EP4704877A2 - Modulation optogénétique par opsines à caractéristiques multiples pour la restauration de la vision et d'autres applications associées - Google Patents

Modulation optogénétique par opsines à caractéristiques multiples pour la restauration de la vision et d'autres applications associées

Info

Publication number
EP4704877A2
EP4704877A2 EP24797864.6A EP24797864A EP4704877A2 EP 4704877 A2 EP4704877 A2 EP 4704877A2 EP 24797864 A EP24797864 A EP 24797864A EP 4704877 A2 EP4704877 A2 EP 4704877A2
Authority
EP
European Patent Office
Prior art keywords
emco1
light
retinal
mice
patient
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
EP24797864.6A
Other languages
German (de)
English (en)
Inventor
Samarendra Kumar Mohanty
Sulagna BHATTACHARYA
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.)
Nanoscope Therapeutics Inc
Original Assignee
Nanoscope Therapeutics 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 Nanoscope Therapeutics Inc filed Critical Nanoscope Therapeutics Inc
Publication of EP4704877A2 publication Critical patent/EP4704877A2/fr
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
    • 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
    • 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)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Organic Chemistry (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
  • Peptides Or Proteins (AREA)

Abstract

Selon un aspect, la présente invention concerne de manière générale des compositions et des procédés de modulation d'activités cellulaires par opsines synthétiques. En outre, l'invention concerne un procédé d'utilisation d'opsines synthétiques pour la restauration de la vision et d'autres applications, la séquence d'acides aminés de l'opsine synthétique étant modifiée pour fournir une sensibilité à la lumière, une cinétique et une sélectivité ionique améliorées.
EP24797864.6A 2023-04-24 2024-04-24 Modulation optogénétique par opsines à caractéristiques multiples pour la restauration de la vision et d'autres applications associées Pending EP4704877A2 (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202363461546P 2023-04-24 2023-04-24
PCT/US2024/026082 WO2024226667A2 (fr) 2023-04-24 2024-04-24 Modulation optogénétique par opsines à caractéristiques multiples pour la restauration de la vision et d'autres applications associées

Publications (1)

Publication Number Publication Date
EP4704877A2 true EP4704877A2 (fr) 2026-03-11

Family

ID=93257469

Family Applications (1)

Application Number Title Priority Date Filing Date
EP24797864.6A Pending EP4704877A2 (fr) 2023-04-24 2024-04-24 Modulation optogénétique par opsines à caractéristiques multiples pour la restauration de la vision et d'autres applications associées

Country Status (2)

Country Link
EP (1) EP4704877A2 (fr)
WO (1) WO2024226667A2 (fr)

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10947281B2 (en) * 2012-03-05 2021-03-16 Wayne State University Identification of channelrhodopsin-2 (ChR2) mutations and methods of use
US12459977B2 (en) * 2016-11-06 2025-11-04 Nanoscope Therapeutics, Inc. Optogenetic modulation by multi-characteristic opsins for vision restoration and other applications thereof
WO2018106369A2 (fr) * 2016-11-06 2018-06-14 Nanoscope Technologies Llc Modulation optogénétique par opsines à caractéristiques multiples pour la restauration de la vision et d'autres applications associées

Also Published As

Publication number Publication date
WO2024226667A3 (fr) 2025-01-09
WO2024226667A2 (fr) 2024-10-31

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