EP4735604A2 - Auf die expression von huntingtin abzielende inhibitorische rna - Google Patents

Auf die expression von huntingtin abzielende inhibitorische rna

Info

Publication number
EP4735604A2
EP4735604A2 EP24833032.6A EP24833032A EP4735604A2 EP 4735604 A2 EP4735604 A2 EP 4735604A2 EP 24833032 A EP24833032 A EP 24833032A EP 4735604 A2 EP4735604 A2 EP 4735604A2
Authority
EP
European Patent Office
Prior art keywords
sequence
seq
rna
raav
polynucleotide
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
EP24833032.6A
Other languages
English (en)
French (fr)
Inventor
Christopher CALI
Francesca CARGNIN
Benjamin J. HALEY
Juha Savola
Quanyi Li
Mathew Maoxiang LI
Philip Lee PRICE
Elizabeth Ramsburg
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.)
Genentech Inc
Spark Therapeutics Inc
Original Assignee
Genentech Inc
Spark 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 Genentech Inc, Spark Therapeutics Inc filed Critical Genentech Inc
Publication of EP4735604A2 publication Critical patent/EP4735604A2/de
Pending legal-status Critical Current

Links

Classifications

    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141—MicroRNAs, miRNAs
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific
    • 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)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Neurosurgery (AREA)
  • Veterinary Medicine (AREA)
  • Neurology (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Hospice & Palliative Care (AREA)
  • General Chemical & Material Sciences (AREA)
  • Psychiatry (AREA)
  • Epidemiology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Virology (AREA)
EP24833032.6A 2023-06-30 2024-06-28 Auf die expression von huntingtin abzielende inhibitorische rna Pending EP4735604A2 (de)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202363511187P 2023-06-30 2023-06-30
US202363591868P 2023-10-20 2023-10-20
US202463557370P 2024-02-23 2024-02-23
US202463654508P 2024-05-31 2024-05-31
PCT/US2024/036090 WO2025006937A2 (en) 2023-06-30 2024-06-28 Inhibitory rna targeting huntingtin expression

Publications (1)

Publication Number Publication Date
EP4735604A2 true EP4735604A2 (de) 2026-05-06

Family

ID=93940054

Family Applications (1)

Application Number Title Priority Date Filing Date
EP24833032.6A Pending EP4735604A2 (de) 2023-06-30 2024-06-28 Auf die expression von huntingtin abzielende inhibitorische rna

Country Status (8)

Country Link
EP (1) EP4735604A2 (de)
KR (1) KR20260051037A (de)
AU (1) AU2024307975A1 (de)
CO (1) CO2026000936A2 (de)
IL (1) IL325355A (de)
MX (1) MX2025014836A (de)
TW (1) TW202517782A (de)
WO (1) WO2025006937A2 (de)

Family Cites Families (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP7009356B2 (ja) * 2015-04-03 2022-01-25 ユニバーシティ・オブ・マサチューセッツ ハンチンチンｍＲＮＡをターゲティングするオリゴヌクレオチド化合物

Also Published As

Publication number Publication date
WO2025006937A2 (en) 2025-01-02
TW202517782A (zh) 2025-05-01
KR20260051037A (ko) 2026-04-15
IL325355A (en) 2026-02-01
AU2024307975A1 (en) 2026-01-15
WO2025006937A9 (en) 2025-08-21
MX2025014836A (es) 2026-04-01
WO2025006937A3 (en) 2025-04-24
CO2026000936A2 (es) 2026-04-16

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