JP2021515037A - 寛容原性リポソーム及びその使用方法 - Google Patents

寛容原性リポソーム及びその使用方法 Download PDF

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JP2021515037A
JP2021515037A JP2020567462A JP2020567462A JP2021515037A JP 2021515037 A JP2021515037 A JP 2021515037A JP 2020567462 A JP2020567462 A JP 2020567462A JP 2020567462 A JP2020567462 A JP 2020567462A JP 2021515037 A JP2021515037 A JP 2021515037A
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lipid
liposomes
ite
composition
composition according
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Japanese (ja)
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JPWO2019165436A5 (https=
JP2021515037A5 (https=
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ジャヴェリ，アディティ
スタントン，ヴィンセント，ピー．，ジュニア
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アントルクス，インコーポレーテッド
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/41—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with two or more ring hetero atoms, at least one of which being nitrogen, e.g. tetrazole
    • A61K31/425—Thiazoles
    • A61K31/427—Thiazoles not condensed and containing further heterocyclic rings
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/0005—Vertebrate antigens
    • A61K39/0008—Antigens related to auto-immune diseases; Preparations to induce self-tolerance
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/0005—Vertebrate antigens
    • A61K39/0011—Cancer antigens
    • A61K39/001193—Prostate associated antigens e.g. Prostate stem cell antigen [PSCA]; Prostate carcinoma tumor antigen [PCTA]; PAP or PSGR
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0031—Rectum, anus
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0034—Urogenital system, e.g. vagina, uterus, cervix, penis, scrotum, urethra, bladder; Personal lubricants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0053—Mouth and digestive tract, i.e. intraoral and peroral administration
    • A61K9/006—Oral mucosa, e.g. mucoadhesive forms, sublingual droplets; Buccal patches or films; Buccal sprays
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/007—Pulmonary tract; Aromatherapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/107—Emulsions ; Emulsion preconcentrates; Micelles
    • A61K9/1075—Microemulsions or submicron emulsions; Preconcentrates or solids thereof; Micelles, e.g. made of phospholipids or block copolymers
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • A61K9/1271—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers
    • A61K9/1272—Non-conventional liposomes, e.g. PEGylated liposomes or liposomes coated or grafted with polymers comprising non-phosphatidyl surfactants as bilayer-forming substances, e.g. cationic lipids or non-phosphatidyl liposomes coated or grafted with polymers
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/51—Medicinal preparations containing antigens or antibodies comprising whole cells, viruses or DNA/RNA
    • A61K2039/525—Virus
    • A61K2039/5256—Virus expressing foreign proteins
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/51—Medicinal preparations containing antigens or antibodies comprising whole cells, viruses or DNA/RNA
    • A61K2039/53—DNA (RNA) vaccination
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/555—Medicinal preparations containing antigens or antibodies characterised by a specific combination antigen/adjuvant
    • A61K2039/55511—Organic adjuvants
    • A61K2039/55555—Liposomes; Vesicles, e.g. nanoparticles; Spheres, e.g. nanospheres; Polymers
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/57—Medicinal preparations containing antigens or antibodies characterised by the type of response, e.g. Th1, Th2
    • A61K2039/577—Medicinal preparations containing antigens or antibodies characterised by the type of response, e.g. Th1, Th2 tolerising response

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • General Health & Medical Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Dispersion Chemistry (AREA)
  • Molecular Biology (AREA)
  • Immunology (AREA)
  • Mycology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Cell Biology (AREA)
  • Oncology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Rheumatology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Organic Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physiology (AREA)
  • Pulmonology (AREA)
  • Nutrition Science (AREA)
  • Dermatology (AREA)
  • Gynecology & Obstetrics (AREA)
  • Reproductive Health (AREA)
  • Urology & Nephrology (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Peptides Or Proteins (AREA)
JP2020567462A 2018-02-26 2019-02-26 寛容原性リポソーム及びその使用方法 Ceased JP2021515037A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201862635376P 2018-02-26 2018-02-26
US62/635,376 2018-02-26
US201862755946P 2018-11-05 2018-11-05
US62/755,946 2018-11-05
PCT/US2019/019609 WO2019165436A1 (en) 2018-02-26 2019-02-26 Tolerogenic liposomes and methods of use thereof

Publications (3)

Publication Number Publication Date
JP2021515037A true JP2021515037A (ja) 2021-06-17
JPWO2019165436A5 JPWO2019165436A5 (https=) 2022-03-07
JP2021515037A5 JP2021515037A5 (https=) 2022-03-07

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JP2020567462A Ceased JP2021515037A (ja) 2018-02-26 2019-02-26 寛容原性リポソーム及びその使用方法

