BR112021023473A2 - Vetores de terapia genética para osteopetrose maligna infantil - Google Patents

Vetores de terapia genética para osteopetrose maligna infantil

Info

Publication number
BR112021023473A2
BR112021023473A2 BR112021023473A BR112021023473A BR112021023473A2 BR 112021023473 A2 BR112021023473 A2 BR 112021023473A2 BR 112021023473 A BR112021023473 A BR 112021023473A BR 112021023473 A BR112021023473 A BR 112021023473A BR 112021023473 A2 BR112021023473 A2 BR 112021023473A2
Authority
BR
Brazil
Prior art keywords
gene therapy
therapy vectors
childhood malignant
malignant osteopetrosis
osteopetrosis
Prior art date
Application number
BR112021023473A
Other languages
English (en)
Inventor
Brian Beard
David Ricks
Raj Prabhakar
Original Assignee
Spacecraft Seven Llc
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 Spacecraft Seven Llc filed Critical Spacecraft Seven Llc
Publication of BR112021023473A2 publication Critical patent/BR112021023473A2/pt

Links

Classifications

    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • 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
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702—Regulators; Modulating activity
    • 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
    • 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
    • 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
    • A01K2267/035—Animal model for multifactorial diseases
    • A01K2267/0387—Animal model for diseases of the immune 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/16011—Human Immunodeficiency Virus, HIV
    • C12N2740/16041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/16043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Microbiology (AREA)
  • Toxicology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Immunology (AREA)
  • Cell Biology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

vetores de terapia genética para osteopetrose maligna infantil. a presente divulgação fornece vetores de terapia gênica aprimorados com-preendendo uma sequência polinucleotídica que codifica um polipeptídeo tcirg1 ou variante funcional do mesmo, métodos de uso dos mesmos, composições farma-cêuticas e muito mais. em particular, a divulgação fornece vetores lentivirais para o tratamento de osteopetrose maligna infantil (imo).
BR112021023473A 2019-05-23 2020-05-22 Vetores de terapia genética para osteopetrose maligna infantil BR112021023473A2 (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962852216P 2019-05-23 2019-05-23
PCT/US2020/034394 WO2020237219A1 (en) 2019-05-23 2020-05-22 Gene therapy vectors for infantile malignant osteopetrosis

Publications (1)

Publication Number Publication Date
BR112021023473A2 true BR112021023473A2 (pt) 2022-02-01

Family

ID=73458718

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112021023473A BR112021023473A2 (pt) 2019-05-23 2020-05-22 Vetores de terapia genética para osteopetrose maligna infantil

Country Status (12)

Country Link
US (1) US20220218844A1 (pt)
EP (1) EP3973066A4 (pt)
JP (1) JP2022532802A (pt)
KR (1) KR20220012266A (pt)
CN (1) CN113994008A (pt)
AU (1) AU2020278046A1 (pt)
BR (1) BR112021023473A2 (pt)
CA (1) CA3137700A1 (pt)
IL (1) IL288193A (pt)
MX (1) MX2021014337A (pt)
SG (1) SG11202112347RA (pt)
WO (1) WO2020237219A1 (pt)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN116194154A (zh) 2020-08-07 2023-05-30 太空飞船七有限责任公司 使用aav载体的plakophilin-2(pkp2)基因疗法

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2650507A1 (en) * 2006-04-27 2007-11-08 Universite De Montreal Assessment and reduction of risk of graft-versus-host disease
AU2013309488A1 (en) * 2012-08-29 2015-03-05 Nature Technology Corporation DNA plasmids with improved expression
WO2014077863A1 (en) * 2012-11-19 2014-05-22 Nature Technology Corporation Replicative minicircle vectors with improved expression
AU2017207906B2 (en) * 2016-01-15 2021-03-11 American Gene Technologies International Inc. Methods and compositions for the activation of gamma-delta T-cells
KR102529012B1 (ko) * 2016-04-22 2023-05-09 크라제 메디컬 씨오 리미티드 세포성 면역요법을 위한 조성물 및 방법

Also Published As

Publication number Publication date
US20220218844A1 (en) 2022-07-14
JP2022532802A (ja) 2022-07-19
SG11202112347RA (en) 2021-12-30
CN113994008A (zh) 2022-01-28
WO2020237219A1 (en) 2020-11-26
CA3137700A1 (en) 2020-11-26
EP3973066A4 (en) 2023-07-26
AU2020278046A1 (en) 2022-01-27
MX2021014337A (es) 2022-03-17
IL288193A (en) 2022-01-01
EP3973066A1 (en) 2022-03-30
KR20220012266A (ko) 2022-02-03

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