CA3049771A1 - Compositions and methods for treating farber disease - Google Patents

Compositions and methods for treating farber disease Download PDF

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Publication number
CA3049771A1
CA3049771A1 CA3049771A CA3049771A CA3049771A1 CA 3049771 A1 CA3049771 A1 CA 3049771A1 CA 3049771 A CA3049771 A CA 3049771A CA 3049771 A CA3049771 A CA 3049771A CA 3049771 A1 CA3049771 A1 CA 3049771A1
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Canada
Prior art keywords
rhac
subject
effective amount
cell
pharmaceutical composition
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Pending
Application number
CA3049771A
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English (en)
French (fr)
Inventor
Edward H. Schuchman
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.)
Icahn School of Medicine at Mount Sinai
Original Assignee
Icahn School of Medicine at Mount Sinai
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Application filed by Icahn School of Medicine at Mount Sinai filed Critical Icahn School of Medicine at Mount Sinai
Publication of CA3049771A1 publication Critical patent/CA3049771A1/en
Pending legal-status Critical Current

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Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/50—Hydrolases (3) acting on carbon-nitrogen bonds, other than peptide bonds (3.5), e.g. asparaginase
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • 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
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/78—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
    • C12N9/80—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5) acting on amide bonds in linear amides (3.5.1)
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/072—Animals genetically altered by homologous recombination maintaining or altering function, i.e. knock in
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00—Animals characterised by species
    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • A01K2267/0306—Animal model for genetic diseases
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y305/00—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
    • C12Y305/01—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in linear amides (3.5.1)
    • C12Y305/01023—Ceramidase (3.5.1.23)

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Virology (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Obesity (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
CA3049771A 2017-01-13 2018-01-12 Compositions and methods for treating farber disease Pending CA3049771A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762446166P 2017-01-13 2017-01-13
US62/446,166 2017-01-13
PCT/US2018/013509 WO2018132667A1 (en) 2017-01-13 2018-01-12 Compositions and methods for treating farber disease

Publications (1)

Publication Number Publication Date
CA3049771A1 true CA3049771A1 (en) 2018-07-19

Family

ID=62839661

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3049771A Pending CA3049771A1 (en) 2017-01-13 2018-01-12 Compositions and methods for treating farber disease

Country Status (8)

Country Link
US (4) US20190343936A1 (https=)
EP (2) EP3568154B1 (https=)
JP (3) JP7576293B2 (https=)
AU (2) AU2018207564A1 (https=)
CA (1) CA3049771A1 (https=)
JO (1) JOP20190164B1 (https=)
MX (1) MX2019008038A (https=)
WO (1) WO2018132667A1 (https=)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX2019008038A (es) * 2017-01-13 2020-02-05 Icahn School Med Mount Sinai Composiciones y metodos para tratar la enfermedad de farber.
EP3648776A4 (en) * 2017-07-06 2021-04-07 The Medical College of Wisconsin, Inc. NEW IN-VITRO AND IN-VIVO ENRICHMENT STRATEGY AGAINST LYMPHOCYTE FROM VECTOR-TRANSDUCED HSC FOR THE TREATMENT OF DISEASES
KR20250016468A (ko) 2018-02-02 2025-02-03 엔지반트 테라퓨틱스 게엠베하 파버병을 치료하는 방법
EP3784695B1 (en) 2018-04-27 2023-08-09 The Medical College of Wisconsin, Inc. Use of lentivector-transduced t-rapa cells for amelioration of lysosomal storage disorders
WO2020152532A1 (en) 2019-01-23 2020-07-30 Enzyvant Therapeutics Gmbh Method of ameliorating a pro-inflammatory immunophenotype in farber disease subjects by repeated administration of a recombinant human acid ceramidase
WO2021055801A1 (en) 2019-09-18 2021-03-25 The Medical College Of Wisconsin, Inc. Improved alpha-galactosidase protein for enzyme replacement therapy (ert) and methods of use

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PL2854910T3 (pl) 2012-06-01 2020-07-27 Icahn School Of Medicine At Mount Sinai Poziomy ceramidu w leczeniu i zapobieganiu infekcjom
CA2905449C (en) 2013-03-14 2024-09-10 Icahn School Of Medicine At Mount Sinai THERAPEUTIC COMPOSITIONS OF ACID CERAMIDASE AND THEIR PROCESSES OF MANUFACTURE AND USE
MX2019008038A (es) * 2017-01-13 2020-02-05 Icahn School Med Mount Sinai Composiciones y metodos para tratar la enfermedad de farber.
WO2019060837A1 (en) * 2017-09-25 2019-03-28 Enzyvant Farber Gmbh PROCESS FOR PRODUCING RECOMBINANT HUMAN ACID CERAMIDASE
KR20250016468A (ko) * 2018-02-02 2025-02-03 엔지반트 테라퓨틱스 게엠베하 파버병을 치료하는 방법

Also Published As

Publication number Publication date
EP3568154B1 (en) 2023-07-12
JP2023011938A (ja) 2023-01-24
WO2018132667A1 (en) 2018-07-19
JP7576293B2 (ja) 2024-10-31
AU2025200254A1 (en) 2025-01-30
EP4295903A2 (en) 2023-12-27
JP2020514305A (ja) 2020-05-21
MX2019008038A (es) 2020-02-05
US20240075113A1 (en) 2024-03-07
EP4295903A3 (en) 2024-03-27
US20250170225A1 (en) 2025-05-29
JP2024169509A (ja) 2024-12-05
EP3568154A4 (en) 2020-11-18
US20190343936A1 (en) 2019-11-14
JOP20190164A1 (ar) 2019-07-02
EP3568154A1 (en) 2019-11-20
AU2018207564A1 (en) 2019-08-08
US20220313800A1 (en) 2022-10-06
JOP20190164B1 (ar) 2023-09-17

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