SG11201808422QA - Gene therapy for treating hemophilia a - Google Patents
Gene therapy for treating hemophilia aInfo
- Publication number
- SG11201808422QA SG11201808422QA SG11201808422QA SG11201808422QA SG11201808422QA SG 11201808422Q A SG11201808422Q A SG 11201808422QA SG 11201808422Q A SG11201808422Q A SG 11201808422QA SG 11201808422Q A SG11201808422Q A SG 11201808422QA SG 11201808422Q A SG11201808422Q A SG 11201808422QA
- Authority
- SG
- Singapore
- Prior art keywords
- international
- pct
- treating hemophilia
- phoenixville
- howson
- Prior art date
Links
Classifications
-
- 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
- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- A61K38/36—Blood coagulation or fibrinolysis factors
- A61K38/37—Factors VIII
-
- 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
- A61K48/0075—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 delivery route, e.g. oral, subcutaneous
-
- 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
- A61K9/10—Dispersions; Emulsions
-
- 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
- A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
-
- 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
-
- 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
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
-
- 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
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14132—Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biotechnology (AREA)
- Organic Chemistry (AREA)
- Epidemiology (AREA)
- Biomedical Technology (AREA)
- Zoology (AREA)
- Molecular Biology (AREA)
- Wood Science & Technology (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Hematology (AREA)
- Virology (AREA)
- Microbiology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Physics & Mathematics (AREA)
- Diabetes (AREA)
- Biophysics (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Plant Pathology (AREA)
- Immunology (AREA)
- Dermatology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Dispersion Chemistry (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Abstract
INTERNATIONAL APPLICATION PUBLISHED UNDER THE PATENT COOPERATION TREATY (PCT) (19) World Intellectual Property Organization International Bureau (10) International Publication Number (43) International Publication Date WO 2017/180857 Al 19 October 2017 (19.10.2017) WIPO I PCT 111111111111110111011111111111010111110 0111111011111H111111111111111111110111111 (51) International Patent Classification: A61K 48/00 (2006.01) C07K 14/755 (2006.01) C12N 15/86 (2006.01) (21) International Application Number: PCT/US2017/027396 (22) International Filing Date: 13 April 2017 (13.04.2017) (25) Filing Language: English (26) Publication Language: English (30) Priority Data: 62/323,336 15 April 2016 (15.04.2016) US 62/331,807 4 May 2016 (04.05.2016) US 62/428,866 1 December 2016 (01.12.2016) 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, 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). ished: with international search report (Art. 21(3)) before the expiration of the time limit for amending the claims and to be republished in the event of receipt of amendments (Rule 48.2(h)) with sequence listing part of description (Rule 5.2(a)) (71) Applicant: THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA [US/US]; 3160 Chestnut Street, Suite 200, Philadelphia, PA 19104 (US). (72) Inventors: WANG, Lili; 35 Sheffield Court, Phoenixville, PA 19460 (US). WILSON, James, M.; 1831 Delancey Street, Philadelphia, PA 19103 (US). SIDRANE, Jenny, Publ Agnes; 1280 W Evergreen Drive, Phoenixville, PA 19460 (US). (74) Agents: SCHALLER, Colleen M. et al.; Howson & Howson LLP, 350 Sentry Parkway, Building 620, Suite 210, Blue Bell, PA 19422 (US). W O 20 17 / 180857 Al (54) Title: GENE THERAPY FOR TREATING HEMOPHILIA A (57) : Compositions and regimens useful in treating hemophilia A are provided. The compositions include recombinant ad- eno-associated virus (rAAV) with a transthyretin enhancer and promoter driving expression of a human Factor VIII.
