WO2023240157A3 - Compositions and methods for the targeting of dmd - Google Patents

Compositions and methods for the targeting of dmd Download PDF

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Publication number
WO2023240157A3
WO2023240157A3 PCT/US2023/068091 US2023068091W WO2023240157A3 WO 2023240157 A3 WO2023240157 A3 WO 2023240157A3 US 2023068091 W US2023068091 W US 2023068091W WO 2023240157 A3 WO2023240157 A3 WO 2023240157A3
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WO
WIPO (PCT)
Prior art keywords
dmd
methods
systems
gene
cells
Prior art date
Application number
PCT/US2023/068091
Other languages
French (fr)
Other versions
WO2023240157A2 (en
Inventor
Wenyuan ZHOU
Cole URNES
Isabel COLIN
Cécile FORTUNY
Lola TAKHIROV
Sarah DENNY
Sean Higgins
Brett T. STAAHL
Maroof ADIL
Benjamin OAKES
Original Assignee
Scribe 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 Scribe Therapeutics Inc. filed Critical Scribe Therapeutics Inc.
Publication of WO2023240157A2 publication Critical patent/WO2023240157A2/en
Publication of WO2023240157A3 publication Critical patent/WO2023240157A3/en

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    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • 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
    • C12N15/52—Genes encoding for enzymes or proenzymes
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases RNAses, DNAses
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
    • 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 [CRISPRs]
    • 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
    • C12N2320/33—Alteration of splicing

Abstract

Provided herein are Class 2 Type V CRISPR gene editing systems useful in the modification of a dystrophin (DMD) gene. In some aspects, provided herein are CasX:guide RNA (gRNA) systems for modifying a DMD gene. The systems are also useful for introduction into cells, for example eukaryotic cells having mutations in the dystrophin protein. Also provided are methods of using such CasX:gRNA systems to modify cells having such mutations and utility in methods of treatment of a subject with a DMD-related disease.
PCT/US2023/068091 2022-06-08 2023-06-07 Compositions and methods for the targeting of dmd WO2023240157A2 (en)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US202263350318P 2022-06-08 2022-06-08
US63/350,318 2022-06-08
US202363504695P 2023-05-26 2023-05-26
US63/504,695 2023-05-26
US202363505975P 2023-06-02 2023-06-02
US63/505,975 2023-06-02

Publications (2)

Publication Number Publication Date
WO2023240157A2 WO2023240157A2 (en) 2023-12-14
WO2023240157A3 true WO2023240157A3 (en) 2024-01-18

Family

ID=87070857

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2023/068091 WO2023240157A2 (en) 2022-06-08 2023-06-07 Compositions and methods for the targeting of dmd

Country Status (1)

Country Link
WO (1) WO2023240157A2 (en)

Citations (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019152609A1 (en) * 2018-01-31 2019-08-08 The Board Of Regents Of The University Of Texas System Compositions and methods for correcting dystrophin mutations in human cardiomyocytes
WO2019246480A1 (en) * 2018-06-21 2019-12-26 The Board Of Regents Of The University Of Texas System Correction of dystrophin exon 43, exon 45, or exon 52 deletions in duchenne muscular dystrophy
WO2020247882A1 (en) * 2019-06-07 2020-12-10 Scribe Therapeutics Inc. Engineered casx systems
WO2021113763A1 (en) * 2019-12-06 2021-06-10 Scribe Therapeutics Inc. Compositions and methods for the targeting of rhodopsin
WO2021113772A1 (en) * 2019-12-06 2021-06-10 Scribe Therapeutics Inc. Particle delivery systems
WO2021188729A1 (en) * 2020-03-18 2021-09-23 Scribe Therapeutics Inc. Compositions and methods for the targeting of c9orf72
WO2022056000A1 (en) * 2020-09-09 2022-03-17 Vertex Pharmaceuticals Incorporated Compositions and methods for treatment of duchenne muscular dystrophy
WO2022060841A2 (en) * 2020-09-15 2022-03-24 Research Institute At Nationwide Children's Hospital Aav-mediated homology-independent targeted integration gene editing for correction of diverse dmd mutations in patients with muscular dystrophy
WO2022125843A1 (en) * 2020-12-09 2022-06-16 Scribe Therapeutics Inc. Aav vectors for gene editing

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
EP3374494A4 (en) 2015-11-11 2019-05-01 Coda Biotherapeutics, Inc. Crispr compositions and methods of using the same for gene therapy
US11773409B2 (en) 2017-04-21 2023-10-03 The Board Of Trustees Of The Leland Stanford Junior University CRISPR/Cas 9-mediated integration of polynucleotides by sequential homologous recombination of AAV donor vectors
WO2020247883A2 (en) 2019-06-07 2020-12-10 Scribe Therapeutics Inc. Deep mutational evolution of biomolecules
WO2021133829A1 (en) 2019-12-23 2021-07-01 The Regents Of The University Of California Crispr-cas effector polypeptides and methods of use thereof
TW202237836A (en) 2020-12-03 2022-10-01 美商斯奎柏治療公司 Engineered class 2 type v crispr systems
EP4351660A2 (en) 2021-06-09 2024-04-17 Scribe Therapeutics Inc. Particle delivery systems
WO2022261148A1 (en) 2021-06-09 2022-12-15 Scribe Therapeutics Inc. Compositions of glycoprotein particles
AU2022349684A1 (en) 2021-09-23 2024-03-21 Scribe Therapeutics Inc. Self-inactivating vectors for gene editing

Patent Citations (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019152609A1 (en) * 2018-01-31 2019-08-08 The Board Of Regents Of The University Of Texas System Compositions and methods for correcting dystrophin mutations in human cardiomyocytes
WO2019246480A1 (en) * 2018-06-21 2019-12-26 The Board Of Regents Of The University Of Texas System Correction of dystrophin exon 43, exon 45, or exon 52 deletions in duchenne muscular dystrophy
WO2020247882A1 (en) * 2019-06-07 2020-12-10 Scribe Therapeutics Inc. Engineered casx systems
WO2021113763A1 (en) * 2019-12-06 2021-06-10 Scribe Therapeutics Inc. Compositions and methods for the targeting of rhodopsin
WO2021113772A1 (en) * 2019-12-06 2021-06-10 Scribe Therapeutics Inc. Particle delivery systems
WO2021188729A1 (en) * 2020-03-18 2021-09-23 Scribe Therapeutics Inc. Compositions and methods for the targeting of c9orf72
WO2022056000A1 (en) * 2020-09-09 2022-03-17 Vertex Pharmaceuticals Incorporated Compositions and methods for treatment of duchenne muscular dystrophy
WO2022060841A2 (en) * 2020-09-15 2022-03-24 Research Institute At Nationwide Children's Hospital Aav-mediated homology-independent targeted integration gene editing for correction of diverse dmd mutations in patients with muscular dystrophy
WO2022125843A1 (en) * 2020-12-09 2022-06-16 Scribe Therapeutics Inc. Aav vectors for gene editing

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