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US (1) US12178908B2 (https=)
EP (1) EP3758682A4 (https=)
JP (1) JP2021515037A (https=)
KR (1) KR20200139679A (https=)
CN (1) CN112384204B (https=)
AU (1) AU2019225251A1 (https=)
CA (1) CA3092372A1 (https=)
IL (1) IL276957B2 (https=)
WO (1) WO2019165436A1 (https=)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2021515037A (ja) 2018-02-26 2021-06-17 アントルクス，インコーポレーテッド 寛容原性リポソーム及びその使用方法
US10736847B2 (en) 2018-07-03 2020-08-11 Becton, Dickinson And Company Inverting device for liposome preparation by centrifugation
WO2021011806A1 (en) * 2019-07-16 2021-01-21 AnTolRx, Inc. Immune tolerization agents and potentiators
CN113740525A (zh) * 2020-05-29 2021-12-03 中国药科大学 一种检测肾损伤标志物胱抑素c的方法
EP4219501B1 (en) * 2021-05-27 2026-01-28 Shandong University Compound containing 2,4-thiazole ring, preparation method therefor, and application thereof
US20260041747A1 (en) 2022-07-29 2026-02-12 Repertoire Immune Medicines, Inc. T cell epitopes associated with type 1 diabetes
KR20250115413A (ko) * 2022-11-23 2025-07-30 어헤드 테라퓨틱스, 에스.엘. 관용원성 조성물
WO2025072849A1 (en) 2023-09-29 2025-04-03 Repertoire Immune Medicines, Inc. Mhc-presentable peptides associated with type 1 diabetes

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20170121683A1 (en) * 2015-11-03 2017-05-04 Regents Of The University Of Minnesota Aryl hydrocarbon receptor disruption and enhancement