Applications Claiming Priority (4)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201662323336P | 2016-04-15 | 2016-04-15 | |
US201662331807P | 2016-05-04 | 2016-05-04 | |
US201662428866P | 2016-12-01 | 2016-12-01 | |
PCT/US2017/027396 WO2017180857A1 (en) | 2016-04-15 | 2017-04-13 | Gene therapy for treating hemophilia a |
Publications (1)
Publication Number | Publication Date |
---|---|
SG11201808422QA true SG11201808422QA (en) | 2018-10-30 |
Family
ID=59253992
Family Applications (2)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
SG11201808422QA SG11201808422QA (en) | 2016-04-15 | 2017-04-13 | Gene therapy for treating hemophilia a |
SG10202009849WA SG10202009849WA (en) | 2016-04-15 | 2017-04-13 | Gene therapy for treating hemophilia a |
Family Applications After (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
SG10202009849WA SG10202009849WA (en) | 2016-04-15 | 2017-04-13 | Gene therapy for treating hemophilia a |
Country Status (16)
Country | Link |
---|---|
US (3) | US11779656B2 (en) |
EP (1) | EP3452102A1 (en) |
JP (2) | JP7347933B2 (en) |
KR (1) | KR102450833B1 (en) |
CN (1) | CN109562191A (en) |
AU (1) | AU2017248659B2 (en) |
BR (1) | BR112018071200A2 (en) |
CA (1) | CA3019425A1 (en) |
IL (1) | IL262215B2 (en) |
MX (1) | MX2018012537A (en) |
PE (1) | PE20181922A1 (en) |
RU (1) | RU2762257C2 (en) |
SA (1) | SA518400233B1 (en) |
SG (2) | SG11201808422QA (en) |
TW (1) | TWI791433B (en) |
WO (1) | WO2017180857A1 (en) |
Families Citing this family (14)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO2017180861A1 (en) | 2016-04-15 | 2017-10-19 | The Trustees Of The University Of Pennsulvania | Gene therapy for treating hemophilia b |
JOP20210054A1 (en) * | 2018-09-21 | 2020-03-21 | Nightstarx Ltd | Compositions and methods for treating retinitis pigmentosa |
CA3115524A1 (en) * | 2018-10-15 | 2020-04-23 | Win Den Cheung | Methods for measuring the infectivity of replication defective viral vectors and viruses |
KR20210133242A (en) * | 2019-02-26 | 2021-11-05 | 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 | Adeno-associated viral vector delivery of B-sarcoglycans and treatment of muscular dystrophy |
US20220177545A1 (en) * | 2019-03-13 | 2022-06-09 | Generation Bio Co. | Non-viral dna vectors and uses thereof for expressing fviii therapeutics |
CN114929735A (en) * | 2019-11-01 | 2022-08-19 | 自由行疗法有限公司 | Factor VIII constructs |
CN111218446B (en) * | 2019-12-25 | 2023-03-28 | 劲帆生物医药科技(武汉)有限公司 | Liver specificity promoter and application thereof |
US20230304034A1 (en) | 2020-05-12 | 2023-09-28 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
WO2022165245A1 (en) * | 2021-01-29 | 2022-08-04 | The Trustees Of The University Of Pennsylvania | Compositions and method of use of mutant ace2 decoy variants |
CA3209779A1 (en) | 2021-02-01 | 2022-08-04 | Regenxbio Inc. | Gene therapy for neuronal ceroid lipofuscinoses |
WO2022165421A1 (en) * | 2021-02-01 | 2022-08-04 | The Trustees Of The University Of Pennsylvania | Compositions and methods for treatment of niemann pick type a disease |
WO2023087019A2 (en) | 2021-11-15 | 2023-05-19 | The Trustees Of The University Of Pennsylvania | Compositions for drg-specific reduction of transgene expression |
WO2023205300A2 (en) * | 2022-04-20 | 2023-10-26 | Asklepios Biopharmaceutical, Inc. | Therapeutic adeno-associated virus using codon optimized nucleic acid encoding factor viii |
WO2023240222A2 (en) * | 2022-06-10 | 2023-12-14 | University Of Florida Research Foundation, Incorporated | Immune tolerance induction to viral capsids |
