und einem anti-pd-1-antikörper oder antigenbindendem fragment davon
WO2022174101A1 (en) 2021-02-12 2022-08-18 Synthorx, Inc. Skin cancer combination therapy with il-2 conjugates and cemiplimab
EP4291654A2 (de) 2021-02-12 2023-12-20 Alnylam Pharmaceuticals, Inc. Superoxid-dismutase-1 (sod1)-irna-zusammensetzungen und verfahren zur verwendung davon zur behandlung oder vorbeugung von mit superoxid-dismutase 1 (sod1-) assoziierten neurodegenerativen erkrankungen
WO2022182864A1 (en) 2021-02-25 2022-09-01 Alnylam Pharmaceuticals, Inc. Prion protein (prnp) irna compositions and methods and methods of use thereof
TW202302847A (zh) 2021-02-26 2023-01-16 美商艾拉倫製藥股份有限公司 己酮糖激酶(KHK)iRNA組成物及其使用方法
BR112023017737A2 (pt) 2021-03-04 2023-10-03 Alnylam Pharmaceuticals Inc Composições de irna de angiopoietina-semelhantes 3 (angptl3) e métodos de uso das mesmas
WO2022192519A1 (en) 2021-03-12 2022-09-15 Alnylam Pharmaceuticals, Inc. Glycogen synthase kinase 3 alpha (gsk3a) irna compositions and methods of use thereof
JP2024511092A (ja) 2021-03-26 2024-03-12 ミナ セラピューティクス リミテッド TMEM173saRNA組成物及び使用方法
CN117203336A (zh) 2021-03-29 2023-12-08 阿尔尼拉姆医药品有限公司 亨廷顿蛋白(HTT)iRNA药剂组合物以及其使用方法
EP4314293A1 (de) 2021-04-01 2024-02-07 Alnylam Pharmaceuticals, Inc. Prolindehydrogenase 2 (prodh2)-irna-zusammensetzungen und verfahren zur verwendung davon
CA3214540A1 (en) 2021-04-22 2022-10-27 Pasi A. Janne Compositions and methods for treating cancer
JP2024517686A (ja) 2021-04-26 2024-04-23 アルナイラム ファーマシューティカルズ, インコーポレイテッド 膜貫通プロテアーゼ、セリン6(TMPRSS6)iRNA組成物およびその使用方法
WO2022232343A1 (en) 2021-04-29 2022-11-03 Alnylam Pharmaceuticals, Inc. Signal transducer and activator of transcription factor 6 (stat6) irna compositions and methods of use thereof
WO2022245583A1 (en) 2021-05-18 2022-11-24 Alnylam Pharmaceuticals, Inc. Sodium-glucose cotransporter-2 (sglt2) irna compositions and methods of use thereof
EP4341405A1 (de) 2021-05-20 2024-03-27 Korro Bio, Inc. Verfahren und zusammensetzungen für adar-vermittelte bearbeitung
WO2022256283A2 (en) 2021-06-01 2022-12-08 Korro Bio, Inc. Methods for restoring protein function using adar
TW202317762A (zh) 2021-06-02 2023-05-01 美商艾拉倫製藥股份有限公司 含有類PATATIN磷脂酶結構域3(PNPLA3)的iRNA組成物及其使用方法
TW202313117A (zh) 2021-06-03 2023-04-01 美商欣爍克斯公司 包含il-2接合物及西妥昔單抗(cetuximab)之頭頸癌組合療法
IL308743A (en) 2021-06-04 2024-01-01 Alnylam Pharmaceuticals Inc Compositions of human chromosome 9 open reading frame 72 iRNA factor (C9ORF72) and methods of using them
WO2022260939A2 (en) 2021-06-08 2022-12-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for treating or preventing stargardt's disease and/or retinal binding protein 4 (rbp4)-associated disorders
EP4352214A1 (de) 2021-06-11 2024-04-17 Bayer AG Typ-v-rna-programmierbare endonukleasesysteme
EP4101928A1 (de) 2021-06-11 2022-12-14 Bayer AG Programmierbare typ-v-rna-endonukleasesysteme
EP4363574A1 (de) 2021-06-29 2024-05-08 Korro Bio, Inc. Verfahren und zusammensetzungen für adar-vermittelte bearbeitung
US20230194709A9 (en) 2021-06-29 2023-06-22 Seagate Technology Llc Range information detection using coherent pulse sets with selected waveform characteristics
AU2022303164A1 (en) 2021-06-30 2024-01-18 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating an angiotensinogen- (agt-) associated disorder
WO2023283403A2 (en) 2021-07-09 2023-01-12 Alnylam Pharmaceuticals, Inc. Bis-rnai compounds for cns delivery
WO2023285431A1 (en) 2021-07-12 2023-01-19 Alia Therapeutics Srl Compositions and methods for allele specific treatment of retinitis pigmentosa
WO2023003805A1 (en) 2021-07-19 2023-01-26 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating subjects having or at risk of developing a non-primary hyperoxaluria disease or disorder
