CA3255025A1 - METHODS AND COMPOSITIONS INTENDED FOR THE TREATMENT OF PARKINSON'S DISEASE - Google Patents

METHODS AND COMPOSITIONS INTENDED FOR THE TREATMENT OF PARKINSON'S DISEASE

Info

Publication number
CA3255025A1
CA3255025A1 CA3255025A CA3255025A CA3255025A1 CA 3255025 A1 CA3255025 A1 CA 3255025A1 CA 3255025 A CA3255025 A CA 3255025A CA 3255025 A CA3255025 A CA 3255025A CA 3255025 A1 CA3255025 A1 CA 3255025A1
Authority
CA
Canada
Prior art keywords
subject
points
months
see seq
raav
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
CA3255025A
Other languages
English (en)
French (fr)
Inventor
Krystof S. Bankiewicz
Adrian P. Kells
Amber VAN LAAR
Massimo S. FIANDACA
Original Assignee
AskBio 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 AskBio Inc filed Critical AskBio Inc
Publication of CA3255025A1 publication Critical patent/CA3255025A1/en
Pending legal-status Critical Current

Links

Classifications

    • 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
    • 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)
CA3255025A 2022-03-25 2023-03-24 METHODS AND COMPOSITIONS INTENDED FOR THE TREATMENT OF PARKINSON'S DISEASE Pending CA3255025A1 (en)

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
CA3255025A1 true CA3255025A1 (en) 2023-09-28

Family

ID=88102133

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3255025A Pending CA3255025A1 (en) 2022-03-25 2023-03-24 METHODS AND COMPOSITIONS INTENDED FOR THE TREATMENT OF PARKINSON'S DISEASE

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
EP4499155A2 (en) 2025-02-05
MX2024011436A (es) 2024-11-08
WO2023183594A3 (en) 2024-01-18
CN119256090A (zh) 2025-01-03
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
US12491235B2 (en) Methods and compositions for treating metabolic imbalance in neurodegenerative disease
US11759506B2 (en) AADC polynucleotides for the treatment of Parkinson's disease
US20260078407A1 (en) RAAV Vectors for the Treatment of GM1 and GM2 Gangliosidosis
TWI730952B (zh) 病毒顆粒至紋狀體和皮質的增強遞送
US12319929B2 (en) Compositions and methods for the treatment of Parkinson's disease
JP2015521612A (ja) 遺伝子治療ベクターの広範な遺伝子送達
JP2012516357A (ja) 神経学的障害を治療するための皮質全体に亘る高レベルの治療薬の分布方法
WO2021007382A1 (en) Compositions and methods for the treatment of parkinson's disease
JP2024539123A (ja) アデノ随伴ウイルス（ａａｖ）送達を改善する方法
CN115052617A (zh) 一种包括tmem176b、其表达或活性调节剂作为活性成分的用于预防或治疗退行性脑疾病的组合物
US20250222072A1 (en) Methods and compositions for the treatment of parkinson's disease
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
Staneka et al. Gene therapy of neurological diseases
TW202111126A (zh) 用於治療聖菲利柏氏病及其他病症之組合物及方法
Murlidharan Biology of AAV vectors in the central nervous system
NZ735290B2 (en) Enhanced delivery of viral particles to the striatum and cortex

Legal Events

Date Code Title Description
A00 Application filed

Free format text: ST27 STATUS EVENT CODE: A-0-1-A10-A00-A101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: APPLICATION RECEIVED - PCT

Effective date: 20240920

MFA Maintenance fee for application paid

Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 2ND ANNIV.) - STANDARD

Year of fee payment: 2

A00 Application filed

Free format text: ST27 STATUS EVENT CODE: A-1-1-A10-A00-A102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: COMPLIANCE REQUIREMENTS DETERMINED MET

Effective date: 20250123

A15 Pct application entered into the national or regional phase

Free format text: ST27 STATUS EVENT CODE: A-1-1-A10-A15-X000 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: NATIONAL ENTRY REQUIREMENTS DETERMINED COMPLIANT

Effective date: 20250123

P18 Priority claim added or amended

Free format text: ST27 STATUS EVENT CODE: A-1-1-P10-P18-P105 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: PRIORITY CLAIM REQUIREMENTS DETERMINED COMPLIANT

Effective date: 20250123

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-1-1-W10-W00-W100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: LETTER SENT

Effective date: 20250131

R11 Change to the name of applicant or owner or transfer of ownership requested

Free format text: ST27 STATUS EVENT CODE: A-1-1-R10-R11-R103 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: CHANGE OF NAME REQUEST RECEIVED

Effective date: 20250224

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-1-1-W10-W00-W111 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: CORRESPONDENT DETERMINED COMPLIANT

Effective date: 20250224

P13 Application amended

Free format text: ST27 STATUS EVENT CODE: A-1-1-P10-P13-P146 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: BSL VERIFIED - NO DEFECTS

Effective date: 20250311

R13 Change to the name of applicant or owner recorded

Free format text: ST27 STATUS EVENT CODE: A-1-1-R10-R13-R104 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: CHANGE OF NAME REQUIREMENTS DETERMINED COMPLIANT

Effective date: 20250328

MFA Maintenance fee for application paid

Free format text: FEE DESCRIPTION TEXT: MF (APPLICATION, 3RD ANNIV.) - STANDARD

Year of fee payment: 3

U00 Fee paid

Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U00-U101 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE REQUEST RECEIVED

Effective date: 20260217

U11 Full renewal or maintenance fee paid

Free format text: ST27 STATUS EVENT CODE: A-1-1-U10-U11-U102 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: MAINTENANCE FEE PAYMENT PAID IN FULL

Effective date: 20260217