Family Cites Families (64)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US5595886A (en) | 1986-01-27 | 1997-01-21 | Chiron Corporation | Protein complexes having Factor VIII:C activity and production thereof |
US5661008A (en) | 1991-03-15 | 1997-08-26 | Kabi Pharmacia Ab | Recombinant human factor VIII derivatives |
US5439824A (en) | 1993-05-06 | 1995-08-08 | The United States Of America | Increased expression of α-1-antitrypsin in expression vectors through the inclusion of intron II |
US6268212B1 (en) | 1993-10-18 | 2001-07-31 | Amgen Inc. | Tissue specific transgene expression |
US6818439B1 (en) | 1994-12-30 | 2004-11-16 | Chiron Corporation | Methods for administration of recombinant gene delivery vehicles for treatment of hemophilia and other disorders |
WO1999029848A1 (en) | 1997-12-05 | 1999-06-17 | The Immune Response Corporation | Novel vectors and genes exhibiting increased expression |
US6783961B1 (en) | 1999-02-26 | 2004-08-31 | Genset S.A. | Expressed sequence tags and encoded human proteins |
US6797505B2 (en) | 1998-05-27 | 2004-09-28 | Cell Genesys, Inc. | Recombinant AAV vectors for gene therapy of hemophilia A |
US6221349B1 (en) | 1998-10-20 | 2001-04-24 | Avigen, Inc. | Adeno-associated vectors for expression of factor VIII by target cells |
US6200560B1 (en) | 1998-10-20 | 2001-03-13 | Avigen, Inc. | Adeno-associated virus vectors for expression of factor VIII by target cells |
EP1038959A1 (en) | 1999-03-17 | 2000-09-27 | Aventis Behring Gesellschaft mit beschränkter Haftung | Factor VIII without B-domain, comprising one or more insertions of a truncated intron I of factor IX |
US6610906B1 (en) | 1999-06-09 | 2003-08-26 | The Regents Of The University Of Michigan | Nucleotide sequences for gene regulation and methods of use thereof |
EP1224312A1 (en) | 1999-10-12 | 2002-07-24 | The University of North Carolina at Chapel Hill | Adeno-associated virus vectors encoding factor viii and methods of using the same |
WO2001036620A2 (en) | 1999-11-16 | 2001-05-25 | Genzyme Corporation | Vectors and transgenies with regulatory elements for gene delivery to the liver |
IL151857A0 (en) | 2000-03-22 | 2003-04-10 | Octagene Gmbh | Production of recombinant blood clotting factors in human cell lines |
US6974667B2 (en) | 2000-06-14 | 2005-12-13 | Gene Logic, Inc. | Gene expression profiles in liver cancer |
US7351813B2 (en) | 2000-06-20 | 2008-04-01 | The Board Of Trustees Of The Leland Stanford Junior University | Liver-specific gene expression cassettes, and methods of use |
US6812339B1 (en) | 2000-09-08 | 2004-11-02 | Applera Corporation | Polymorphisms in known genes associated with human disease, methods of detection and uses thereof |
US20030147853A1 (en) | 2001-03-14 | 2003-08-07 | Mcclelland Alan | Recombinant adeno-associated virus-mediated gene transfer via retroductal infusion of virions |
US8021875B2 (en) | 2001-08-27 | 2011-09-20 | Roche Madison Inc. | Methods for expression of transgenes |
EP1572889B1 (en) | 2001-10-05 | 2008-12-17 | Expression Therapeutics, LLC | Nucleic acid and amino acid sequences encoding high-level expressor factor viii polypeptides and methods of use |
DK1310571T3 (en) | 2001-11-13 | 2006-06-19 | Univ Pennsylvania | Method for identifying unknown adeno-associated virus sequences (AAV sequences) and a kit for the method |
EP1453547B1 (en) | 2001-12-17 | 2016-09-21 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (aav) serotype 8 sequences, vectors containing same, and uses therefor |