IL309897A (en) 2021-07-21 2024-03-01 Alnylam Pharmaceuticals Inc Target gene IRNA compositions associated with a metabolic disorder and methods of using them
AU2022316139A1 (en) 2021-07-23 2024-01-18 Alnylam Pharmaceuticals, Inc. Beta-catenin (ctnnb1) irna compositions and methods of use thereof
WO2023009687A1 (en) 2021-07-29 2023-02-02 Alnylam Pharmaceuticals, Inc. 3-hydroxy-3-methylglutaryl-coa reductase (hmgcr) irna compositions and methods of use thereof
CN117795074A (zh) 2021-08-03 2024-03-29 阿尔尼拉姆医药品有限公司 转甲状腺素蛋白(TTR)iRNA组合物和其使用方法
WO2023014765A1 (en) 2021-08-04 2023-02-09 Alnylam Pharmaceuticals, Inc. iRNA COMPOSITIONS AND METHODS FOR SILENCING ANGIOTENSINOGEN (AGT)
WO2023019246A1 (en) 2021-08-13 2023-02-16 Alnylam Pharmaceuticals, Inc. Factor xii (f12) irna compositions and methods of use thereof
EP4144841A1 (de) 2021-09-07 2023-03-08 Bayer AG Neue programmierbare rna-endonuklease-systeme mit verbesserter pam-spezifität und deren verwendung
WO2023044370A2 (en) 2021-09-17 2023-03-23 Alnylam Pharmaceuticals, Inc. Irna compositions and methods for silencing complement component 3 (c3)
WO2023044094A1 (en) 2021-09-20 2023-03-23 Alnylam Pharmaceuticals, Inc. Inhibin subunit beta e (inhbe) modulator compositions and methods of use thereof
CA3234887A1 (en) 2021-10-15 2023-04-20 Alnylam Pharmaceuticals, Inc. Extra-hepatic delivery irna compositions and methods of use thereof
WO2023069603A1 (en) 2021-10-22 2023-04-27 Korro Bio, Inc. Methods and compositions for disrupting nrf2-keap1 protein interaction by adar mediated rna editing
WO2023076451A1 (en) 2021-10-29 2023-05-04 Alnylam Pharmaceuticals, Inc. Complement factor b (cfb) irna compositions and methods of use thereof
WO2023076450A2 (en) 2021-10-29 2023-05-04 Alnylam Pharmaceuticals, Inc. HUNTINGTIN (HTT) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
WO2023099884A1 (en) 2021-12-01 2023-06-08 Mina Therapeutics Limited Pax6 sarna compositions and methods of use
WO2023122573A1 (en) 2021-12-20 2023-06-29 Synthorx, Inc. Head and neck cancer combination therapy comprising an il-2 conjugate and pembrolizumab
WO2023118349A1 (en) 2021-12-21 2023-06-29 Alia Therapeutics Srl Type ii cas proteins and applications thereof
WO2023118068A1 (en) 2021-12-23 2023-06-29 Bayer Aktiengesellschaft Novel small type v rna programmable endonuclease systems
WO2023122750A1 (en) 2021-12-23 2023-06-29 Synthorx, Inc. Cancer combination therapy with il-2 conjugates and cetuximab
WO2023141314A2 (en) 2022-01-24 2023-07-27 Alnylam Pharmaceuticals, Inc. Heparin sulfate biosynthesis pathway enzyme irna agent compositions and methods of use thereof
WO2023170435A1 (en) 2022-03-07 2023-09-14 Mina Therapeutics Limited Il10 sarna compositions and methods of use
WO2023194359A1 (en) 2022-04-04 2023-10-12 Alia Therapeutics Srl Compositions and methods for treatment of usher syndrome type 2a
WO2023220744A2 (en) 2022-05-13 2023-11-16 Alnylam Pharmaceuticals, Inc. Single-stranded loop oligonucleotides
CN114790161B (zh) * 2022-05-16 2023-12-01 百顺药业有限公司 4-甲氧基羰基乙基-3-甲基-2-吡咯醛的合成方法及其中间体
WO2023237587A1 (en) 2022-06-10 2023-12-14 Bayer Aktiengesellschaft Novel small type v rna programmable endonuclease systems
WO2024006999A2 (en) 2022-06-30 2024-01-04 Alnylam Pharmaceuticals, Inc. Cyclic-disulfide modified phosphate based oligonucleotide prodrugs
WO2024039776A2 (en) 2022-08-18 2024-02-22 Alnylam Pharmaceuticals, Inc. Universal non-targeting sirna compositions and methods of use thereof
WO2024059165A1 (en) 2022-09-15 2024-03-21 Alnylam Pharmaceuticals, Inc. 17b-hydroxysteroid dehydrogenase type 13 (hsd17b13) irna compositions and methods of use thereof
WO2024056880A2 (en) 2022-09-16 2024-03-21 Alia Therapeutics Srl Enqp type ii cas proteins and applications thereof
WO2024073732A1 (en) 2022-09-30 2024-04-04 Alnylam Pharmaceuticals, Inc. Modified double-stranded rna agents

