IL317905A - Increasing tissue-specific gene delivery through capsid modification - Google Patents
Increasing tissue-specific gene delivery through capsid modificationInfo
- Publication number
- IL317905A IL317905A IL317905A IL31790524A IL317905A IL 317905 A IL317905 A IL 317905A IL 317905 A IL317905 A IL 317905A IL 31790524 A IL31790524 A IL 31790524A IL 317905 A IL317905 A IL 317905A
- Authority
- IL
- Israel
- Prior art keywords
- pharmaceutical composition
- aavrh74
- amino acid
- cell
- substitution
- Prior art date
Links
Classifications
-
- 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
-
- 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
- A61K35/66—Microorganisms or materials therefrom
- A61K35/76—Viruses; Subviral particles; Bacteriophages
-
- 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
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
-
- 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
- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
-
- 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
- C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
-
- 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
- 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
- C12N2750/14145—Special targeting system for viral vectors
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Molecular Biology (AREA)
- Medicinal Chemistry (AREA)
- Virology (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Epidemiology (AREA)
- General Chemical & Material Sciences (AREA)
- Neurology (AREA)
- Immunology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Physical Education & Sports Medicine (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Mycology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Peptides Or Proteins (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862644317P | 2018-03-16 | 2018-03-16 | |
| PCT/US2019/022353 WO2019178412A1 (en) | 2018-03-16 | 2019-03-14 | Increasing tissue specific gene delivery by capsid modification |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| IL317905A true IL317905A (en) | 2025-02-01 |
Family
ID=67906893
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL317905A IL317905A (en) | 2018-03-16 | 2019-03-14 | Increasing tissue-specific gene delivery through capsid modification |
| IL277270A IL277270B2 (en) | 2018-03-16 | 2019-03-14 | Increasing tissue-specific gene delivery through capsid modification |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL277270A IL277270B2 (en) | 2018-03-16 | 2019-03-14 | Increasing tissue-specific gene delivery through capsid modification |
Country Status (11)
| Country | Link |
|---|---|
| US (2) | US12385062B2 (https=) |
| EP (1) | EP3765624A4 (https=) |
| JP (3) | JP7304878B2 (https=) |
| KR (3) | KR20250111395A (https=) |
| CN (2) | CN111819286B (https=) |
| AU (2) | AU2019234918C1 (https=) |
| BR (1) | BR112020018354A2 (https=) |
| CA (3) | CA3293599A1 (https=) |
| IL (2) | IL317905A (https=) |
| SG (1) | SG11202008687XA (https=) |
| WO (1) | WO2019178412A1 (https=) |
Families Citing this family (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| ES2865487T3 (es) | 2015-09-28 | 2021-10-15 | Univ North Carolina Chapel Hill | Métodos y composiciones para vectores virales que evaden los anticuerpos |
| EP3765624A4 (en) * | 2018-03-16 | 2022-05-25 | Research Institute at Nationwide Children's Hospital | Increasing tissue specific gene delivery by capsid modification |
| EP3774852A1 (en) | 2018-04-03 | 2021-02-17 | Stridebio, Inc. | Antibody-evading virus vectors |
| MX2020010465A (es) | 2018-04-03 | 2021-01-08 | Vectores de virus para direccionamiento a tejidos oftalmicos. | |
| AU2019247748A1 (en) | 2018-04-03 | 2020-10-08 | Ginkgo Bioworks, Inc. | Antibody-evading virus vectors |
| AR118465A1 (es) | 2019-03-21 | 2021-10-06 | Stridebio Inc | Vectores de virus adenoasociados recombinantes |
| US12611436B2 (en) | 2019-10-17 | 2026-04-28 | Sarepta Therapeutics, Inc. | AAV transfer cassette |
| JP2022551739A (ja) | 2019-10-17 | 2022-12-13 | ストライドバイオ，インコーポレイテッド | ニーマン・ピック病ｃ型の治療のためのアデノ随伴ウイルスベクター |
| AU2021328475A1 (en) | 2020-08-19 | 2023-03-16 | Sarepta Therapeutics, Inc. | Adeno-associated virus vectors for treatment of Rett syndrome |
| US20240299586A1 (en) * | 2021-05-06 | 2024-09-12 | The Regents Of The University Of California | Novel aav serotypes derived from a library screen |
| WO2023196862A1 (en) | 2022-04-06 | 2023-10-12 | Genzyme Corporation | Targeted gene therapy for dm-1 myotonic dystrophy |