EP2434420A3 (en) | 2003-08-01 | 2012-07-25 | Dna Twopointo Inc. | Systems and methods for biopolymer engineering |
US8005620B2 (en) | 2003-08-01 | 2011-08-23 | Dna Twopointo Inc. | Systems and methods for biopolymer engineering |
ES2411479T3 (en) | 2003-09-30 | 2013-07-05 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus clades (AAV), sequences, vectors that contain them, and uses thereof |
WO2005040213A1 (en) | 2003-10-16 | 2005-05-06 | Zlb Behring Gmbh | MODIFIED cDNA FOR HIGH EXPRESSION LEVELS OF FACTOR VIII AND ITS DERIVATIVES |
CA2547569C (en) | 2003-12-03 | 2013-04-16 | University Of Rochester | Recombinant factor viii having increased specific activity |
EP2438931B1 (en) | 2004-09-22 | 2013-11-13 | St. Jude Children's Research Hospital | Improved expression of factor IX in gene therapy vectors |
US8008468B2 (en) | 2005-02-16 | 2011-08-30 | Benitec, Inc. | RNAi expression constructs with liver-specific enhancer/promoter |
WO2006102072A2 (en) | 2005-03-23 | 2006-09-28 | The Trustees Of The University Of Pennsylvania | Use of a pa131 polypeptide in treatment of atherosclerosis |
AU2006233638A1 (en) | 2005-04-14 | 2006-10-19 | Csl Behring Gmbh | Modified coagulation Factor VIII with enhanced stability and its derivates |
WO2007016331A2 (en) | 2005-07-29 | 2007-02-08 | Children's Hospital Medical Center | Classification and diagnosis of the molecular basis of cholestasis |
US7943374B2 (en) | 2005-08-21 | 2011-05-17 | Markus Hildinger | Super-size adeno-associated viral vector harboring a recombinant genome larger than 5.7 kb |
WO2007078599A2 (en) | 2005-12-16 | 2007-07-12 | The Board Of Trustees Of The Leland Stanford Junior University | Functional arrays for high throughput characterization of gene expression regulatory elements |
US9150882B2 (en) | 2006-01-31 | 2015-10-06 | The Board Of Trustees Of The Leland Stanford Junior University | Self-complementary parvoviral vectors, and methods for making and using the same |
WO2007120533A2 (en) * | 2006-03-30 | 2007-10-25 | The Board Of Trustees Of The Leland Stanford Junior University | Minigene expression cassette |
EP2037892B1 (en) | 2006-06-19 | 2015-03-18 | Asklepios Biopharmaceutical, Inc. | Modified factor viii and factor ix genes and vectors for gene therapy |
WO2008073303A2 (en) | 2006-12-07 | 2008-06-19 | Switchgear Genomics | Transcriptional regulatory elements of biological pathways, tools, and methods |
JP5448839B2 (en) | 2006-12-22 | 2014-03-19 | ツェー・エス・エル・ベーリング・ゲー・エム・ベー・ハー | Modified clotting factor with long half-life in vivo |
US20100120628A1 (en) | 2007-02-06 | 2010-05-13 | Genizon Biosciences Inc. | Genemap of the human genes associated with adhd |
KR101235560B1 (en) | 2008-02-14 | 2013-02-25 | 재단법인 목암생명공학연구소 | Expression vector suitable for expression of a coding sequence for gene therapy |
KR100981092B1 (en) | 2008-02-29 | 2010-09-08 | 고려대학교 산학협력단 | Recombinant expression vector system for variants of coagulation factor VIII and von Willebrand factor |
WO2009122401A2 (en) | 2008-04-03 | 2009-10-08 | Yeda Research And Development Co. Ltd. | Methods of expressing a polypeptide in the brain and nucleic acid constructs capable of same |
JP5797551B2 (en) | 2008-04-22 | 2015-10-21 | フエー・イー・ベー・フエー・ゼツト・ウエー | Liver-specific nucleic acid regulatory elements and methods and uses thereof |
US20110306512A1 (en) | 2008-06-25 | 2011-12-15 | Dxterity Diagnostics | Gene Expression Profiling for Identification, Monitoring, and Treatment of Osteoarthritis |