Family Cites Families (26)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4318825A (en) * 1979-08-15 1982-03-09 Frame Robert R Catalytic composite, and method of manufacture
US4647447A (en) * 1981-07-24 1987-03-03 Schering Aktiengesellschaft Diagnostic media
US4957939A (en) * 1981-07-24 1990-09-18 Schering Aktiengesellschaft Sterile pharmaceutical compositions of gadolinium chelates useful enhancing NMR imaging
US4613322A (en) * 1982-12-08 1986-09-23 Edelson Richard Leslie Method and system for externally treating the blood
FR2540122B1 (fr) * 1983-01-27 1985-11-29 Centre Nat Rech Scient Nouveaux composes comportant une sequence d'oligonucleotide liee a un agent d'intercalation, leur procede de synthese et leur application
JPS61275228A (ja) * 1985-03-14 1986-12-05 バクスタ−、トラベノ−ル、ラボラトリ−ズ、インコ−ポレイテツド 治療用タンパク組成物中のビ−ルスの光動的不活性化
US4977177A (en) * 1985-04-30 1990-12-11 Nippon Petrochemicals Company, Ltd. Tetrapyrrole polyaminomonocarboxylic acid therapeutic agents
US4899755A (en) * 1985-05-08 1990-02-13 The General Hospital Corporation Hepatobiliary NMR contrast agents
US4880008A (en) * 1985-05-08 1989-11-14 The General Hospital Corporation Vivo enhancement of NMR relaxivity
CA1315780C (en) * 1986-01-17 1993-04-06 Yozo Fukuda Porphyrin derivatives
US5242797A (en) * 1986-03-21 1993-09-07 Myron J. Block Nucleic acid assay method
US4915683A (en) * 1986-11-21 1990-04-10 The Medical College Of Wisconsin, Inc. Antiviral method, agents and apparatus
US4883790A (en) * 1987-01-20 1989-11-28 University Of British Columbia Wavelength-specific cytotoxic agents
US4878891A (en) * 1987-06-25 1989-11-07 Baylor Research Foundation Method for eradicating infectious biological contaminants in body tissues
EP0396651A4 (en) * 1988-07-28 1990-12-12 Best Industries, Inc. Device and method for encapsulating radioactive materials
US5272142A (en) * 1989-03-06 1993-12-21 Board Of Regents, The University Of Texas System Expanded porphyrins: large porphyrin-like tripyrroledimethine-derived macrocycles and methods for treating tumors
US4935498A (en) * 1989-03-06 1990-06-19 Board Of Regents, The University Of Texas System Expanded porphyrins: large porphyrin-like tripyrroledimethine-derived macrocycles
US5256399A (en) * 1989-03-06 1993-10-26 Board Of Regents, The University Of Texas System Aromatic pentadentate expanded porphyrins in magnetic resonance imaging
US5252720A (en) * 1989-03-06 1993-10-12 Board Of Regents, The University Of Texas System Metal complexes of water soluble texaphyrins
US5041078A (en) * 1989-03-06 1991-08-20 Baylor Research Foundation, A Nonprofit Corporation Of The State Of Texas Photodynamic viral deactivation with sapphyrins
US5162509A (en) * 1989-03-06 1992-11-10 Board Of Regents, The University Of Texas System Process for preparing expanded porphyrins: large porphyrin-like tripyrroledimethine-derived macrocycles
US4959363A (en) * 1989-06-23 1990-09-25 Sterling Drug Inc. Quinolonecarboxamide compounds, their preparation and use as antivirals.
FR2650761B1 (fr) * 1989-08-10 1994-03-04 Elf Aquitaine Ste Nale Catalyseur d'oxydation a base de metalloporphyrine sur support
US5159065A (en) * 1989-12-21 1992-10-27 Board Of Regents, The University Of Texas System Sapphyrins, derivatives and syntheses
US5272056A (en) * 1991-01-03 1993-12-21 The Research Foundation Of State University Of New York Modification of DNA and oligonucleotides using metal complexes of polyaza ligands
US5371199B1 (en) * 1992-08-14 1995-12-26 Univ Pennsylvania Substituted porphyrins porphyrin-containing polymers and synthetic methods therefor