| US20240384271A1 (en) * | 2023-04-05 | 2024-11-21 | Genzyme Corporation | Targeted gene therapy for dm-1 myotonic dystrophy |
Family Cites Families (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5846782A (en) * | 1995-11-28 | 1998-12-08 | Genvec, Inc. | Targeting adenovirus with use of constrained peptide motifs |
| 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 |
| WO2012109570A1 (en) | 2011-02-10 | 2012-08-16 | The University Of North Carolina At Chapel Hill | Viral vectors with modified transduction profiles and methods of making and using the same |
| NZ701693A (en) * | 2012-04-18 | 2017-02-24 | The Children’S Hospital Of Philadelphia | Composition and methods for highly efficient gene transfer using aav capsid variants |
| IL297919A (en) * | 2013-07-22 | 2023-01-01 | Childrens Hospital Philadelphia | Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues |
| GB201403684D0 (en) * | 2014-03-03 | 2014-04-16 | King S College London | Vector |
| CN107073051B (zh) * | 2014-10-21 | 2021-08-24 | 马萨诸塞大学 | 重组aav变体及其用途 |
| ES2865487T3 (es) * | 2015-09-28 | 2021-10-15 | Univ North Carolina Chapel Hill | Métodos y composiciones para vectores virales que evaden los anticuerpos |
| ES2977730T3 (es) * | 2016-04-15 | 2024-08-29 | Res Inst Nationwide Childrens Hospital | Administración de microARN-29 y micro-distrofina por virus adenoasociado para tratar la distrofia muscular |
| US11364308B2 (en) * | 2016-05-13 | 2022-06-21 | 4D Molecular Therapeutics Inc. | Adeno-associated virus variant capsids and methods of use thereof |
| EP3765624A4 (en) * | 2018-03-16 | 2022-05-25 | Research Institute at Nationwide Children's Hospital | Increasing tissue specific gene delivery by capsid modification |
| BR112021003174A2 (pt) * | 2018-08-30 | 2021-05-11 | Research Institute At Nationwide Children's Hospital | terapia gênica para o tratamento de galactosemia |
-
2019
- 2019-03-14 EP EP19768552.2A patent/EP3765624A4/en active Pending
- 2019-03-14 US US16/980,821 patent/US12385062B2/en active Active
- 2019-03-14 CA CA3293599A patent/CA3293599A1/en active Pending
- 2019-03-14 KR KR1020257023347A patent/KR20250111395A/ko active Pending
- 2019-03-14 JP JP2020548674A patent/JP7304878B2/ja active Active
- 2019-03-14 CA CA3293598A patent/CA3293598A1/en active Pending
- 2019-03-14 WO PCT/US2019/022353 patent/WO2019178412A1/en not_active Ceased
- 2019-03-14 KR KR1020257023337A patent/KR20250111393A/ko active Pending
- 2019-03-14 CA CA3092353A patent/CA3092353A1/en active Pending
- 2019-03-14 IL IL317905A patent/IL317905A/en unknown
- 2019-03-14 BR BR112020018354-7A patent/BR112020018354A2/pt unknown
- 2019-03-14 CN CN201980018151.0A patent/CN111819286B/zh active Active
- 2019-03-14 CN CN202410503648.4A patent/CN118373887A/zh active Pending
- 2019-03-14 SG SG11202008687XA patent/SG11202008687XA/en unknown
- 2019-03-14 IL IL277270A patent/IL277270B2/en unknown
- 2019-03-14 KR KR1020207028750A patent/KR102834868B1/ko active Active
- 2019-03-14 AU AU2019234918A patent/AU2019234918C1/en active Active
-
2023
- 2023-06-27 JP JP2023104920A patent/JP2023115149A/ja active Pending
-
2024
- 2024-07-23 JP JP2024117744A patent/JP2024144521A/ja active Pending
-
2025
- 2025-06-09 AU AU2025204275A patent/AU2025204275A1/en active Pending
- 2025-07-23 US US19/277,998 patent/US20250346924A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| IL277270B1 (en) | 2025-01-01 |
| JP2023115149A (ja) | 2023-08-18 |
| US12385062B2 (en) | 2025-08-12 |
| KR20250111393A (ko) | 2025-07-22 |
| CN111819286A (zh) | 2020-10-23 |
| KR20200131843A (ko) | 2020-11-24 |
| IL277270B2 (en) | 2025-05-01 |
| EP3765624A4 (en) | 2022-05-25 |
| AU2025204275A1 (en) | 2025-07-03 |
| EP3765624A1 (en) | 2021-01-20 |
| BR112020018354A2 (pt) | 2020-12-29 |
| CN118373887A (zh) | 2024-07-23 |
| KR20250111395A (ko) | 2025-07-22 |
| CA3092353A1 (en) | 2019-09-19 |
| IL277270A (en) | 2020-10-29 |
| RU2020131998A (ru) | 2022-04-18 |
| JP2024144521A (ja) | 2024-10-11 |
| JP2021515572A (ja) | 2021-06-24 |
| JP7304878B2 (ja) | 2023-07-07 |
| SG11202008687XA (en) | 2020-10-29 |
| AU2019234918B2 (en) | 2025-08-14 |
| US20210087584A1 (en) | 2021-03-25 |
| AU2019234918A1 (en) | 2020-09-17 |
| CN111819286B (zh) | 2024-05-24 |
| AU2019234918C1 (en) | 2026-03-05 |
| CA3293598A1 (en) | 2026-03-02 |
| US20250346924A1 (en) | 2025-11-13 |
| KR102834868B1 (ko) | 2025-07-17 |
| CA3293599A1 (en) | 2026-03-02 |
| WO2019178412A1 (en) | 2019-09-19 |
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