EP4712948A2 - Geneditierungssysteme und zusammensetzungen zur behandlung von hämoglobinopathien und verfahren zur verwendung davon - Google Patents

Geneditierungssysteme und zusammensetzungen zur behandlung von hämoglobinopathien und verfahren zur verwendung davon

Info

Publication number
EP4712948A2
EP4712948A2 EP24808122.6A EP24808122A EP4712948A2 EP 4712948 A2 EP4712948 A2 EP 4712948A2 EP 24808122 A EP24808122 A EP 24808122A EP 4712948 A2 EP4712948 A2 EP 4712948A2
Authority
EP
European Patent Office
Prior art keywords
mol
certain embodiments
optionally substituted
lipid nanoparticle
lipid
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
EP24808122.6A
Other languages
English (en)
French (fr)
Inventor
Aisha ALJANAHI
Rajat Das
Daniel Egan
Giedrius GASIUNAS
Sierra HARKEN
Megan HOBAN
Hari JAYARAM
Muthusamy Jayaraman
Shinu JOHN
Jacob LAYER
Kourtney MENDELLO
Elisabeth NARAYANAN
Matthew PANDELAKIS
Vladimir PRESNYAK
Karolina Anna KOSAKOWSKA
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.)
Renagade Therapeutics Management Inc
Original Assignee
Renagade Therapeutics Management 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 Renagade Therapeutics Management Inc filed Critical Renagade Therapeutics Management Inc
Publication of EP4712948A2 publication Critical patent/EP4712948A2/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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/48—Preparations in capsules, e.g. of gelatin, of chocolate
    • A61K9/50—Microcapsules having a gas, liquid or semi-solid filling; Solid microparticles or pellets surrounded by a distinct coating layer, e.g. coated microspheres, coated drug crystals
    • A61K9/51—Nanocapsules; Nanoparticles
    • A61K9/5107—Excipients; Inactive ingredients
    • A61K9/5123—Organic compounds, e.g. fats, sugars
    • 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
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
EP24808122.6A 2023-05-17 2024-05-16 Geneditierungssysteme und zusammensetzungen zur behandlung von hämoglobinopathien und verfahren zur verwendung davon Pending EP4712948A2 (de)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US202363502846P 2023-05-17 2023-05-17
US202363584014P 2023-09-20 2023-09-20
US202363593764P 2023-10-27 2023-10-27
US202463557134P 2024-02-23 2024-02-23
US202463570053P 2024-03-26 2024-03-26
US202463638004P 2024-04-24 2024-04-24
PCT/US2024/029749 WO2024238828A2 (en) 2023-05-17 2024-05-16 Gene editing systems and compositions for treatment of hemoglobinopathies and methods of using the same

Publications (1)

Publication Number Publication Date
EP4712948A2 true EP4712948A2 (de) 2026-03-25

Family

ID=93520226

Family Applications (1)

Application Number Title Priority Date Filing Date
EP24808122.6A Pending EP4712948A2 (de) 2023-05-17 2024-05-16 Geneditierungssysteme und zusammensetzungen zur behandlung von hämoglobinopathien und verfahren zur verwendung davon

Country Status (3)

Country Link
EP (1) EP4712948A2 (de)
AU (1) AU2024272060A1 (de)
WO (1) WO2024238828A2 (de)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN119410609B (zh) * 2023-11-24 2025-10-14 山东舜丰生物科技有限公司 一种突变的Cas蛋白及其应用
WO2025155753A2 (en) * 2024-01-17 2025-07-24 Renagade Therapeutics Management Inc. Improved gene editing system, guides, and methods

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11268077B2 (en) * 2018-02-05 2022-03-08 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of hemoglobinopathies
WO2019178426A1 (en) * 2018-03-14 2019-09-19 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
WO2022251665A1 (en) * 2021-05-28 2022-12-01 Renagade Therapeutics Management Inc. Lipid nanoparticles and methods of use thereof

Also Published As

Publication number Publication date
WO2024238828A2 (en) 2024-11-21
WO2024238828A3 (en) 2024-12-26
AU2024272060A1 (en) 2026-01-08

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