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
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- 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
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- 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
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- 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
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- 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
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- 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
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- 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
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- 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
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- 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)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (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)
- Diabetes (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Plant Pathology (AREA)
- Immunology (AREA)
- Dermatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (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 (17)
| Country | Link |
|---|---|
| US (3) | US11779656B2 (en) |
| EP (1) | EP3452102B9 (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) |
| ZA (1) | ZA201806477B (en) |
Families Citing this family (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3442597A1 (en) | 2016-04-15 | 2019-02-20 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating hemophilia b |
| JOP20210054A1 (en) * | 2018-09-21 | 2020-03-21 | Nightstarx Ltd | Compositions and methods for treating retinitis pigmentosa |
| EP3867412A1 (en) * | 2018-10-15 | 2021-08-25 | REGENXBIO Inc. | Method for measuring the infectivity of replication defective viral vectors and viruses |
| WO2020176614A1 (en) * | 2019-02-26 | 2020-09-03 | Research Institute At Nationwide Children's Hospital | Adeno-associated virus vector delivery of b-sarcoglycan and the treatment of muscular dystrophy |
| EP3938523A4 (en) * | 2019-03-13 | 2023-06-21 | Generation Bio Co. | Non-viral dna vectors and uses thereof for expressing fviii therapeutics |
| US20220387558A1 (en) * | 2019-11-01 | 2022-12-08 | Freeline Therapeutics Limited | Transcription regulatory elements |
| 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 |
| BR112022022212A2 (en) | 2020-05-12 | 2022-12-13 | Univ Pennsylvania | COMPOSITIONS FOR SPECIFIC DRG REDUCTION OF TRANSGENE EXPRESSION |
| WO2022165246A1 (en) * | 2021-01-29 | 2022-08-04 | The Trustees Of The University Of Pennsylvania | Compositions and method of use of mutant ace2 decoy variants |
| EP4284442A1 (en) * | 2021-02-01 | 2023-12-06 | The Trustees of The University of Pennsylvania | Compositions and methods for treatment of niemann pick type a disease |
| JP2024505257A (en) | 2021-02-01 | 2024-02-05 | レジェンクスバイオ インコーポレーテッド | Gene therapy for neuronal ceroid lipofuscinosis |
| 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 |
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2017
- 2017-04-13 SG SG11201808422QA patent/SG11201808422QA/en unknown
- 2017-04-13 JP JP2018554331A patent/JP7347933B2/en active Active
- 2017-04-13 BR BR112018071200-0A patent/BR112018071200A2/en unknown
- 2017-04-13 KR KR1020187032617A patent/KR102450833B1/en active IP Right Grant
- 2017-04-13 WO PCT/US2017/027396 patent/WO2017180857A1/en active Application Filing
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| JP2022062121A (en) | 2022-04-19 |
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| AU2017248659A1 (en) | 2018-10-18 |
| RU2762257C2 (en) | 2021-12-17 |
| US11779656B2 (en) | 2023-10-10 |
| SG10202009849WA (en) | 2020-11-27 |
| AU2017248659B2 (en) | 2022-08-11 |
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