AR128785A1 - COMPOSICIONES LIOFILIZADAS QUE COMPRENDEN PARTICULAS DE VIRUS ADENOASOCIADO RECOMBINANTE (rAAV) - Google Patents

COMPOSICIONES LIOFILIZADAS QUE COMPRENDEN PARTICULAS DE VIRUS ADENOASOCIADO RECOMBINANTE (rAAV)

Info

Publication number
AR128785A1
AR128785A1 ARP230100629A ARP230100629A AR128785A1 AR 128785 A1 AR128785 A1 AR 128785A1 AR P230100629 A ARP230100629 A AR P230100629A AR P230100629 A ARP230100629 A AR P230100629A AR 128785 A1 AR128785 A1 AR 128785A1
Authority
AR
Argentina
Prior art keywords
raav
particles
concentration
associated virus
recombinant adeno
Prior art date
Application number
ARP230100629A
Other languages
English (en)
Spanish (es)
Inventor
Dingjiang Liu
Li Zhi
Yao Chen
Kuan Lai
- Wert Jonathan Yu
Xiaoyan Wang
Original Assignee
Regeneron Pharma
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Regeneron Pharma filed Critical Regeneron Pharma
Publication of AR128785A1 publication Critical patent/AR128785A1/es

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/14Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/19Particulate 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66Microorganisms or materials therefrom
    • A61K35/76Viruses; Subviral particles; Bacteriophages
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/16Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing nitrogen, e.g. nitro-, nitroso-, azo-compounds, nitriles, cyanates
    • A61K47/18Amines; Amides; Ureas; Quaternary ammonium compounds; Amino acids; Oligopeptides having up to five amino acids
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/26Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharides; Derivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/30Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
    • A61K47/34Macromolecular 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008Medicinal 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
    • A61K48/0025Medicinal 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
    • A61K48/0041Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/14Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/16Agglomerates; Granulates; Microbeadlets ; Microspheres; Pellets; Solid products obtained by spray drying, spray freeze drying, spray congealing,(multiple) emulsion solvent evaporation or extraction
    • A61K9/1605Excipients; Inactive ingredients
    • A61K9/1617Organic compounds, e.g. phospholipids, fats
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/14Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/16Agglomerates; Granulates; Microbeadlets ; Microspheres; Pellets; Solid products obtained by spray drying, spray freeze drying, spray congealing,(multiple) emulsion solvent evaporation or extraction
    • A61K9/1605Excipients; Inactive ingredients
    • A61K9/1617Organic compounds, e.g. phospholipids, fats
    • A61K9/1623Sugars or sugar alcohols, e.g. lactose; Derivatives thereof; Homeopathic globules
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/14Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/16Agglomerates; Granulates; Microbeadlets ; Microspheres; Pellets; Solid products obtained by spray drying, spray freeze drying, spray congealing,(multiple) emulsion solvent evaporation or extraction
    • A61K9/1605Excipients; Inactive ingredients
    • A61K9/1629Organic macromolecular compounds
    • A61K9/1641Organic 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)
ARP230100629A 2022-03-14 2023-03-14 COMPOSICIONES LIOFILIZADAS QUE COMPRENDEN PARTICULAS DE VIRUS ADENOASOCIADO RECOMBINANTE (rAAV) AR128785A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US202263319657P 2022-03-14 2022-03-14

Publications (1)

Publication Number Publication Date
AR128785A1 true AR128785A1 (es) 2024-06-12

Family

ID=85979703

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP230100629A AR128785A1 (es) 2022-03-14 2023-03-14 COMPOSICIONES LIOFILIZADAS QUE COMPRENDEN PARTICULAS DE VIRUS ADENOASOCIADO RECOMBINANTE (rAAV)

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)

* Cited by examiner, † Cited by third party
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)

* Cited by examiner, † Cited by third party
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

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
AU2023233503A1 (en) 2024-09-19
KR20250002229A (ko) 2025-01-07
CA3245651A1 (en) 2023-09-21
WO2023177626A1 (en) 2023-09-21
IL315322A (en) 2024-10-01
CN119072302A (zh) 2024-12-03

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