SG11201901718VA - Gene therapy for patients with fanconi anemia - Google Patents

Gene therapy for patients with fanconi anemia

Info

Publication number
SG11201901718VA
SG11201901718VA SG11201901718VA SG11201901718VA SG11201901718VA SG 11201901718V A SG11201901718V A SG 11201901718VA SG 11201901718V A SG11201901718V A SG 11201901718VA SG 11201901718V A SG11201901718V A SG 11201901718VA SG 11201901718V A SG11201901718V A SG 11201901718VA
Authority
SG
Singapore
Prior art keywords
new york
suite
rtw
llc
international
Prior art date
Application number
SG11201901718VA
Inventor
Juan A Bueren
Paula Rio
Susana Navarro
Julian Sevilla
Jose Carlos Segovia
Africa Gonzalez
Jose Antonio Casado
Guillermo Guenechea
Original Assignee
Centro De Investigaciones Energeticas Medioambientales Y Tecnologicas
Fundacion Instituto De Investig Sanitaria Fundacion Jimenez Diaz
Consorcio Centro De Investigacion Biomedica En Red M P
Fundacion Para La Investigacion Biomedica Del Hospital Infantil Univ Nino Jesus
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 Centro De Investigaciones Energeticas Medioambientales Y Tecnologicas, Fundacion Instituto De Investig Sanitaria Fundacion Jimenez Diaz, Consorcio Centro De Investigacion Biomedica En Red M P, Fundacion Para La Investigacion Biomedica Del Hospital Infantil Univ Nino Jesus filed Critical Centro De Investigaciones Energeticas Medioambientales Y Tecnologicas
Publication of SG11201901718VA publication Critical patent/SG11201901718VA/en

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/06—Antianaemics
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0634—Cells from the blood or the immune system
    • C12N5/0647—Haematopoietic stem cells; Uncommitted or multipotent progenitors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • C12N9/12—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • C12N9/1217—Phosphotransferases with a carboxyl group as acceptor (2.7.2)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/02—Phosphotransferases with a carboxy group as acceptor (2.7.2)
    • C12Y207/02003—Phosphoglycerate kinase (2.7.2.3)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2510/00—Genetically modified cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2730/00—Reverse transcribing DNA viruses
    • C12N2730/00011—Details
    • C12N2730/10011—Hepadnaviridae
    • C12N2730/10111—Orthohepadnavirus, e.g. hepatitis B virus
    • C12N2730/10141—Use of virus, viral particle or viral elements as a vector
    • C12N2730/10143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2730/00—Reverse transcribing DNA viruses
    • C12N2730/00011—Details
    • C12N2730/10011—Hepadnaviridae
    • C12N2730/10111—Orthohepadnavirus, e.g. hepatitis B virus
    • C12N2730/10171—Demonstrated in vivo effect
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041—Use of virus, viral particle or viral elements as a vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/15043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15071—Demonstrated in vivo effect

