CA3137135A1 - Gene therapies for stargardt disease (abca4) - Google Patents

Gene therapies for stargardt disease (abca4) Download PDF

Info

Publication number
CA3137135A1
CA3137135A1 CA3137135A CA3137135A CA3137135A1 CA 3137135 A1 CA3137135 A1 CA 3137135A1 CA 3137135 A CA3137135 A CA 3137135A CA 3137135 A CA3137135 A CA 3137135A CA 3137135 A1 CA3137135 A1 CA 3137135A1
Authority
CA
Canada
Prior art keywords
nucleic acid
isolated nucleic
cell
vector
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
CA3137135A
Other languages
English (en)
French (fr)
Inventor
Hemant Khanna
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.)
University of Massachusetts Amherst
Original Assignee
University of Massachusetts Amherst
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 University of Massachusetts Amherst filed Critical University of Massachusetts Amherst
Publication of CA3137135A1 publication Critical patent/CA3137135A1/en
Pending legal-status Critical Current

Links

Classifications

    • 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
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • 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
    • 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)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Microbiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Cell Biology (AREA)
  • Toxicology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
CA3137135A 2019-04-19 2020-04-16 Gene therapies for stargardt disease (abca4) Pending CA3137135A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962836493P 2019-04-19 2019-04-19
US62/836,493 2019-04-19
PCT/US2020/028504 WO2020214809A2 (en) 2019-04-19 2020-04-16 Gene therapies for stargardt disease (abca4)

Publications (1)

Publication Number Publication Date
CA3137135A1 true CA3137135A1 (en) 2020-10-22

Family

ID=72838359

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3137135A Pending CA3137135A1 (en) 2019-04-19 2020-04-16 Gene therapies for stargardt disease (abca4)

Country Status (7)

Country Link
US (2) US12516097B2 (https=)
EP (1) EP3955973A4 (https=)
JP (1) JP2022529782A (https=)
CN (1) CN114126665B (https=)
AU (1) AU2020257392A1 (https=)
CA (1) CA3137135A1 (https=)
WO (1) WO2020214809A2 (https=)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3137135A1 (en) 2019-04-19 2020-10-22 University Of Massachusetts Gene therapies for stargardt disease (abca4)
GB2700048B (en) 2019-09-18 2026-03-04 Aldevron Llc synthetic dna vectors and method of use
CN116234917A (zh) 2020-07-27 2023-06-06 安杰瑞姆生物科学公司 Dna分子组合物及其制备方法和使用方法
KR20230161941A (ko) * 2021-01-22 2023-11-28 코넬 유니버시티 리포푸신 세포독성으로부터 보호하는 화합물의 식별을위한 조성물 및 방법
CA3211687A1 (en) * 2021-03-19 2022-09-22 Ozan ALKAN Non-viral dna vectors and uses thereof for expressing pfic therapeutics
EP4330409A4 (en) 2021-04-26 2025-04-09 University of Massachusetts GENE THERAPIES FOR STARGARDT'S DISEASE (ABCA4)
EP4334447A1 (en) * 2021-05-07 2024-03-13 UCL Business Ltd Abca4 genome editing
CN115074369B (zh) * 2022-06-24 2023-10-20 成都金唯科生物科技有限公司 表达全长abca4基因的腺相关病毒载体及应用