KR101034811B1 (en) | 2008-08-25 | 2011-05-16 | 단국대학교 산학협력단 | Recombinant adenovirus comprising tissu-specific promoter and tumor-targeting trans-splicing ribozyme and uses thereof |
WO2010109053A1 (en) | 2009-03-27 | 2010-09-30 | Proyeto De Biomedicina Cima, S.L. | Methods and compositions for the treatment of cirrhosis and liver fibrosis |
CN101935674A (en) * | 2009-06-30 | 2011-01-05 | 中国医学科学院血液学研究所 | Adenovirus vector for gene therapy on hemophilia B and application thereof |
GB0911870D0 (en) | 2009-07-08 | 2009-08-19 | Ucl Business Plc | Optimised coding sequence and promoter |
EP2542247A4 (en) | 2010-03-01 | 2014-05-07 | Philadelphia Children Hospital | Nucleic acids for targeting multiple regions of the hcv genome |
US9315825B2 (en) * | 2010-03-29 | 2016-04-19 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
JP5922095B2 (en) | 2010-03-29 | 2016-05-24 | ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | Pharmacologically induced transgene ablation system |
EP2394667A1 (en) | 2010-06-10 | 2011-12-14 | Laboratorios Del Dr. Esteve, S.A. | Vectors and sequences for the treatment of diseases |
KR101990068B1 (en) * | 2010-09-02 | 2019-06-17 | 몰메드 에스피에이 | Stable production of lentiviral vectors |
FR2977562B1 (en) | 2011-07-06 | 2016-12-23 | Gaztransport Et Technigaz | SEALED AND THERMALLY INSULATING TANK INTEGRATED IN A CARRIER STRUCTURE |
AU2013336601B2 (en) | 2012-10-26 | 2018-01-25 | Vrije Universiteit Brussel | Vector for liver-directed gene therapy of hemophilia and methods and use thereof |
US10398787B2 (en) * | 2012-10-26 | 2019-09-03 | Vrije Universiteit Brussel | Vectors for liver-directed gene therapy of hemophilia and methods and use thereof |
WO2015012924A2 (en) | 2013-04-29 | 2015-01-29 | The Trustees Of The University Of Pennsylvania | Tissue preferential codon modified expression cassettes, vectors containing same, and use thereof |
JP6735672B2 (en) | 2013-09-12 | 2020-08-05 | バイオマリン ファーマシューティカル インコーポレイテッド | Adeno-associated virus factor VIII vector |
EP3253786A4 (en) * | 2015-02-06 | 2018-10-17 | The University of North Carolina at Chapel Hill | Optimized human clotting factor viii gene expression cassettes and their use |
US20180110877A1 (en) * | 2015-04-27 | 2018-04-26 | The Trustees Of The University Of Pennsylvania | DUAL AAV VECTOR SYSTEM FOR CRISPR/Cas9 MEDIATED CORRECTION OF HUMAN DISEASE |
TW202302627A (en) * | 2015-11-13 | 2023-01-16 | 日商武田藥品工業股份有限公司 | Viral vectors encoding recombinant fviii variants with increased expression for gene therapy of hemophilia a |
US11028372B2 (en) | 2015-12-11 | 2021-06-08 | The Trustees Of The University Of Pennsylvania | Scalable purification method for AAVRH10 |
-
2017
- 2017-04-13 KR KR1020187032617A patent/KR102450833B1/en active IP Right Grant
- 2017-04-13 CN CN201780023787.5A patent/CN109562191A/en active Pending
- 2017-04-13 TW TW106112302A patent/TWI791433B/en active
- 2017-04-13 SG SG11201808422QA patent/SG11201808422QA/en unknown
- 2017-04-13 US US16/093,798 patent/US11779656B2/en active Active
- 2017-04-13 WO PCT/US2017/027396 patent/WO2017180857A1/en active Application Filing
- 2017-04-13 RU RU2018136611A patent/RU2762257C2/en active
- 2017-04-13 BR BR112018071200-0A patent/BR112018071200A2/en unknown
- 2017-04-13 MX MX2018012537A patent/MX2018012537A/en unknown
- 2017-04-13 SG SG10202009849WA patent/SG10202009849WA/en unknown
- 2017-04-13 JP JP2018554331A patent/JP7347933B2/en active Active
- 2017-04-13 EP EP17734159.1A patent/EP3452102A1/en active Pending
- 2017-04-13 AU AU2017248659A patent/AU2017248659B2/en active Active
- 2017-04-13 CA CA3019425A patent/CA3019425A1/en active Pending
