SG11202008400SA - Compositions and methods for stem cell transplant - Google Patents

Compositions and methods for stem cell transplant

Info

Publication number
SG11202008400SA
SG11202008400SA SG11202008400SA SG11202008400SA SG11202008400SA SG 11202008400S A SG11202008400S A SG 11202008400SA SG 11202008400S A SG11202008400S A SG 11202008400SA SG 11202008400S A SG11202008400S A SG 11202008400SA SG 11202008400S A SG11202008400S A SG 11202008400SA
Authority
SG
Singapore
Prior art keywords
compositions
methods
stem cell
cell transplant
transplant
Prior art date
Application number
SG11202008400SA
Inventor
Brian Beard
Navarro Julian Sevilla
Gaurav Shah
Kinnari Patel
Raj Prabhakar
Original Assignee
Rocket Pharmaceuticals Ltd
Fundacion Para La Investigacion Biomedica Del Hospital Infantil Univ Nino Jesus
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 Rocket Pharmaceuticals Ltd, Fundacion Para La Investigacion Biomedica Del Hospital Infantil Univ Nino Jesus filed Critical Rocket Pharmaceuticals Ltd
Publication of SG11202008400SA publication Critical patent/SG11202008400SA/en

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/14—Blood; Artificial blood
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/06—Antianaemics
    • 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
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • 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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0634—Cells from the blood or the immune system
    • C12N5/0647—Haematopoietic stem cells; Uncommitted or multipotent progenitors
    • 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
    • C12N2510/00—Genetically modified 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/10—Plasmid DNA
    • C12N2800/106—Plasmid DNA for vertebrates
    • C12N2800/107—Plasmid DNA for vertebrates for mammalian
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Hematology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Immunology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Developmental Biology & Embryology (AREA)
  • Cell Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Diabetes (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
SG11202008400SA 2018-04-11 2019-04-11 Compositions and methods for stem cell transplant SG11202008400SA (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862656292P 2018-04-11 2018-04-11
PCT/US2019/027083 WO2019200167A1 (en) 2018-04-11 2019-04-11 Compositions and methods for stem cell transplant

Publications (1)

Publication Number Publication Date
SG11202008400SA true SG11202008400SA (en) 2020-09-29

Family

ID=68162990

Family Applications (1)

Application Number Title Priority Date Filing Date
SG11202008400SA SG11202008400SA (en) 2018-04-11 2019-04-11 Compositions and methods for stem cell transplant

Country Status (11)

Country Link
US (1) US20210154237A1 (en)
JP (1) JP2021521180A (en)
KR (1) KR20210003738A (en)
CN (1) CN112153973A (en)
AU (1) AU2019252662A1 (en)
BR (1) BR112020020841A2 (en)
CA (1) CA3093708A1 (en)
IL (2) IL307996A (en)
MX (1) MX2020010718A (en)
SG (1) SG11202008400SA (en)
WO (1) WO2019200167A1 (en)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IL300263A (en) 2020-08-07 2023-03-01 Spacecraft Seven Llc Plakophilin-2 (pkp2) gene therapy using aav vector
CN116724109A (en) * 2020-12-31 2023-09-08 Vor生物制药股份有限公司 Compositions and methods for CD34 gene modification
WO2023183909A2 (en) * 2022-03-25 2023-09-28 Modernatx, Inc. Polynucleotides encoding fanconi anemia, complementation group proteins for the treatment of fanconi anemia

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN104419683A (en) * 2013-09-10 2015-03-18 中国科学院生物物理研究所 Method for preparing autologous induced pluripotent stem cells of patient with Fanconi anemia and application of method
US20170175143A1 (en) * 2014-05-20 2017-06-22 Regents Of The University Of Minnesota Method for editing a genetic sequence
US11030531B2 (en) * 2014-06-06 2021-06-08 Trustees Of Boston University DNA recombinase circuits for logical control of gene expression
EP3247808B1 (en) * 2015-01-21 2021-05-05 Fred Hutchinson Cancer Research Center Point-of-care and/or portable platform for gene therapy
KR20240096679A (en) * 2016-04-20 2024-06-26 센트로 데 인베스띠가씨오네스 에너제티까스 메디오암비엔딸레스 와이 테크놀로지까스, 오.에이., 엠.피. Compositions and methods for enhanced gene expression of pklr
US20190345450A1 (en) * 2016-06-17 2019-11-14 Fred Hutchinson Cancer Research Center Strategies to assess and/or produce cell populations with predictive engraftment potential
SG11201901718VA (en) * 2016-09-08 2019-03-28 Centro De Investigaciones Energeticas Medioambientales Y Tecnologicas Gene therapy for patients with fanconi anemia

Also Published As

Publication number Publication date
MX2020010718A (en) 2021-01-20
JP2021521180A (en) 2021-08-26
IL307996A (en) 2023-12-01
CN112153973A (en) 2020-12-29
WO2019200167A1 (en) 2019-10-17
CA3093708A1 (en) 2019-10-17
IL277878A (en) 2020-11-30
KR20210003738A (en) 2021-01-12
BR112020020841A2 (en) 2021-01-19
RU2020129103A (en) 2022-03-03
AU2019252662A1 (en) 2020-09-24
EP3773636A1 (en) 2021-02-17
US20210154237A1 (en) 2021-05-27

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