Family Cites Families (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5549910A (en) 1989-03-31 1996-08-27 The Regents Of The University Of California Preparation of liposome and lipid complex compositions
JP3218637B2 (ja) 1990-07-26 2001-10-15 大正製薬株式会社 安定なリポソーム水懸濁液
JP2958076B2 (ja) 1990-08-27 1999-10-06 株式会社ビタミン研究所 遺伝子導入用多重膜リポソーム及び遺伝子捕捉多重膜リポソーム製剤並びにその製法
US5399363A (en) 1991-01-25 1995-03-21 Eastman Kodak Company Surface modified anticancer nanoparticles
US5543158A (en) 1993-07-23 1996-08-06 Massachusetts Institute Of Technology Biodegradable injectable nanoparticles
US5741516A (en) 1994-06-20 1998-04-21 Inex Pharmaceuticals Corporation Sphingosomes for enhanced drug delivery
US5795587A (en) 1995-01-23 1998-08-18 University Of Pittsburgh Stable lipid-comprising drug delivery complexes and methods for their production
IE80468B1 (en) 1995-04-04 1998-07-29 Elan Corp Plc Controlled release biodegradable nanoparticles containing insulin
US5738868A (en) 1995-07-18 1998-04-14 Lipogenics Ltd. Liposome compositions and kits therefor
NZ532635A (en) 2001-11-13 2007-05-31 Univ Pennsylvania A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method
US7943374B2 (en) * 2005-08-21 2011-05-17 Markus Hildinger Super-size adeno-associated viral vector harboring a recombinant genome larger than 5.7 kb
US20090214478A1 (en) 2008-02-21 2009-08-27 Alberto Auricchio Method of treating ocular diseases by gene therapy
WO2009134418A2 (en) * 2008-04-30 2009-11-05 Fox Chase Cancer Center Assay for identifying agents that modulate epigenetic silencing, and agents identified thereby
KR102537394B1 (ko) * 2014-03-17 2023-05-30 애드베룸 바이오테크놀로지스, 인코포레이티드 원추세포에서 증강된 유전자 발현을 위한 조성물 및 방법
EP3377116A4 (en) * 2015-11-19 2019-07-10 The Trustees of The University of Pennsylvania COMPOSITIONS AND METHODS FOR CORRECTING ECG-RELATED EYE DISEASE
WO2018109011A1 (en) 2016-12-13 2018-06-21 Stichting Katholieke Universiteit Antisense oligonucleotides for the treatment of stargardt disease
AU2018223810B2 (en) 2017-02-27 2022-04-21 Case Western Reserve University Visual cycle modulators
CA3137135A1 (en) 2019-04-19 2020-10-22 University Of Massachusetts Gene therapies for stargardt disease (abca4)
EP4330409A4 (en) 2021-04-26 2025-04-09 University of Massachusetts GENE THERAPIES FOR STARGARDT'S DISEASE (ABCA4)

Also Published As

Publication number Publication date
CN114126665A (zh) 2022-03-01
WO2020214809A3 (en) 2020-11-19
WO2020214809A2 (en) 2020-10-22
US20220177539A1 (en) 2022-06-09
US20260022154A1 (en) 2026-01-22
EP3955973A2 (en) 2022-02-23
US12516097B2 (en) 2026-01-06
AU2020257392A1 (en) 2021-11-18
JP2022529782A (ja) 2022-06-24
CN114126665B (zh) 2025-04-25
EP3955973A4 (en) 2023-01-18

Similar Documents

Publication Publication Date Title
US20260022154A1 (en) Gene therapies for stargardt disease (abca4)
AU2023200880B2 (en) Minigene therapy
EP3972610B1 (en) Minigene therapy
US20220202959A1 (en) Gene therapies for usher syndrome (ush2a)
US12522845B2 (en) Aav vectors encoding NF1 and uses thereof
US20260091132A1 (en) Gene therapies for usher syndrome (ush1b)
US20240207450A1 (en) Gene therapies for stargardt disease (abca4)
WO2020210592A1 (en) Recombinant aav gene therapy for ngyl1 deficiency
HK40021856A (en) Minigene therapy
HK40021856B (en) Minigene therapy

Legal Events

Date Code Title Description
EEER Examination request

Effective date: 20220817

D15 Examination report completed

Free format text: ST27 STATUS EVENT CODE: A-2-2-D10-D15-D126 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: EXAMINER'S REPORT

Effective date: 20241025

B12 Application deemed to be withdrawn, abandoned or lapsed

Free format text: ST27 STATUS EVENT CODE: N-6-6-B10-B12-B303 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: DEEMED ABANDONED - FAILURE TO RESPOND TO AN EXAMINER'S REQUISITION

Effective date: 20250225

W00 Other event occurred

Free format text: ST27 STATUS EVENT CODE: A-2-2-W10-W00-W100 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: LETTER SENT

Effective date: 20251003

U13 Renewal or maintenance fee not paid

Free format text: ST27 STATUS EVENT CODE: N-2-6-U10-U13-U300 (AS PROVIDED BY THE NATIONAL OFFICE); EVENT TEXT: DEEMED ABANDONED - FAILURE TO RESPOND TO MAINTENANCE FEE NOTICE

Effective date: 20251203