CN116323925A - 高度纯化的病毒颗粒的制剂 - Google Patents
高度纯化的病毒颗粒的制剂 Download PDFInfo
- Publication number
- CN116323925A CN116323925A CN202180067655.9A CN202180067655A CN116323925A CN 116323925 A CN116323925 A CN 116323925A CN 202180067655 A CN202180067655 A CN 202180067655A CN 116323925 A CN116323925 A CN 116323925A
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- China
- Prior art keywords
- pharmaceutical composition
- aav particles
- buffer
- purified
- aav
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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
- A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
- A61K9/19—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles lyophilised, i.e. freeze-dried, solutions or dispersions
-
- 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
- A61K47/16—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing nitrogen, e.g. nitro-, nitroso-, azo-compounds, nitriles, cyanates
- A61K47/18—Amines; Amides; Ureas; Quaternary ammonium compounds; Amino acids; Oligopeptides having up to five amino acids
-
- 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
- A61K47/16—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing nitrogen, e.g. nitro-, nitroso-, azo-compounds, nitriles, cyanates
- A61K47/18—Amines; Amides; Ureas; Quaternary ammonium compounds; Amino acids; Oligopeptides having up to five amino acids
- A61K47/183—Amino acids, e.g. glycine, EDTA or aspartame
-
- 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
- A61K47/22—Heterocyclic compounds, e.g. ascorbic acid, tocopherol or pyrrolidones
-
- 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
- A61K47/24—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing atoms other than carbon, hydrogen, oxygen, halogen, nitrogen or sulfur, e.g. cyclomethicone or phospholipids
-
- 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
- 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
- A61K47/30—Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
- A61K47/34—Macromolecular compounds obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyesters, polyamino acids, polysiloxanes, polyphosphazines, copolymers of polyalkylene glycol or poloxamers
-
- 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
- A61K48/0091—Purification or manufacturing processes for gene therapy compositions
-
- 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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- 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
-
- 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
-
- 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
- C12N2750/14123—Virus like particles [VLP]
-
- 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
- C12N2750/14151—Methods of production or purification of viral material
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- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Genetics & Genomics (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Oil, Petroleum & Natural Gas (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- General Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- Inorganic Chemistry (AREA)
- Virology (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Immunology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Manufacturing & Machinery (AREA)
- Dermatology (AREA)
- Medicinal Preparation (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063063128P | 2020-08-07 | 2020-08-07 | |
| US202063063108P | 2020-08-07 | 2020-08-07 | |
| US63/063128 | 2020-08-07 | ||
| US63/063108 | 2020-08-07 | ||
| PCT/US2021/044955 WO2022032104A1 (en) | 2020-08-07 | 2021-08-06 | Formulations for highly purified viral particles |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CN116323925A true CN116323925A (zh) | 2023-06-23 |
Family
ID=80114720
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN202180067655.9A Pending CN116323925A (zh) | 2020-08-07 | 2021-08-06 | 高度纯化的病毒颗粒的制剂 |
