IL294278A - Improved aav-abcd1 constructs and use for the treatment or prevention of adrenoleukodystrophy (ald) and/or adrenomyeloneuropathy (amn) - Google Patents
Improved aav-abcd1 constructs and use for the treatment or prevention of adrenoleukodystrophy (ald) and/or adrenomyeloneuropathy (amn)Info
- Publication number
- IL294278A IL294278A IL294278A IL29427822A IL294278A IL 294278 A IL294278 A IL 294278A IL 294278 A IL294278 A IL 294278A IL 29427822 A IL29427822 A IL 29427822A IL 294278 A IL294278 A IL 294278A
- Authority
- IL
- Israel
- Prior art keywords
- acid sequence
- seq
- optionally
- aav
- amino acid
- Prior art date
Links
Classifications
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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
-
- 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
- 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
-
- 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
- C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
- C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
- C07K14/4702—Regulators; Modulating activity
-
- 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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
-
- 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
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—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
- C12N2800/00—Nucleic acids vectors
- C12N2800/10—Plasmid DNA
- C12N2800/106—Plasmid DNA for vertebrates
- C12N2800/107—Plasmid DNA for vertebrates for mammalian
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- Molecular Biology (AREA)
- Medicinal Chemistry (AREA)
- Biomedical Technology (AREA)
- Wood Science & Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Engineering & Computer Science (AREA)
- Animal Behavior & Ethology (AREA)
- Epidemiology (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Microbiology (AREA)
- Dermatology (AREA)
- Toxicology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962955667P | 2019-12-31 | 2019-12-31 | |
| PCT/US2020/067664 WO2021138559A1 (en) | 2019-12-31 | 2020-12-31 | Improved aav-abcd1 constructs and use for treatment or prevention of adrenoleukodystrophy (ald) and/or adrenomyeloneuropathy (amn) |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| IL294278A true IL294278A (en) | 2022-08-01 |
Family
ID=76686949
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL294278A IL294278A (en) | 2019-12-31 | 2020-12-31 | Improved aav-abcd1 constructs and use for the treatment or prevention of adrenoleukodystrophy (ald) and/or adrenomyeloneuropathy (amn) |
Country Status (11)
| Country | Link |
|---|---|
| US (2) | US11779655B2 (https=) |
| EP (1) | EP4085143B1 (https=) |
| JP (1) | JP2023509443A (https=) |
| KR (1) | KR20220128632A (https=) |
| CN (1) | CN116096904A (https=) |
| AU (1) | AU2020416291A1 (https=) |
| BR (1) | BR112022013027A2 (https=) |
| CA (1) | CA3166374A1 (https=) |
| IL (1) | IL294278A (https=) |
| MX (1) | MX2022008201A (https=) |
| WO (1) | WO2021138559A1 (https=) |
Families Citing this family (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AR126727A1 (es) * | 2021-08-06 | 2023-11-08 | Wuhan Neurophth Biotechnology Ltd Company | Composición y método para tratar la neuropatía óptica hereditaria de leber causada por la mutación nd4 |
| US20240229067A9 (en) * | 2022-05-12 | 2024-07-11 | AAVnerGene Inc. | Compositions and methods for recombinant parvovirus production |
| EP4522729A1 (en) * | 2022-05-12 | 2025-03-19 | Aavnergene Inc. | Compositions and methods for recombinant parvovirus production |
| EP4536862A1 (en) * | 2022-06-09 | 2025-04-16 | Wisconsin Alumni Research Foundation | Generation of next generation recombinant aav gene therapy vectors that adopt 3d conformation |
| CN121693566A (zh) * | 2023-07-28 | 2026-03-17 | 瑞泽恩制药公司 | 使用bGH-SV40L串联PolyA在单向基因插入期间增强转基因表达 |
Family Cites Families (23)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| MX359371B (es) | 2001-11-13 | 2018-09-25 | Univ Pennsylvania | Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo. |
| PT1453547T (pt) | 2001-12-17 | 2016-12-28 | Univ Pennsylvania | Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas |
| EP2345731B1 (en) | 2003-09-30 | 2015-10-21 | The Trustees of the University of Pennsylvania | Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof |
| ES2525067T3 (es) | 2005-04-07 | 2014-12-17 | The Trustees Of The University Of Pennsylvania | Método de incremento de la función de un vector de AAV |
| US7588772B2 (en) | 2006-03-30 | 2009-09-15 | Board Of Trustees Of The Leland Stamford Junior University | AAV capsid library and AAV capsid proteins |
| EA201100225A1 (ru) * | 2008-07-23 | 2011-08-30 | Бёрингер Ингельхайм Фарма Гмбх Унд Ко. Кг | Новые регуляторные элементы |
| US8865881B2 (en) * | 2011-02-22 | 2014-10-21 | California Institute Of Technology | Delivery of proteins using adeno-associated virus (AAV) vectors |
| KR20150029756A (ko) * | 2011-06-10 | 2015-03-18 | 블루버드 바이오, 인코포레이티드. | 부신백질이영양증 및 부신척수신경병증을 위한 유전자 요법 벡터 |
| US9163259B2 (en) * | 2012-05-04 | 2015-10-20 | Novartis Ag | Viral vectors for the treatment of retinal dystrophy |
| US9938541B2 (en) * | 2012-12-25 | 2018-04-10 | Takara Bio Inc. | AAV variant |
| WO2015153889A2 (en) * | 2014-04-02 | 2015-10-08 | University Of Florida Research Foundation, Incorporated | Materials and methods for the treatment of latent viral infection |
| WO2016154055A1 (en) * | 2015-03-20 | 2016-09-29 | Bluebird Bio, Inc. | Vector formulations |
| EP3285788B1 (en) * | 2015-04-23 | 2024-12-18 | University of Massachusetts | Modulation of aav vector transgene expression |
| EP4066862A1 (en) | 2015-11-05 | 2022-10-05 | The General Hospital Corporation | Intrathecal delivery of nucleic acid sequences encoding abcd1 for treatment of adrenomyeloneuropathy |
| EP3411484B1 (en) * | 2016-02-05 | 2023-10-04 | Emory University | Injection of single-stranded or self-complementary adeno-associated virus 9 into the cerebrospinal fluid |
| WO2017137585A1 (en) * | 2016-02-12 | 2017-08-17 | Combigene Ab | Vector |
| CN107287239B (zh) * | 2016-04-11 | 2020-09-22 | 厦门继景生物技术有限责任公司 | 一种用于视网膜色素变性的基因治疗载体及药物 |
| JP7165410B2 (ja) * | 2016-08-19 | 2022-11-04 | ユニバーシティ オブ フロリダ リサーチ ファウンデーション，インコーポレイティド | 組換え自己相補的アデノ随伴ウイルスを使用して疾患を治療するための方法及び組成物 |
| CA3050690A1 (en) * | 2017-01-17 | 2018-07-26 | Dana-Farber Cancer Institute, Inc. | Compositions and methods for diagnosing and treating peroxisomal diseases |
| US12012602B2 (en) * | 2017-03-10 | 2024-06-18 | The Medical College Of Wisconsin, Inc. | Riboswitch modulated gene therapy for retinal diseases |
| US20200377887A1 (en) * | 2017-09-22 | 2020-12-03 | Voyager Therapeutics, Inc. | Compositions and methods of treating huntington's disease |
| MX2020004092A (es) * | 2017-10-18 | 2021-01-08 | Composiciones de virus adenoasociados para restaurar la funcion del gen hbb y metodos de uso de estas. | |
| GB201817470D0 (en) * | 2018-10-26 | 2018-12-12 | Univ Oxford Innovation Ltd | Gene therapy |
-
2020
- 2020-12-31 WO PCT/US2020/067664 patent/WO2021138559A1/en not_active Ceased
- 2020-12-31 JP JP2022540917A patent/JP2023509443A/ja active Pending
- 2020-12-31 AU AU2020416291A patent/AU2020416291A1/en active Pending
- 2020-12-31 KR KR1020227026264A patent/KR20220128632A/ko not_active Ceased
- 2020-12-31 BR BR112022013027A patent/BR112022013027A2/pt unknown
- 2020-12-31 IL IL294278A patent/IL294278A/en unknown
- 2020-12-31 EP EP20909162.8A patent/EP4085143B1/en active Active
- 2020-12-31 CA CA3166374A patent/CA3166374A1/en active Pending
- 2020-12-31 CN CN202080097613.5A patent/CN116096904A/zh active Pending
- 2020-12-31 MX MX2022008201A patent/MX2022008201A/es unknown
-
2021
- 2021-12-21 US US17/557,409 patent/US11779655B2/en active Active
-
2023
- 2023-08-30 US US18/458,665 patent/US20240009326A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| CA3166374A1 (en) | 2021-07-08 |
| US20240009326A1 (en) | 2024-01-11 |
| EP4085143B1 (en) | 2025-08-13 |
| WO2021138559A1 (en) | 2021-07-08 |
| US20220362403A2 (en) | 2022-11-17 |
| BR112022013027A2 (pt) | 2022-09-06 |
| JP2023509443A (ja) | 2023-03-08 |
| MX2022008201A (es) | 2022-10-18 |
| US20220175965A1 (en) | 2022-06-09 |
| AU2020416291A1 (en) | 2022-07-21 |
| KR20220128632A (ko) | 2022-09-21 |
| EP4085143A4 (en) | 2024-02-28 |
| EP4085143A1 (en) | 2022-11-09 |
| CN116096904A (zh) | 2023-05-09 |
| US11779655B2 (en) | 2023-10-10 |
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