Family Cites Families (80)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB8918616D0 (en) 1989-08-15 1989-09-27 Univ Glasgow Herpes simplex virus type 1 mutant
US5849572A (en) 1990-10-10 1998-12-15 Regents Of The University Of Michigan HSV-1 vector containing a lat promoter
US5804413A (en) 1992-07-31 1998-09-08 University Of Pittsburgh Of The Commonwealth System Of Higher Education Herpes simplex virus strains for gene transfer
EP1637608B1 (en) 1993-10-25 2009-07-22 CANJI, Inc. Recombinant adenoviral vector and methods of use
GB9415319D0 (en) 1994-07-29 1994-09-21 Medical Res Council HSV viral vector
US5846782A (en) 1995-11-28 1998-12-08 Genvec, Inc. Targeting adenovirus with use of constrained peptide motifs
US5801030A (en) 1995-09-01 1998-09-01 Genvec, Inc. Methods and vectors for site-specific recombination
US5837511A (en) 1995-10-02 1998-11-17 Cornell Research Foundation, Inc. Non-group C adenoviral vectors
US5849561A (en) 1997-05-22 1998-12-15 Cornell Research Foundation, Inc. Method for the production of non-group C adenoviral vectors
AU8605598A (en) 1997-07-31 1999-02-22 University Of Pittsburgh Targeted hsv vectors
US6436392B1 (en) 1998-05-20 2002-08-20 University Of Iowa Research Foundation Adeno-associated virus vectors
US6759237B1 (en) 1998-11-05 2004-07-06 The Trustees Of The University Of Pennsylvania Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
US6562943B1 (en) * 1999-04-21 2003-05-13 Zycos, Inc. Peptide epitopes recognized by disease promoting CD4+ T lymphocytes
NZ522840A (en) 2000-06-01 2004-12-24 Univ North Carolina A parvovirus vector that carries a duplexed genome resulting in co-packaging of strands of plus and minus polarity tethered together
US7122181B2 (en) 2000-12-19 2006-10-17 Research Development Foundation Lentiviral vector-mediated gene transfer and uses thereof
AU2002303330B2 (en) 2001-05-31 2008-07-24 Novartis Vaccines And Diagnostics, Inc. Chimeric alphavirus replicon particles
US20070129307A1 (en) 2001-06-22 2007-06-07 The University Of British Columbia Insulin epitopes for the treatment of type 1 diabetes
EP1412493B1 (en) 2001-08-02 2011-10-05 Institut Clayton De La Recherche Methods and compositions relating to improved lentiviral vector production systems
WO2003023026A1 (en) 2001-09-06 2003-03-20 Alphavax, Inc. Alphavirus replicon vector systems
EP1438075A4 (en) 2001-10-02 2006-04-19 Inst Clayton De La Rech METHOD AND COMPOSITIONS ASSOCIATED WITH LENTIVIRAL VECTORS WITH LIMITED EXPRESSION AND ITS APPLICATIONS
DK2359869T3 (en) 2001-12-17 2019-04-15 Univ Pennsylvania Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof
DE60333487D1 (de) 2002-12-13 2010-09-02 Genetix Pharmaceuticals Inc Therapeutische retrovirus-vektoren für gentherapie
WO2004075861A2 (en) 2003-02-26 2004-09-10 Children's Hospital, Inc. Recombinant adeno-associated virus production
US7186699B2 (en) 2003-06-03 2007-03-06 Cell Genesys, Inc. Method for treating cancer by vector-mediated delivery of one or more anti-angiogenic or pro-apoptotic genes
EP1486567A1 (en) 2003-06-11 2004-12-15 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Improved adeno-associated virus (AAV) vector for gene therapy
US20050100890A1 (en) 2003-10-15 2005-05-12 Davidson Beverly L. Methods for producing and using in vivo pseudotyped retroviruses
AU2006214278C1 (en) 2005-02-16 2012-07-19 Miltenyi Biotec Technology, Inc. Lentiviral vectors and their use
CA2609276A1 (en) 2005-05-23 2006-11-30 De-Chu C. Tang Rapid production of adenovirus-free recombinant adenovirus vectors
FI20050814A0 (fi) 2005-08-11 2005-08-11 Procollagen Oy Menetelmä nivelreumassa muodostuvien kollageeniautovasta-aineiden havaitsemiseksi
DE602006019916D1 (de) 2005-12-12 2011-03-10 Canji Inc Adenovirale expressionsvektoren mit einer expressionskassette im e1-bereich und einer inaktivierten e2b-polymerase
GB0526211D0 (en) 2005-12-22 2006-02-01 Oxford Biomedica Ltd Viral vectors
US20080026485A1 (en) 2006-04-18 2008-01-31 Wolfgang Hueber Antibody profiling for determination of patient responsiveness
ES2465996T3 (es) 2006-05-25 2014-06-09 Sangamo Biosciences, Inc. Métodos y composiciones para la inactivación genética
KR101520507B1 (ko) 2006-12-14 2015-05-29 다우 아그로사이언시즈 엘엘씨 최적화된 비-정규 아연 손가락 단백질
US20100047256A1 (en) 2007-01-25 2010-02-25 Imperial Innovations Limited Methods
ES2708856T3 (es) 2007-08-03 2019-04-11 Pasteur Institut Vectores de transferencia de gen lentivírico y sus aplicaciones medicinales
EP2215223B1 (en) 2007-10-31 2013-05-01 Precision Biosciences, Inc. Rationally-designed single-chain meganucleases with non-palindromic recognition sequences
JP2011503232A (ja) 2007-11-20 2011-01-27 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 免疫応答の調節
EP2215252A4 (en) 2007-12-07 2011-01-26 Prec Biosciences Inc RATIONAL-DESIGNED MEGANUCLEASES WITH DETECTION SEQUENCES FROM DNASE-HYPERSENSITIVE REGIONS OF THE HUMAN GENOME
WO2009114321A2 (en) 2008-03-11 2009-09-17 Precision Biosciencs, Inc. Rationally-designed meganucleases for maize genome engineering
EP2276833A4 (en) 2008-03-21 2011-06-29 Brigham & Womens Hospital MODULATION OF THE IMMUNE RESPONSE