Country Status (10)
| Country | Link |
|---|---|
| US (2) | US20220040305A1 (https=) |
| EP (1) | EP4192437A4 (https=) |
| JP (1) | JP2023536883A (https=) |
| KR (1) | KR20230049670A (https=) |
| CN (1) | CN116323925A (https=) |
| AU (1) | AU2021320395A1 (https=) |
| CA (1) | CA3190596A1 (https=) |
| IL (1) | IL300323A (https=) |
| MX (1) | MX2023001411A (https=) |
| WO (1) | WO2022032104A1 (https=) |
Cited By (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN119015447A (zh) * | 2023-09-01 | 2024-11-26 | 康霖生物科技(杭州)有限公司 | 一种缓冲系统及其相应液体制剂及其用途 |
Families Citing this family (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20230212528A1 (en) * | 2020-06-02 | 2023-07-06 | Janssen Biotech, Inc. | Materials and methods for viral purification |
| WO2023028510A1 (en) * | 2021-08-24 | 2023-03-02 | Homology Medicines, Inc. | Adeno-associated virus formulations |
| CN120603600A (zh) * | 2023-01-19 | 2025-09-05 | 优尼科生物制药有限公司 | 基因递送媒介物的药物配制品 |
| WO2024252024A1 (en) * | 2023-06-09 | 2024-12-12 | Sartorius Bia Separations D.O.O. | A method of enhanced separation of full adeno-associated virus (aav) capsids |
| WO2025114524A1 (en) * | 2023-11-30 | 2025-06-05 | Uniqure Biopharma B.V. | Formulations for viral drug products |
| CN118957152B (zh) * | 2024-08-30 | 2025-11-25 | 浙江恒驭生物科技有限公司 | 一种腺相关病毒载体基因组滴度检测方法 |
Family Cites Families (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2420247A1 (en) * | 2000-03-07 | 2012-02-22 | Merck Sharp & Dohme Corp. | Adenovirus formulations |
| EP1751275B1 (en) * | 2004-06-01 | 2017-08-16 | Avigen, Inc. | Compositions and methods to prevent aav vector aggregation |
| GB201513010D0 (en) * | 2015-07-23 | 2015-09-09 | Glaxosmithkline Biolog Sa | Novel formulation |
| EA201890543A1 (ru) * | 2015-09-17 | 2018-08-31 | Рисёрч Инститьют Эт Нэйшнвайд Чилдрен'С Хоспитэл | Способы и материалы для генной терапии galgt2 |
| WO2019241535A2 (en) * | 2018-06-14 | 2019-12-19 | Regenxbio Inc. | Anion exchange chromatography for recombinant aav production |
| AU2019310459A1 (en) * | 2018-07-24 | 2021-02-18 | Voyager Therapeutics, Inc. | Systems and methods for producing gene therapy formulations |
| US20220143115A1 (en) * | 2019-04-19 | 2022-05-12 | Regenxbio Inc. | Adeno-Associated Virus Vector Formulations and Methods |
-
2021
- 2021-08-06 AU AU2021320395A patent/AU2021320395A1/en not_active Abandoned
- 2021-08-06 KR KR1020237007796A patent/KR20230049670A/ko not_active Withdrawn
- 2021-08-06 CA CA3190596A patent/CA3190596A1/en active Pending
- 2021-08-06 EP EP21853878.3A patent/EP4192437A4/en active Pending
- 2021-08-06 JP JP2023507428A patent/JP2023536883A/ja active Pending
- 2021-08-06 US US17/396,090 patent/US20220040305A1/en not_active Abandoned
- 2021-08-06 CN CN202180067655.9A patent/CN116323925A/zh active Pending
- 2021-08-06 IL IL300323A patent/IL300323A/en unknown
- 2021-08-06 MX MX2023001411A patent/MX2023001411A/es unknown
- 2021-08-06 WO PCT/US2021/044955 patent/WO2022032104A1/en not_active Ceased
-
2025
- 2025-11-04 US US19/379,616 patent/US20260061059A1/en active Pending
Cited By (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN119015447A (zh) * | 2023-09-01 | 2024-11-26 | 康霖生物科技(杭州)有限公司 | 一种缓冲系统及其相应液体制剂及其用途 |
Also Published As
| Publication number | Publication date |
|---|---|
| US20220040305A1 (en) | 2022-02-10 |
| MX2023001411A (es) | 2023-05-15 |
| EP4192437A4 (en) | 2024-09-25 |
| KR20230049670A (ko) | 2023-04-13 |
| AU2021320395A1 (en) | 2023-04-13 |
| JP2023536883A (ja) | 2023-08-30 |
| US20260061059A1 (en) | 2026-03-05 |
| WO2022032104A1 (en) | 2022-02-10 |
| EP4192437A1 (en) | 2023-06-14 |
| IL300323A (en) | 2023-04-01 |
| CA3190596A1 (en) | 2022-02-10 |
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| PB01 | Publication | ||
| PB01 | Publication | ||
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| SE01 | Entry into force of request for substantive examination |