Also Published As

Publication number Publication date
AU1921795A (en) 1995-08-29
NO308473B1 (no) 2000-09-18
NO963396D0 (no) 1996-08-14
ES2173951T3 (es) 2002-11-01
DE69526782D1 (de) 2002-06-27
KR970701193A (ko) 1997-03-17
NZ282185A (en) 1998-03-25
NO963396L (no) 1996-10-14
US5599923A (en) 1997-02-04
WO1995021845A1 (en) 1995-08-17
DE69526782T2 (de) 2003-03-06
AU688008B2 (en) 1998-03-05
EP0745085A1 (de) 1996-12-04
CA2182960A1 (en) 1995-08-17
EP0745085B1 (de) 2002-05-22
JPH10500659A (ja) 1998-01-20
US5599928A (en) 1997-02-04
DK0745085T3 (da) 2002-06-17
PT745085E (pt) 2002-10-31

Similar Documents

Publication Publication Date Title
ATE217874T1 (de) Texaphyrin metallkomplexe mit verbesserten funktionellen gruppen
CA2245142A1 (en) New phenanthridines
BG104812A (en) New dihydropyrimidines
AU2291097A (en) Novel phenanthridines substituted in the 6 position
NO20010228D0 (no) Farmasöytiske preparater omfattende 2-kinoloner
AU2795000A (en) Epothilone derivatives and their use as antitumor agents
NZ335383A (en) Epothilone C,D,E and F for plant protection and cytostatic effects shown in kidney cells
PL346875A1 (en) Oral care compositions
DE69807074T2 (de) Substituierte 1,2,4-triazolo[3,4,-a]phthalazin-derivate als gaba-alpha 5-liganden
BG105783A (en) Triazole compounds with dopamine-d3-receptor affinity
MXPA03008755A (es) Derivados de 2-amino-propanol.
PL338339A1 (en) Novel derivatives of tetrazole
YU19499A (sh) Benzonaftiridini kao bronhijalni terapeutici
BG105444A (en) Power coating compositions
AU6203399A (en) Polyamide composition stabilized with copper complexes and organic halogen compounds
MX9805499A (es) Compuestos de azacicloheptano y diazacicloheptano y ciclooctano sustituidos y su uso.
WO1994005732A3 (en) Carbon blacks
ATE277624T1 (de) Kovalent gebundenes n,o-carboxymethylchitosan und seine verwendungen
SI1042319T1 (en) Dihydrobenzofurans
WO1996041627A3 (en) Iron compounds, compositions, methods of making the same and uses thereof
AU3307100A (en) Crosslinkable coating compositions
EE200100287A (et) Puidukaitsevahendite lahused
AU6832498A (en) Substituted benzyl oximino compounds
EP1096021A3 (de) Melanine mit verbesserten HIV replikationshemmenden Eigenschaften
DE60003560D1 (de) 1,2-dihydro-1-oxo-pyrazino[1,2-]indolederivate

Legal Events

Date Code Title Description
REN Ceased due to non-payment of the annual fee