AU2021319065A1 - Improved AAV-mediated X-linked retinoschisis therapies - Google Patents

Improved AAV-mediated X-linked retinoschisis therapies Download PDF

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Publication number
AU2021319065A1
AU2021319065A1 AU2021319065A AU2021319065A AU2021319065A1 AU 2021319065 A1 AU2021319065 A1 AU 2021319065A1 AU 2021319065 A AU2021319065 A AU 2021319065A AU 2021319065 A AU2021319065 A AU 2021319065A AU 2021319065 A1 AU2021319065 A1 AU 2021319065A1
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vector
nucleic acid
raav
sequence
seq
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AU2021319065A
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Sanford L. Boye
Shannon E. Boye
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University of Florida Research Foundation Inc
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University of Florida Research Foundation Inc
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    • 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
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    • A—HUMAN NECESSITIES
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    • 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
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • 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
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    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
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    • C12N2830/00—Vector systems having a special element relevant for transcription
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    • C12N2830/38—Vector systems having a special element relevant for transcription being a stuffer
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

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  • Health & Medical Sciences (AREA)
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  • Animal Behavior & Ethology (AREA)
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  • Virology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Ophthalmology & Optometry (AREA)
  • Epidemiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Microbiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Toxicology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Transition And Organic Metals Composition Catalysts For Addition Polymerization (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
AU2021319065A 2020-07-29 2021-07-28 Improved AAV-mediated X-linked retinoschisis therapies Pending AU2021319065A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063058437P 2020-07-29 2020-07-29
US63/058,437 2020-07-29
PCT/US2021/043582 WO2022026632A2 (en) 2020-07-29 2021-07-28 Improved aav-mediated x-linked retinoschisis therapies

Publications (1)

Publication Number Publication Date
AU2021319065A1 true AU2021319065A1 (en) 2023-02-16

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ID=77726514

Family Applications (1)

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AU2021319065A Pending AU2021319065A1 (en) 2020-07-29 2021-07-28 Improved AAV-mediated X-linked retinoschisis therapies

Country Status (8)

Country Link
US (1) US20230265455A1 (https=)
EP (1) EP4188450A2 (https=)
JP (1) JP2023535956A (https=)
CN (1) CN116568338A (https=)
AU (1) AU2021319065A1 (https=)
CA (1) CA3190214A1 (https=)
IL (1) IL299927A (https=)
WO (1) WO2022026632A2 (https=)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20240124893A1 (en) * 2021-11-03 2024-04-18 Teamedon International, Inc. Methods of Treating Human X-Linked Retinoschisis Using Gene Therapy
EP4499847A4 (en) * 2022-03-30 2026-03-25 Univ Pittsburgh Commonwealth Sys Higher Education Adeno-associated viral vectors for the delivery of nucleic acids across retinal regions
CN114848850B (zh) * 2022-04-28 2023-12-15 武汉中眸生物科技有限责任公司 Rs1基因在制备xlrs治疗剂中的应用及治疗剂
CN118359687A (zh) * 2023-01-18 2024-07-19 上海朗昇生物科技有限公司 一种新衣壳蛋白变体以及其用途
EP4704920A1 (en) * 2023-05-04 2026-03-11 Abeona Therapeutics Inc. Recombinant adeno-associated viral vectors for multipartite gene delivery and stargardt disease treatment
WO2025022144A1 (en) * 2023-07-26 2025-01-30 Sorbonne Universite Chimeric promoter for targeted expression in aii amacrine cells
WO2025091025A1 (en) * 2023-10-26 2025-05-01 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders relating to sirt1
WO2025113432A1 (zh) * 2023-11-27 2025-06-05 北京因诺惟康医药科技有限公司 一种用于治疗x染色体连锁视网膜劈裂症的基因药物
WO2025231339A1 (en) * 2024-05-02 2025-11-06 Avista Therapeutics, Inc. Adeno-associated virus vectors for therapeutic nucleic acid delivery to retinal cells

Family Cites Families (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2003274397A1 (en) 2002-06-05 2003-12-22 University Of Florida Production of pseudotyped recombinant aav virions
US20120322861A1 (en) 2007-02-23 2012-12-20 Barry John Byrne Compositions and Methods for Treating Diseases
US9567376B2 (en) * 2013-02-08 2017-02-14 The Trustees Of The University Of Pennsylvania Enhanced AAV-mediated gene transfer for retinal therapies
WO2014127196A1 (en) * 2013-02-15 2014-08-21 The United States Of America, As Represented By The Secretary, Dept. Of Health And Human Services Aav8 retinoschisin expression vector for treating x-linked retinoschisis
HK1220488A1 (zh) * 2013-03-15 2017-05-05 The Children's Hospital Of Philadelphia 含有填充者/填充物多核苷酸序列的载体及其使用方法
EP3113787B1 (en) * 2014-03-04 2019-12-04 University of Florida Research Foundation, Inc. Improved raav vectors and methods for transduction of photoreceptors and rpe cells
EP3800191B1 (en) * 2014-03-17 2025-08-20 Adverum Biotechnologies, Inc. Compositions and methods for enhanced gene expression in cone cells
PL3628334T3 (pl) * 2014-03-21 2023-12-18 Genzyme Corporation Terapia genowa w retinopatii barwnikowej
US10533187B2 (en) 2015-03-18 2020-01-14 University Of Florida Research Foundation, Incorporated Methods and compositions for restoration of cone function in BCM
CA3001594A1 (en) * 2015-10-14 2017-04-20 Audentes Therapeutics, Inc. Nucleic acid molecules containing spacers and methods of use thereof
WO2017070491A1 (en) * 2015-10-23 2017-04-27 Applied Genetic Technologies Corporation Ophthalmic formulations
EP3585883A4 (en) 2017-02-21 2021-04-14 University of Florida Research Foundation, Incorporated MODIFIED AAV CAPSID PROTEINS AND USES THEREOF
US20210228738A1 (en) * 2017-07-17 2021-07-29 INSERM (Institut National de la Santé et de la Recherche Médicale) Compositions and methods for increasing or enhancing transduction of gene therapy vectors and for removing or reducing immunoglobulins
US20200390907A1 (en) * 2018-01-22 2020-12-17 The Usa, As Represented By The Secretary, Department Of Health And Human Services Methods and compositions for treating genetically linked diseases of the eye
US12605466B2 (en) * 2019-03-04 2026-04-21 University Of Florida Research Foundation, Incorporated Enhanced human opsin promoter for rod specific expression
US20220403417A1 (en) * 2019-11-20 2022-12-22 University Of Massachusetts Aav-based delivery of thymine kinase 2

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EP4188450A2 (en) 2023-06-07
WO2022026632A3 (en) 2022-03-10
CA3190214A1 (en) 2022-02-03
JP2023535956A (ja) 2023-08-22
US20230265455A1 (en) 2023-08-24
WO2022026632A2 (en) 2022-02-03
IL299927A (en) 2023-03-01
CN116568338A (zh) 2023-08-08

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