IL303737A - Improved pharmaceutical compositions containing adeno-associated viral vector - Google Patents
Improved pharmaceutical compositions containing adeno-associated viral vectorInfo
- Publication number
- IL303737A IL303737A IL303737A IL30373723A IL303737A IL 303737 A IL303737 A IL 303737A IL 303737 A IL303737 A IL 303737A IL 30373723 A IL30373723 A IL 30373723A IL 303737 A IL303737 A IL 303737A
- Authority
- IL
- Israel
- Prior art keywords
- composition
- optionally
- formulation
- chloride
- sucrose
- Prior art date
Links
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- 229960002675 xylitol Drugs 0.000 description 1
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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
- A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
-
- 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
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/02—Inorganic compounds
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
- A61K47/08—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
- A61K47/10—Alcohols; Phenols; Salts thereof, e.g. glycerol; Polyethylene glycols [PEG]; Poloxamers; PEG/POE alkyl ethers
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
- A61K47/26—Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharides; Derivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
-
- 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
-
- 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
- A61K9/08—Solutions
-
- 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
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Epidemiology (AREA)
- Genetics & Genomics (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Zoology (AREA)
- Oil, Petroleum & Natural Gas (AREA)
- Biomedical Technology (AREA)
- Biochemistry (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Hematology (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Inorganic Chemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Diabetes (AREA)
- Dermatology (AREA)
- Gastroenterology & Hepatology (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063127826P | 2020-12-18 | 2020-12-18 | |
| PCT/US2021/064222 WO2022133324A1 (en) | 2020-12-18 | 2021-12-17 | Improved pharmaceutical compositions containing adeno-associated viral vector |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| IL303737A true IL303737A (en) | 2023-08-01 |
Family
ID=79686851
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL303737A IL303737A (en) | 2020-12-18 | 2021-12-17 | Improved pharmaceutical compositions containing adeno-associated viral vector |
Country Status (10)
| Country | Link |
|---|---|
| US (1) | US20240066146A1 (https=) |
| EP (1) | EP4262754A1 (https=) |
| JP (1) | JP2024500133A (https=) |
| KR (1) | KR20230122076A (https=) |
| CN (1) | CN116710149A (https=) |
| AU (1) | AU2021403118A1 (https=) |
| CA (1) | CA3204462A1 (https=) |
| IL (1) | IL303737A (https=) |
| MX (1) | MX2023006471A (https=) |
| WO (1) | WO2022133324A1 (https=) |
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| JP2024505060A (ja) * | 2021-01-27 | 2024-02-02 | エイラ・セラピューティクス・インコーポレイテッド | Ｐｎｍａ２ベースのキャプシド及びその使用 |
| US20240307553A1 (en) * | 2021-04-01 | 2024-09-19 | Pfizer Inc. | Pharmaceutical compositions containing adeno-associated viral vector |
| CN116350801A (zh) * | 2022-11-22 | 2023-06-30 | 四川至善唯新生物科技有限公司 | 一种重组腺相关病毒载体的药物组合物及其用途 |
| EP4651883A1 (en) * | 2023-01-19 | 2025-11-26 | uniQure biopharma B.V. | Pharmaceutical formulations of gene delivery vehicles |
| WO2025045083A1 (zh) * | 2023-09-01 | 2025-03-06 | 康霖生物科技（杭州）有限公司 | 一种液体制剂及其用途 |
| WO2025076729A1 (zh) * | 2023-10-11 | 2025-04-17 | 朗信启昇(苏州)生物制药有限公司 | 包含aav的药物组合物及其制备方法 |
| WO2025108407A2 (en) * | 2023-11-23 | 2025-05-30 | Neuexcell Therapeutics (Suzhou) Co., Ltd. | Gene therapy compositions and methods for treating glioma |
| WO2025113676A1 (en) * | 2023-11-29 | 2025-06-05 | Neuexcell Therapeutics (Suzhou) Co., Ltd. | Compositions and methods for treating stroke in primates |
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| ATE226449T1 (de) * | 1998-12-03 | 2002-11-15 | Avigen Inc | Hilfsstoffe zur verwendung in adeno-assoziiertes virus enthaltenden pharmazeutischen zubereitungen und damit hergestellte pharmazeutische zubereitungen |
| GB0510057D0 (en) * | 2005-05-17 | 2005-06-22 | Glaxosmithkline Biolog Sa | Novel device |
| ME03008B (me) * | 2011-09-19 | 2018-10-20 | Axon Neuroscience Se | Terapija na osnovu proteina i dijagnoza patologije u којој posreduje tau u alcнajмerovoj bolesti |
| EP3044231B1 (en) * | 2013-09-12 | 2020-08-05 | BioMarin Pharmaceutical Inc. | Aav vectors comprising a gene encoding factor viii |
| MY189674A (en) | 2015-10-28 | 2022-02-24 | Sangamo Therapeutics Inc | Liver-specific constructs, factor viii expression cassettes and methods of use thereof |
| CN112770812A (zh) * | 2018-07-24 | 2021-05-07 | 沃雅戈治疗公司 | 产生基因治疗制剂的系统和方法 |
| US12497632B2 (en) * | 2018-08-03 | 2025-12-16 | Sangamo Therapeutics, Inc. | Clinical parameters by expression of factor VIII |
| US12478553B2 (en) * | 2018-11-13 | 2025-11-25 | Sio2 Medical Products, Llc | Polymer vials with substantially flat bottoms and injection stretch blow molding methods for making the same |
| BR112021009370A2 (pt) * | 2018-11-14 | 2021-08-17 | Regenxbio Inc. | método de tratamento da doença de batten cln2, composição farmacêutica e kit |
| AU2019428629A1 (en) | 2019-02-06 | 2021-01-28 | Sangamo Therapeutics, Inc. | Method for the treatment of mucopolysaccharidosis type I |
| WO2021071835A1 (en) * | 2019-10-07 | 2021-04-15 | Regenxbio Inc. | Adeno-associated virus vector pharmaceutical composition and methods |
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2021
- 2021-12-17 US US18/258,027 patent/US20240066146A1/en active Pending
- 2021-12-17 WO PCT/US2021/064222 patent/WO2022133324A1/en not_active Ceased
- 2021-12-17 IL IL303737A patent/IL303737A/en unknown
- 2021-12-17 AU AU2021403118A patent/AU2021403118A1/en active Pending
- 2021-12-17 KR KR1020237023859A patent/KR20230122076A/ko active Pending
- 2021-12-17 CN CN202180085158.1A patent/CN116710149A/zh active Pending
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- 2021-12-17 MX MX2023006471A patent/MX2023006471A/es unknown
- 2021-12-17 JP JP2023537242A patent/JP2024500133A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| MX2023006471A (es) | 2023-08-11 |
| AU2021403118A9 (en) | 2024-04-18 |
| KR20230122076A (ko) | 2023-08-22 |
| CA3204462A1 (en) | 2022-06-23 |
| WO2022133324A1 (en) | 2022-06-23 |
| CN116710149A (zh) | 2023-09-05 |
| AU2021403118A1 (en) | 2023-06-22 |
| US20240066146A1 (en) | 2024-02-29 |
| EP4262754A1 (en) | 2023-10-25 |
| JP2024500133A (ja) | 2024-01-04 |
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