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Hematology (AREA)
  • Molecular Biology (AREA)
  • Cell Biology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biophysics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Developmental Biology & Embryology (AREA)
  • Immunology (AREA)
  • Virology (AREA)
  • Diabetes (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property 1111111011110 DOI HIM 0111 II 0 0111111111111111111111111111111111111110111111 Organization International Bureau (10) International Publication Number (43) International Publication Date .....0\"\" WO 2018/049273 Al 15 March 2018 (15.03.2018) W I P0 I P C T (51) International Patent Classification: TIL UNIVERSITARIO NINO JESUS [ES/ES]; Avda Cl 2N 15/85 (2006.01) C12Q 1/68 (2006.01) Menendez Pelayo 65, 28009 Madrid (ES). Cl 2N 15/63 (2006.01) (71) Applicant (for OM only): SHERIN, Mridula [US/US]; (21) International Application Number: 1700 Seventh Avenue, Suite 1900, Seattle, Washington PCT/U52017/050837 98101 (US). (22) International Filing Date: (72) Inventors; and 08 September 2017 (08.09.2017) (71) Applicants: BUEREN, Juan A. [ES/US]; c/o RTW Invest- (25) Filing Language: English ments, LLC, 250 W 55th St., Suite A, New York, New York 10019 (US). RIO, Paula [ES/US]; c/o RTW Invest- (26) Publication Language: English ments, LLC, 250 W 55th St., Suite A, New York, New (30) Priority Data: York 10019 (US). NAVARRO, Susana [ES/US]; c/o RTW 62/385,185 08 September 2016 (08.09.2016) US Investments, LLC, 250 W 55th St., Suite A, New York, 62/412,028 24 October 2016 (24.10.2016) US New York 10019 (US). SEVILLA, Julian [ES/US]; c/o RTW Investments, LLC, 250 W 55th St., Suite A, New (71) Applicants: CENTRO DE INVESTIGACIONES York, New York 10019 (US). SEGOVIA, Jose Carlos ENERGETICAS MEDIOAMBIENTALES Y TEC- [ES/US]; c/o RTW Investments, LLC, 250 W 55th St., NOLOGICAS [ES/ES]; Avda. de la Complutense, Suite A, New York, New York 10019 (US). GONZALEZ, 40, 28040 Madrid (ES). FUNDACION INSTITUTO Africa [ES/US]; c/o RTW Investments, LLC, 250 W 55th DE INVESTIGACION SANITARIA FUNDACION St., Suite A, New York, New York 10019 (US). CASA- _ JIMENEZ DIAZ [ES/ES]; Avda. de los Reyes Catoli- DO, Jose Antonio [ES/US]; c/o RTW Investments, LLC, cos, 2, 28040 Madrid (ES). CENTRO DE INVESTI- 250 W 55th St., Suite A, New York, New York 10019 GACION BIOMEDICA EN RED [ES/ES]; Monforte de (US). GUENECHEA, Guillermo [ES/US]; c/o RTW In- — = Lemos, 5, 28029 Madrid (ES). FUNDACION PARA LA vestments, LLC, 250 W 55th St., Suite A, New York, New = INVESTIGACION BIOMEDICA HOSPITAL INFAN- York 10019 (US). = (54) Title: GENE THERAPY FOR PATIENTS WITH FANCONI ANEMIA Figure 1. = = = = = = Lentiviral backbone = = A113 = 43 1-1 ,:•ouorio,-„,,„ i i ppt / w9 r .4 S D SA LTR e 4 \") ei CI' iv Therapeutic cassette 0 -.... 00 1-1 (57) : The present invention provides compositions and methods for rescuing FANCA expression in cells with diminished or C:::: ) no FANCA gene product. In particular, methods and compositions for gene therapy of Fanconi anemia are disclosed. ei O [Continued on next page] WO 2018/049273 Al MIDEDIMOMOIDEIREEMOOVIEEIMEIMOVOIS (74) Agent: SHERIN, Mridula et al.; Cooley LLP, 1299 Penn- sylvania Avenue, NW, Suite 700, Washington, District of Columbia 20004 (US). (81) Designated States (unless otherwise indicated, for every kind of national protection available): AE, AG, AL, AM, AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DJ, DK, DM, DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, HN, HR, HU, ID, IL, IN, IR, IS, JO, JP, KE, KG, KH, KN, KP, KR, KW, KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, RU, RW, SA, SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, TH, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW. (84) Designated States (unless otherwise indicated, for every kind of regional protection available): ARIPO (BW, GH, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, UG, ZM, ZW), Eurasian (AM, AZ, BY, KG, KZ, RU, TJ, TM), European (AL, AT, BE, BG, CH, CY, CZ, DE, DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, SM, TR), OAPI (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG). Published: — with international search report (Art. 21(3)) — with sequence listing part of description (Rule 5.2(a))
SG11201901718VA 2016-09-08 2017-09-08 Gene therapy for patients with fanconi anemia SG11201901718VA (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662385185P 2016-09-08 2016-09-08
US201662412028P 2016-10-24 2016-10-24
PCT/US2017/050837 WO2018049273A1 (en) 2016-09-08 2017-09-08 Gene therapy for patients with fanconi anemia

Publications (1)

Publication Number Publication Date
SG11201901718VA true SG11201901718VA (en) 2019-03-28

Family

ID=61561668

Family Applications (1)

Application Number Title Priority Date Filing Date
SG11201901718VA SG11201901718VA (en) 2016-09-08 2017-09-08 Gene therapy for patients with fanconi anemia

Country Status (11)

Country Link
US (1) US20190203225A1 (en)
EP (1) EP3510162A4 (en)
JP (2) JP7197466B2 (en)
CN (1) CN110536966A (en)
AU (2) AU2017322511B2 (en)
BR (1) BR112019004594A2 (en)
CA (1) CA3035605A1 (en)
IL (1) IL265196A (en)
MX (1) MX2019002699A (en)
SG (1) SG11201901718VA (en)
WO (1) WO2018049273A1 (en)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3093708A1 (en) * 2018-04-11 2019-10-17 Rocket Pharmaceuticals, Ltd. Compositions and methods for treating fanconi anemia
CA3106010A1 (en) * 2018-07-12 2020-01-16 Rocket Pharmaceuticals, Ltd. Gene therapy vectors for treatment of danon disease
CN110904102A (en) * 2018-09-18 2020-03-24 中国科学院上海生命科学研究院 Promoter for recombinant protein expression
US20220136042A1 (en) * 2019-02-21 2022-05-05 Roche Sequencing Solutions, Inc. Improved nucleic acid target enrichment and related methods
KR20230043869A (en) 2020-08-07 2023-03-31 스페이스크래프트 세븐, 엘엘씨 Placophilin-2 (PKP2) gene therapy using AAV vectors
JP2024518770A (en) * 2021-04-26 2024-05-02 ストーム バイオ インコーポレイテッド Compositions and methods relating to megakaryocyte-derived extracellular vesicles for Fanconi anemia
WO2023183909A2 (en) * 2022-03-25 2023-09-28 Modernatx, Inc. Polynucleotides encoding fanconi anemia, complementation group proteins for the treatment of fanconi anemia

