KR20230004617A - 신경계 장애를 치료하기 위한 조성물 및 방법 - Google Patents

신경계 장애를 치료하기 위한 조성물 및 방법 Download PDF

Info

Publication number
KR20230004617A
KR20230004617A KR1020227039686A KR20227039686A KR20230004617A KR 20230004617 A KR20230004617 A KR 20230004617A KR 1020227039686 A KR1020227039686 A KR 1020227039686A KR 20227039686 A KR20227039686 A KR 20227039686A KR 20230004617 A KR20230004617 A KR 20230004617A
Authority
KR
South Korea
Prior art keywords
raav
nucleic acid
acid sequence
viral vector
sequence
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
KR1020227039686A
Other languages
English (en)
Korean (ko)
Inventor
레이첼 엠. 베일리
스티븐 제이. 그레이
Original Assignee
더 보드 오브 리젠츠 오브 더 유니버시티 오브 텍사스 시스템
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 더 보드 오브 리젠츠 오브 더 유니버시티 오브 텍사스 시스템 filed Critical 더 보드 오브 리젠츠 오브 더 유니버시티 오브 텍사스 시스템
Publication of KR20230004617A publication Critical patent/KR20230004617A/ko
Pending legal-status Critical Current

Links

Images

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
    • 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
    • A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C07K14/01—DNA viruses
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/08—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from viruses
    • C07K16/081—DNA viruses
    • 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
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/072—Animals genetically altered by homologous recombination maintaining or altering function, i.e. knock in
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/0306—Animal model for genetic diseases
    • A01K2267/0318—Animal model for neurodegenerative disease, e.g. non- Alzheimer's
    • 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)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Virology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Microbiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • General Chemical & Material Sciences (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
KR1020227039686A 2020-04-15 2021-04-14 신경계 장애를 치료하기 위한 조성물 및 방법 Pending KR20230004617A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063010179P 2020-04-15 2020-04-15
US63/010,179 2020-04-15
PCT/US2021/027266 WO2021211700A1 (en) 2020-04-15 2021-04-14 Compositions and methods for treatment of neurological disorders

Publications (1)

Publication Number Publication Date
KR20230004617A true KR20230004617A (ko) 2023-01-06

Family

ID=78081428

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020227039686A Pending KR20230004617A (ko) 2020-04-15 2021-04-14 신경계 장애를 치료하기 위한 조성물 및 방법

Country Status (9)

Country Link
US (2) US11753655B2 (https=)
EP (1) EP4135661A4 (https=)
JP (1) JP2023522883A (https=)
KR (1) KR20230004617A (https=)
CN (1) CN115715185A (https=)
AU (1) AU2021255934A1 (https=)
CA (1) CA3175406A1 (https=)
IL (1) IL296857A (https=)
WO (1) WO2021211700A1 (https=)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11753655B2 (en) * 2020-04-15 2023-09-12 The Board Of Regents Of The University Of Texas System Compositions and methods for treatment of neurological disorders
WO2023102518A1 (en) * 2021-12-03 2023-06-08 The Board Of Regents Of The University Of Texas System Gnao1 gene therapy vectors and uses thereof
EP4713462A1 (en) * 2023-05-18 2026-03-25 Gencorrex Therapies Inc. Solute carrier family 6 member 8 vectors and methods for use thereof

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8676330B2 (en) * 2009-03-20 2014-03-18 ElectroCore, LLC Electrical and magnetic stimulators used to treat migraine/sinus headache and comorbid disorders
US20070292922A1 (en) * 2006-03-31 2007-12-20 Cell Genesys, Inc. Regulated expression of recombinant proteins from adeno-associated viral vectors
EP2771367B1 (en) * 2011-10-25 2017-12-06 Indiana University Research and Technology Corporation Gigaxonin fusion protein and methods for treating giant axonal neuropathy
CN105745326A (zh) * 2013-10-24 2016-07-06 优尼科Ip有限公司 用于基因治疗神经疾病的aav-5假型载体
US10016514B2 (en) * 2015-05-15 2018-07-10 New Hope Research Foundation Polynucleotides, vectors and methods for insertion and expression of transgenes
AU2016324317A1 (en) * 2015-09-17 2018-03-08 Coda Biotherapeutics, Inc. Compositions and methods for treating neurological disorders
WO2018109207A1 (en) * 2016-12-16 2018-06-21 Uniqure Ip B.V. Immunoadsorption
IL280300B2 (en) 2018-08-10 2025-06-01 Univ North Carolina Chapel Hill Optimized cln7 genes and expression cassettes and their use
US11753655B2 (en) * 2020-04-15 2023-09-12 The Board Of Regents Of The University Of Texas System Compositions and methods for treatment of neurological disorders

Also Published As

Publication number Publication date
WO2021211700A1 (en) 2021-10-21
US12534741B2 (en) 2026-01-27
JP2023522883A (ja) 2023-06-01
CA3175406A1 (en) 2021-10-21
AU2021255934A1 (en) 2022-10-20
US20240102050A1 (en) 2024-03-28
CN115715185A (zh) 2023-02-24
EP4135661A4 (en) 2024-07-10
IL296857A (en) 2022-11-01
EP4135661A1 (en) 2023-02-22
US11753655B2 (en) 2023-09-12
US20210324411A1 (en) 2021-10-21

Similar Documents

Publication Publication Date Title
KR102834868B1 (ko) 캡시드 변형에 의한 증가하는 조직 특정 유전자 전달
JP7702949B2 (ja) ヒトｍｅｃｐ２遺伝子を発現するように設計されたトランスジーンカセット
SG11202111400TA (en) Compositions useful for treatment of pompe disease
US12534741B2 (en) Compositions and methods for treatment of neurological disorders
US12077772B2 (en) Transgene cassettes, AAV vectors and AAV viral vectors for the expression of human codon-optimized SLC6A1
US20250283111A1 (en) Recombinant adeno-associated viral vector for gene delivery
WO2022026516A1 (en) Transgene cassettes, aav vectors, and aav viral vectors for expression of human codon-optimized cstb
JP2024536223A (ja) Ｓｌｃ１３ａ５遺伝子療法ベクターおよびその使用
WO2023081683A1 (en) Gene therapy for prader willi syndrome
US20260022402A1 (en) Recombinant adeno-associated viral vector for gene delivery
WO2023102406A1 (en) Vector genome design to express optimized cln7 transgene
CN118019855A (zh) Slc13a5基因治疗载体及其用途
JP2024506860A (ja) ニーマンピック病ａ型を治療するための組成物及び方法
WO2023102518A1 (en) Gnao1 gene therapy vectors and uses thereof
WO2024163335A2 (en) Pten gene therapy vectors and uses thereof

Legal Events

Date Code Title Description
PA0105 International application

St.27 status event code: A-0-1-A10-A15-nap-PA0105

PG1501 Laying open of application

St.27 status event code: A-1-1-Q10-Q12-nap-PG1501

A201 Request for examination
E13-X000 Pre-grant limitation requested

St.27 status event code: A-2-3-E10-E13-lim-X000

P11-X000 Amendment of application requested

St.27 status event code: A-2-2-P10-P11-nap-X000

P13-X000 Application amended

St.27 status event code: A-2-2-P10-P13-nap-X000

PA0201 Request for examination

St.27 status event code: A-1-2-D10-D11-exm-PA0201

D21 Rejection of application intended

Free format text: ST27 STATUS EVENT CODE: A-1-2-D10-D21-EXM-PE0902 (AS PROVIDED BY THE NATIONAL OFFICE)

PE0902 Notice of grounds for rejection

St.27 status event code: A-1-2-D10-D21-exm-PE0902