CN119256090A - 用于治疗帕金森病的方法和组合物 - Google Patents

用于治疗帕金森病的方法和组合物 Download PDF

Info

Publication number
CN119256090A
CN119256090A CN202380042769.7A CN202380042769A CN119256090A CN 119256090 A CN119256090 A CN 119256090A CN 202380042769 A CN202380042769 A CN 202380042769A CN 119256090 A CN119256090 A CN 119256090A
Authority
CN
China
Prior art keywords
subject
months
raav
introduction
mds
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
CN202380042769.7A
Other languages
English (en)
Chinese (zh)
Inventor
克里斯托夫·S·班基维茨
阿德里安·P·凯尔斯
安珀·范·拉尔
马西莫·S·菲安达卡
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
AskBio Inc
Original Assignee
Asklepios Biopharmaceutical Inc
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 Asklepios Biopharmaceutical Inc filed Critical Asklepios Biopharmaceutical Inc
Publication of CN119256090A publication Critical patent/CN119256090A/zh
Pending legal-status Critical Current

Links

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
    • A61K48/005—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
    • 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
    • A61K38/18—Growth factors; Growth regulators
    • A61K38/185—Nerve growth factor [NGF]; Brain derived neurotrophic factor [BDNF]; Ciliary neurotrophic factor [CNTF]; Glial derived neurotrophic factor [GDNF]; Neurotrophins, e.g. NT-3
    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
    • C07K14/4705—Regulators; Modulating activity stimulating, promoting or activating activity
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/475—Growth factors; Growth regulators
    • 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)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biophysics (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Psychology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Toxicology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Immunology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
CN202380042769.7A 2022-03-25 2023-03-24 用于治疗帕金森病的方法和组合物 Pending CN119256090A (zh)

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US202263323830P 2022-03-25 2022-03-25
US63/323,830 2022-03-25
US202263326236P 2022-03-31 2022-03-31
US63/326,236 2022-03-31
US202263341841P 2022-05-13 2022-05-13
US63/341,841 2022-05-13
US202263393196P 2022-07-28 2022-07-28
US63/393,196 2022-07-28
US202363438164P 2023-01-10 2023-01-10
US63/438,164 2023-01-10
PCT/US2023/016270 WO2023183594A2 (en) 2022-03-25 2023-03-24 Methods and compositions for the treatment of parkinson's disease

Publications (1)

Publication Number Publication Date
CN119256090A true CN119256090A (zh) 2025-01-03

Family

ID=88102133

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202380042769.7A Pending CN119256090A (zh) 2022-03-25 2023-03-24 用于治疗帕金森病的方法和组合物

Country Status (10)

Country Link
US (1) US20250222072A1 (https=)
EP (1) EP4499155A4 (https=)
JP (1) JP2025510150A (https=)
KR (1) KR20250006358A (https=)
CN (1) CN119256090A (https=)
AU (1) AU2023240256A1 (https=)
CA (1) CA3255025A1 (https=)
IL (1) IL315725A (https=)
MX (1) MX2024011436A (https=)
WO (1) WO2023183594A2 (https=)

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN107530447A (zh) * 2015-02-10 2018-01-02 建新公司 病毒颗粒至纹状体和皮质的增强递送
GB202013940D0 (en) * 2020-09-04 2020-10-21 Synpromics Ltd Regulatory nucleic acid sequences

Also Published As

Publication number Publication date
JP2025510150A (ja) 2025-04-14
EP4499155A4 (en) 2026-04-01
AU2023240256A1 (en) 2024-11-07
IL315725A (en) 2024-11-01
WO2023183594A2 (en) 2023-09-28
CA3255025A1 (en) 2023-09-28
EP4499155A2 (en) 2025-02-05
MX2024011436A (es) 2024-11-08
WO2023183594A3 (en) 2024-01-18
US20250222072A1 (en) 2025-07-10
KR20250006358A (ko) 2025-01-10

Similar Documents

Publication Publication Date Title
JP7832294B2 (ja) 網膜およびｃｎｓ遺伝子治療用ａａｖベクター
US11759506B2 (en) AADC polynucleotides for the treatment of Parkinson's disease
US20260078407A1 (en) RAAV Vectors for the Treatment of GM1 and GM2 Gangliosidosis
JP2023058630A (ja) 線条体および皮質へのウイルス粒子の強化された送達
US12319929B2 (en) Compositions and methods for the treatment of Parkinson's disease
JP2023542211A (ja) 神経疾患を処置するための方法
JP2024539123A (ja) アデノ随伴ウイルス（ａａｖ）送達を改善する方法
CN119256090A (zh) 用于治疗帕金森病的方法和组合物
HK40120718A (zh) 用於治疗帕金森病的方法和组合物
Sanchez et al. Recombinant adeno-associated virus type 2 pseudotypes: comparing safety, specificity, and transduction efficiency in the primate striatum
CN116723868A (zh) 治疗神经系统疾病的方法
US20250381307A1 (en) Retro-aav and use in treating neurodegenerative diseases
US20250382588A1 (en) Chimeric aav and uses thereof
Fehrman Feasibility of Large Transgene Delivery to the Mouse Retina: Eyeing the Potential for Gene Therapy Applications
KR20230023637A (ko) 크라베병의 치료에 유용한 조성물
TW202608488A (zh) 基因治療
TW202111126A (zh) 用於治療聖菲利柏氏病及其他病症之組合物及方法
NZ735290B2 (en) Enhanced delivery of viral particles to the striatum and cortex

Legal Events

Date Code Title Description
PB01 Publication
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination
CB02 Change of applicant information
CB02 Change of applicant information

Country or region after: U.S.A.

Address after: Delaware, USA

Applicant after: Ask Biotech Co.,Ltd.

Address before: North Carolina, USA

Applicant before: ASKLEPIOS BIOPHARMACEUTICAL, Inc.

Country or region before: U.S.A.

REG Reference to a national code

Ref country code: HK

Ref legal event code: DE

Ref document number: 40120718

Country of ref document: HK