AU2021399929A1 - Therapeutic lama2 payload for treatment of congenital muscular dystrophy - Google Patents

Therapeutic lama2 payload for treatment of congenital muscular dystrophy Download PDF

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Publication number
AU2021399929A1
AU2021399929A1 AU2021399929A AU2021399929A AU2021399929A1 AU 2021399929 A1 AU2021399929 A1 AU 2021399929A1 AU 2021399929 A AU2021399929 A AU 2021399929A AU 2021399929 A AU2021399929 A AU 2021399929A AU 2021399929 A1 AU2021399929 A1 AU 2021399929A1
Authority
AU
Australia
Prior art keywords
protein
seq
nucleic acid
transposase
amino acid
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
AU2021399929A
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English (en)
Inventor
Marc Guell Cargol
Maria Pallares Masmitja
Avencia Sanchez-mejias Garcia
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.)
Universitat Pompeu Fabra UPF
Original Assignee
Universitat Pompeu Fabra UPF
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 Universitat Pompeu Fabra UPF filed Critical Universitat Pompeu Fabra UPF
Publication of AU2021399929A1 publication Critical patent/AU2021399929A1/en
Pending legal-status Critical Current

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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • 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
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • 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
    • C12N9/10—Transferases (2.)
    • C12N9/12—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • C12N9/1241—Nucleotidyltransferases (2.7.7)
    • 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 [RNase]; Deoxyribonucleases [DNase]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • C07K2319/81—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor containing a Zn-finger domain for DNA binding

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Cell Biology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Mycology (AREA)
  • Toxicology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Epidemiology (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
AU2021399929A 2020-12-16 2021-12-16 Therapeutic lama2 payload for treatment of congenital muscular dystrophy Pending AU2021399929A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
EP20214691 2020-12-16
EP20214691.6 2020-12-16
EP21209721 2021-11-22
EP21209721.6 2021-11-22
PCT/EP2021/086333 WO2022129430A1 (en) 2020-12-16 2021-12-16 Therapeutic lama2 payload for treatment of congenital muscular dystrophy

Publications (1)

Publication Number Publication Date
AU2021399929A1 true AU2021399929A1 (en) 2023-07-06

Family

ID=79269765

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2021399929A Pending AU2021399929A1 (en) 2020-12-16 2021-12-16 Therapeutic lama2 payload for treatment of congenital muscular dystrophy

Country Status (7)

Country Link
US (1) US20250269057A1 (https=)
EP (1) EP4263818A1 (https=)
JP (1) JP2023553701A (https=)
KR (1) KR20230125806A (https=)
AU (1) AU2021399929A1 (https=)
CA (1) CA3202411A1 (https=)
WO (1) WO2022129430A1 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN118185901B (zh) * 2022-12-12 2025-03-21 北京精缮生物科技有限责任公司 一种PBase蛋白、融合蛋白、核酸和基因整合系统及应用

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8048999B2 (en) 2005-12-13 2011-11-01 Kyoto University Nuclear reprogramming factor
JP5813321B2 (ja) 2007-03-23 2015-11-17 ウィスコンシン アラムニ リサーチ ファンデーション 体細胞の再プログラム化
JP2018513681A (ja) * 2015-03-31 2018-05-31 エクセリゲン サイエンティフィック, インコーポレイテッドExeligen Scientific, Inc. 細胞または生物のゲノムへのＤＮＡ配列の標的化組み込みのためのＣａｓ９レトロウイルスインテグラーゼおよびＣａｓ９レコンビナーゼ系
US20200095579A1 (en) * 2017-02-22 2020-03-26 Crispr Therapeutics Ag Materials and methods for treatment of merosin-deficient cogenital muscular dystrophy (mdcmd) and other laminin, alpha 2 (lama2) gene related conditions or disorders
WO2018175872A1 (en) * 2017-03-24 2018-09-27 President And Fellows Of Harvard College Methods of genome engineering by nuclease-transposase fusion proteins
US20220235379A1 (en) * 2019-06-11 2022-07-28 Universitat Pompeu Fabra Targeted gene editing constructs and methods of using the same

Also Published As

Publication number Publication date
EP4263818A1 (en) 2023-10-25
KR20230125806A (ko) 2023-08-29
CA3202411A1 (en) 2022-06-23
JP2023553701A (ja) 2023-12-25
US20250269057A1 (en) 2025-08-28
WO2022129430A1 (en) 2022-06-23

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