- 2017-04-13 PE PE2018001994A patent/PE20181922A1/en unknown
-
2018
- 2018-10-08 IL IL262215A patent/IL262215B2/en unknown
- 2018-10-14 SA SA518400233A patent/SA518400233B1/en unknown
-
2019
- 2019-11-22 US US16/691,822 patent/US10888628B2/en active Active
-
2022
- 2022-01-27 JP JP2022011052A patent/JP2022062121A/en active Pending
-
2023
- 2023-08-08 US US18/446,243 patent/US20240000974A1/en active Pending
Also Published As
Publication number | Publication date |
---|---|
WO2017180857A1 (en) | 2017-10-19 |
JP2019513794A (en) | 2019-05-30 |
US20200085975A1 (en) | 2020-03-19 |
TWI791433B (en) | 2023-02-11 |
JP7347933B2 (en) | 2023-09-20 |
CA3019425A1 (en) | 2017-10-19 |
JP2022062121A (en) | 2022-04-19 |
PE20181922A1 (en) | 2018-12-11 |
SG10202009849WA (en) | 2020-11-27 |
RU2762257C2 (en) | 2021-12-17 |
KR102450833B1 (en) | 2022-10-05 |
RU2018136611A3 (en) | 2020-08-11 |
EP3452102A1 (en) | 2019-03-13 |
RU2018136611A (en) | 2020-05-15 |
IL262215A (en) | 2018-11-29 |
KR20190032274A (en) | 2019-03-27 |
US20190343966A1 (en) | 2019-11-14 |
SA518400233B1 (en) | 2022-08-23 |
CN109562191A (en) | 2019-04-02 |
IL262215B2 (en) | 2023-07-01 |
BR112018071200A2 (en) | 2019-04-09 |
TW201741458A (en) | 2017-12-01 |
MX2018012537A (en) | 2019-02-25 |
US10888628B2 (en) | 2021-01-12 |
US20240000974A1 (en) | 2024-01-04 |
IL262215B1 (en) | 2023-03-01 |
US11779656B2 (en) | 2023-10-10 |
AU2017248659B2 (en) | 2022-08-11 |
AU2017248659A1 (en) | 2018-10-18 |
Similar Documents
Publication | Publication Date | Title |
---|---|---|
SG11201808422QA (en) | Gene therapy for treating hemophilia a | |
SG11201909949XA (en) | Targeted immunotolerance | |
SG11201906297QA (en) | Nucleic acids encoding crispr-associated proteins and uses thereof | |
SG11201806270XA (en) | Gene therapy for treating mucopolysaccharidosis type i | |
SG11201908391XA (en) | Methods for modulating an immune response | |
SG11201808990QA (en) | Compositions for topical application of compounds | |
SG11201811432WA (en) | Rna for cancer therapy | |
SG11201808812RA (en) | Novel aav8 mutant capsids and compositions containing same | |
SG11201808176TA (en) | Gene therapy for treating mucopolysaccharidosis type ii | |
SG11201907714UA (en) | Adeno-associated virus (aav) clade f vector and uses therefor | |
SG11201809344QA (en) | Process for synthesizing 2-hydroxy-6-((2-(1-isopropyl-1h-pyrazol-5-yl)-pyridin-3-yl)methoxy)benzaldehyde | |
SG11201906147VA (en) | Optimized strategy for exon skipping modifications using crispr/cas9 with triple guide sequences | |
SG11201811232XA (en) | Treatment of amd using aav2 variant with aflibercept | |
SG11201909322VA (en) | Treatment of asthma with anti-tslp antibody | |
SG11201805645QA (en) | Lsd1 inhibitors | |
SG11201804814YA (en) | Materials and methods for delivering nucleic acids to cochlear and vestibular cells | |
SG11201907023UA (en) | Method of reducing neutropenia | |
SG11201810525XA (en) | Anti-gitr antibodies and uses thereof | |
SG11201805709RA (en) | Anti-pro/latent myostatin antibodies and methods of use thereof | |
SG11201900815UA (en) | Expression of pten-long with ocolytic viruses | |
SG11201811292RA (en) | Compositions and methods for the depletion of cells | |
SG11201906392WA (en) | Nucleoside-modified rna for inducing an immune response against zika virus | |
SG11201900554YA (en) | Spiro-lactam nmda modulators and methods of using same | |
SG11201500005SA (en) | Pyrimidine pyrazolyl derivatives | |
SG11201804704PA (en) | Compositions and methods for decreasing tau expression |