IL315322A - Lyophilized formulations of aav drug products - Google Patents
Lyophilized formulations of aav drug productsInfo
- Publication number
- IL315322A IL315322A IL315322A IL31532224A IL315322A IL 315322 A IL315322 A IL 315322A IL 315322 A IL315322 A IL 315322A IL 31532224 A IL31532224 A IL 31532224A IL 315322 A IL315322 A IL 315322A
- Authority
- IL
- Israel
- Prior art keywords
- adeno
- frozen
- associated virus
- drug product
- product formulations
- Prior art date
Links
Classifications
-
- 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
- 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
- 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
- A61K47/26—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
- A61K48/0008—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
- A61K48/0025—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 wherein the non-active part clearly interacts with the delivered nucleic acid
- A61K48/0041—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 wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
-
- 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
- A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
- A61K9/16—Agglomerates; Granulates; Microbeadlets ; Microspheres; Pellets; Solid products obtained by spray drying, spray freeze drying, spray congealing,(multiple) emulsion solvent evaporation or extraction
- A61K9/1605—Excipients; Inactive ingredients
- A61K9/1617—Organic compounds, e.g. phospholipids, fats
-
- 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
- A61K9/16—Agglomerates; Granulates; Microbeadlets ; Microspheres; Pellets; Solid products obtained by spray drying, spray freeze drying, spray congealing,(multiple) emulsion solvent evaporation or extraction
- A61K9/1605—Excipients; Inactive ingredients
- A61K9/1617—Organic compounds, e.g. phospholipids, fats
- A61K9/1623—Sugars or sugar alcohols, e.g. lactose; Derivatives thereof; Homeopathic globules
-
- 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
- A61K9/16—Agglomerates; Granulates; Microbeadlets ; Microspheres; Pellets; Solid products obtained by spray drying, spray freeze drying, spray congealing,(multiple) emulsion solvent evaporation or extraction
- A61K9/1605—Excipients; Inactive ingredients
- A61K9/1629—Organic macromolecular compounds
- A61K9/1641—Organic macromolecular compounds obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyethylene glycol, poloxamers
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Epidemiology (AREA)
- Animal Behavior & Ethology (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Molecular Biology (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- General Chemical & Material Sciences (AREA)
- Oil, Petroleum & Natural Gas (AREA)
- Virology (AREA)
- Biotechnology (AREA)
- Genetics & Genomics (AREA)
- Microbiology (AREA)
- Mycology (AREA)
- Inorganic Chemistry (AREA)
- Dermatology (AREA)
- Medicinal Preparation (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202263319657P | 2022-03-14 | 2022-03-14 | |
| PCT/US2023/015112 WO2023177626A1 (en) | 2022-03-14 | 2023-03-13 | Lyophilized formulations of aav drug products |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| IL315322A true IL315322A (en) | 2024-10-01 |
Family
ID=85979703
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL315322A IL315322A (en) | 2022-03-14 | 2023-03-13 | Lyophilized formulations of aav drug products |
Country Status (11)
| Country | Link |
|---|---|
| US (1) | US20230293724A1 (https=) |
| EP (1) | EP4493162A1 (https=) |
| JP (1) | JP2025509566A (https=) |
| KR (1) | KR20250002229A (https=) |
| CN (1) | CN119072302A (https=) |
| AR (1) | AR128785A1 (https=) |
| AU (1) | AU2023233503A1 (https=) |
| CA (1) | CA3245651A1 (https=) |
| IL (1) | IL315322A (https=) |
| TW (1) | TW202406560A (https=) |
| WO (1) | WO2023177626A1 (https=) |
Families Citing this family (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| WO2025114524A1 (en) * | 2023-11-30 | 2025-06-05 | Uniqure Biopharma B.V. | Formulations for viral drug products |
| WO2025185606A1 (en) * | 2024-03-04 | 2025-09-12 | Nanjing Legend Biotech Co., Ltd. | Composition for cryoprotection of viral vector or vlp |
| WO2025188316A1 (en) * | 2024-03-08 | 2025-09-12 | Jaguar Gene Therapy, Llc | Aav pharmaceutical formulations and methods thereof |
Family Cites Families (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| BR112021009370A2 (pt) * | 2018-11-14 | 2021-08-17 | Regenxbio Inc. | método de tratamento da doença de batten cln2, composição farmacêutica e kit |
| SG11202107645RA (en) * | 2019-01-18 | 2021-08-30 | Voyager Therapeutics Inc | Methods and systems for producing aav particles |
| TW202332458A (zh) * | 2019-04-19 | 2023-08-16 | 美商銳進科斯生物股份有限公司 | 腺相關病毒載體調配物及方法 |
| WO2021071835A1 (en) * | 2019-10-07 | 2021-04-15 | Regenxbio Inc. | Adeno-associated virus vector pharmaceutical composition and methods |
| TW202144575A (zh) * | 2020-04-03 | 2021-12-01 | 美商拜奧馬林製藥公司 | 使用aav及治療調配物之苯酮尿症治療 |
-
2023
- 2023-03-13 WO PCT/US2023/015112 patent/WO2023177626A1/en not_active Ceased
- 2023-03-13 CA CA3245651A patent/CA3245651A1/en active Pending
- 2023-03-13 EP EP23716010.6A patent/EP4493162A1/en active Pending
- 2023-03-13 KR KR1020247034157A patent/KR20250002229A/ko active Pending
- 2023-03-13 US US18/120,762 patent/US20230293724A1/en active Pending
- 2023-03-13 IL IL315322A patent/IL315322A/en unknown
- 2023-03-13 TW TW112109096A patent/TW202406560A/zh unknown
- 2023-03-13 AU AU2023233503A patent/AU2023233503A1/en active Pending
- 2023-03-13 CN CN202380035210.1A patent/CN119072302A/zh active Pending
- 2023-03-13 JP JP2024554782A patent/JP2025509566A/ja active Pending
- 2023-03-14 AR ARP230100629A patent/AR128785A1/es unknown
Also Published As
| Publication number | Publication date |
|---|---|
| US20230293724A1 (en) | 2023-09-21 |
| TW202406560A (zh) | 2024-02-16 |
| JP2025509566A (ja) | 2025-04-11 |
| EP4493162A1 (en) | 2025-01-22 |
| AR128785A1 (es) | 2024-06-12 |
| AU2023233503A1 (en) | 2024-09-19 |
| KR20250002229A (ko) | 2025-01-07 |
| CA3245651A1 (en) | 2023-09-21 |
| WO2023177626A1 (en) | 2023-09-21 |
| CN119072302A (zh) | 2024-12-03 |
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