WO2009146179A1 (en) 2008-04-15 2009-12-03 University Of Iowa Research Foundation Zinc finger nuclease for the cftr gene and methods of use thereof
EP2342321B1 (en) 2008-09-17 2018-04-11 Isogenis, Inc. Construction of fully-deleted adenovirus-based gene delivery vectors and uses thereof
US20100216804A1 (en) * 2008-12-15 2010-08-26 Zale Stephen E Long Circulating Nanoparticles for Sustained Release of Therapeutic Agents
US8679837B2 (en) 2009-04-02 2014-03-25 University Of Florida Research Foundation, Inc. Inducible system for highly efficient production of recombinant Adeno-associated virus (rAAV) vectors
WO2011036640A2 (en) 2009-09-24 2011-03-31 Cellectis Meganuclease reagents of uses thereof for treating genetic diseases caused by frame shift/non sense mutations
US20120283318A1 (en) 2009-10-05 2012-11-08 Mei Ya-Fang Replicating viral vectors for gene therapy
US8956828B2 (en) 2009-11-10 2015-02-17 Sangamo Biosciences, Inc. Targeted disruption of T cell receptor genes using engineered zinc finger protein nucleases
HUE041436T2 (hu) 2009-12-10 2019-05-28 Univ Minnesota Tal-effektor-közvetített DNS-módosítás
EP2534163B1 (en) 2010-02-09 2015-11-04 Sangamo BioSciences, Inc. Targeted genomic modification with partially single-stranded donor molecules
US8927514B2 (en) 2010-04-30 2015-01-06 City Of Hope Recombinant adeno-associated vectors for targeted treatment
ES2685611T3 (es) 2011-02-14 2018-10-10 The Children's Hospital Of Philadelphia Vector VAA8 mejorado con una actividad funcional aumentada y métodos de utilización del mismo
WO2012145509A2 (en) 2011-04-19 2012-10-26 The Research Foundation Of State University Of New York Adeno-associated-virus rep sequences, vectors, and viruses
KR20140041505A (ko) * 2011-04-29 2014-04-04 셀렉타 바이오사이언시즈, 인크. 조절 b 세포 유도를 위한 관용원성 합성 나노운반체
KR101873447B1 (ko) 2011-06-22 2018-07-03 삼성디스플레이 주식회사 신규한 화합물 및 이를 포함한 유기발광 소자
RU2650811C2 (ru) 2012-02-24 2018-04-17 Фред Хатчинсон Кэнсер Рисерч Сентер Композиции и способы лечения гемоглобинопатии
EP2638896A1 (en) 2012-03-14 2013-09-18 Bioneer A/S Cationic liposomal drug delivery system for specific targeting of human cd14+ monocytes in whole blood
EP3663395B1 (en) 2012-03-26 2024-07-31 The United States of America, as Represented by The Secretary, Department of Health and Human Services Office of Technology, Transfer, Delivery of packaged rna to mammalian cells
AU2013249202B2 (en) 2012-04-18 2018-08-09 The Children's Hospital Of Philadelphia Composition and methods for highly efficient gene transfer using AAV capsid variants
US9738879B2 (en) 2012-04-27 2017-08-22 Duke University Genetic correction of mutated genes
JP2015529466A (ja) 2012-09-14 2015-10-08 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 鎌状赤血球症の幹細胞遺伝子治療のためのレンチウイルスベクター
US9181535B2 (en) 2012-09-24 2015-11-10 The Chinese University Of Hong Kong Transcription activator-like effector nucleases (TALENs)
US9624510B2 (en) 2013-03-01 2017-04-18 The Wistar Institute Adenoviral vectors comprising partial deletions of E3
WO2014141244A1 (en) 2013-03-12 2014-09-18 Tel Hashomer Medical Research Infrastructure And Services Ltd. Synthetic peptides for the treatment of autoimmune diseases
BR112015026019A2 (pt) 2013-04-11 2017-07-25 Brigham & Womens Hospital Inc métodos e composições de tratamento de doenças autoimunes
US11414695B2 (en) 2013-05-29 2022-08-16 Agilent Technologies, Inc. Nucleic acid enrichment using Cas9
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
KR20160089528A (ko) 2013-12-12 2016-07-27 더 브리검 앤드 우먼즈 하스피털, 인크. 신경퇴행성 질환의 치료
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
US20160060358A1 (en) * 2014-08-28 2016-03-03 California Institute Of Technology Induction of antigen-specific tolerance
EP3188749A4 (en) * 2014-09-03 2018-06-13 Moderna Therapeutics, Inc. Tolerogenic compositions and methods
CN107106543A (zh) 2014-11-12 2017-08-29 布里格姆妇女医院 自身免疫性疾病中的褪黑激素
CA2980730A1 (en) * 2015-03-23 2016-09-29 The Brigham And Women's Hospital, Inc. Tolerogenic nanoparticles for treating diabetes mellitus
GB201506112D0 (en) 2015-04-10 2015-05-27 Midatech Ltd And Inst Nationale De La Sant� Et De Al Rech Medicale And University College Car Nanoparticle-based antigen specific immunotherapy
WO2016168670A2 (en) 2015-04-16 2016-10-20 Longevica Therapeutics Inc. Methods for extending lifespan and methods of screening known pharmacological agents for new uses
EP3095440B1 (en) * 2015-05-19 2020-01-15 PLS-Design GmbH Antigen-specific immunotherapy using tolerizing liposomes
EP3713937A2 (en) 2017-11-20 2020-09-30 Ariagen, Inc. Indole compounds as aryl hydrocarbon receptor (ahr) modulators
JP2021515037A (ja) 2018-02-26 2021-06-17 アントルクス，インコーポレーテッド 寛容原性リポソーム及びその使用方法
US20230054194A1 (en) 2018-11-19 2023-02-23 Ariagen, Inc. Methods of Treating Cancer

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20170121683A1 (en) * 2015-11-03 2017-05-04 Regents Of The University Of Minnesota Aryl hydrocarbon receptor disruption and enhancement

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US20200405642A1 (en) 2020-12-31
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