Family Cites Families (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5952190A (en) * 1996-10-04 1999-09-14 Fanconi Anemia Research Fund, Inc. cDNA for fanconi anemia complementation group A
GB0024550D0 (en) 2000-10-06 2000-11-22 Oxford Biomedica Ltd
JP2007054069A (en) 2005-08-24 2007-03-08 Medizinische Hochschule Hannover Self-inactivating retrovirus vector
CA2760446C (en) 2009-04-30 2018-01-02 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Inducible interleukin-12
DE102009021592A1 (en) * 2009-05-15 2010-11-18 Medizinische Hochschule Hannover ASLV vector system
JP2014513727A (en) 2011-05-16 2014-06-05 ジェンザイム・コーポレーション Use of CXCR4 antagonist
CN105188767A (en) * 2012-07-25 2015-12-23 布罗德研究所有限公司 Inducible DNA binding proteins and genome perturbation tools and applications thereof
WO2014100073A2 (en) * 2012-12-21 2014-06-26 Merck Sharp & Dohme Corp. Expression vectors for recombinant protein production in mammalian cells
GB201318347D0 (en) 2013-10-16 2013-11-27 Ucl Business Plc Retroviral vectors
EP3068440B1 (en) * 2013-11-15 2020-01-08 Northwestern University Inhibition of oxidative stress in atrial fibrillation
JP2017514476A (en) 2014-05-01 2017-06-08 ユニバーシティ・オブ・ワシントン In vivo genetic manipulation using adenoviral vectors
US11030531B2 (en) * 2014-06-06 2021-06-08 Trustees Of Boston University DNA recombinase circuits for logical control of gene expression
US10350245B2 (en) 2015-01-21 2019-07-16 Fred Hutchinson Cancer Research Center Point-of-care and/or portable platform for gene therapy
WO2016183593A2 (en) * 2015-05-14 2016-11-17 The Regents Of The University Of California Prenatal therapy

Also Published As

Publication number Publication date
KR20190062426A (en) 2019-06-05
EP3510162A4 (en) 2020-02-19
JP2022160505A (en) 2022-10-19
JP2019533434A (en) 2019-11-21
RU2019108981A3 (en) 2020-12-24
JP7197466B2 (en) 2022-12-27
EP3510162A1 (en) 2019-07-17
WO2018049273A1 (en) 2018-03-15
AU2017322511B2 (en) 2021-08-26
CA3035605A1 (en) 2018-03-15
US20190203225A1 (en) 2019-07-04
MX2019002699A (en) 2019-12-16
RU2019108981A (en) 2020-10-08
BR112019004594A2 (en) 2019-07-02
CN110536966A (en) 2019-12-03
AU2021273525A1 (en) 2021-12-16
IL265196A (en) 2019-05-30
AU2017322511A1 (en) 2019-03-28

Similar Documents

Publication Publication Date Title
SG11201901718VA (en) Gene therapy for patients with fanconi anemia
SG11201810162PA (en) Polynucleotides encoding citrin for the treatment of citrullinemia type 2
SG11201903021WA (en) Anti-human 4-1 bb antibodies and use thereof
SG11201900135YA (en) N-(phenylsulfonyl)benzamides and related compounds as bcl-2 inhibitors
SG11201407486PA (en) Compositions and methods for modulating utrn expression
SG11201809344QA (en) Process for synthesizing 2-hydroxy-6-((2-(1-isopropyl-1h-pyrazol-5-yl)-pyridin-3-yl)methoxy)benzaldehyde
SG11201804281RA (en) Suture deployment of prosthetic heart valve
SG11201807809XA (en) A transient commensal microorganism for improving gut health
SG11201808222RA (en) Methods of treatment of cholestatic diseases
SG11201908971RA (en) Erbb-2 and erbb3 binding bispecific antibodies for use in the treatment f cells that have an nrg1 fusion gene
SG11201810196RA (en) Cancer treatments
SG11201901494UA (en) Acid-alpha glucosidase variants and uses thereof
SG11201807784SA (en) 3-desoxy derivative and pharmaceutical compositions thereof
SG11201807051VA (en) Replication competent attenuated vaccinia viruses with deletion of thymidine kinase with and without the expression of human flt3l or gm-csf for cancer immunotherapy
SG11201907673YA (en) Pharmaceutical compositions for combination therapy
SG11201901351UA (en) A patient interface, system and method
SG11201808138YA (en) Substituted indole derivatives as dengue viral replication inhibitors
SG11201810441QA (en) Invasive medical devices including magnetic region and systems and methods
SG11201900546UA (en) Spiro-lactam and bis-spiro-lactam nmda receptor modulators and uses thereof
SG11201808525UA (en) Anti-complement factor bb antibodies and uses thereof
SG11201808762XA (en) Diagnostic methods for patient specific therapeutic decision making in cancer care
SG11201804094UA (en) Pack for preparing food or beverage products
SG11201900238UA (en) Compounds and methods for modulation of smn2
SG11201900074WA (en) Soluble fibroblast growth factor receptor 3 (sfgfr3) polypeptides and uses thereof
SG11201901063SA (en) Therapeutic agents for neurodegenerative diseases