IL300187A - Gene therapy CSRP3 (protein 3 rich in cysteine and glycine) - Google Patents

Gene therapy CSRP3 (protein 3 rich in cysteine and glycine)

Info

Publication number
IL300187A
IL300187A IL300187A IL30018723A IL300187A IL 300187 A IL300187 A IL 300187A IL 300187 A IL300187 A IL 300187A IL 30018723 A IL30018723 A IL 30018723A IL 300187 A IL300187 A IL 300187A
Authority
IL
Israel
Prior art keywords
csrp3
cysteine
gene therapy
rich protein
glycine rich
Prior art date
Application number
IL300187A
Other languages
English (en)
Hebrew (he)
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 IL300187A publication Critical patent/IL300187A/en

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/04—Inotropic agents, i.e. stimulants of cardiac contraction; Drugs for heart failure
    • 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
    • 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
    • C07K14/4705—Regulators; Modulating activity stimulating, promoting or activating 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
    • C07K14/46—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
    • C07K14/4716—Muscle proteins, e.g. myosin, actin
    • 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
    • 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
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • 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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—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)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Biotechnology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Cardiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Virology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Hospice & Palliative Care (AREA)
  • Epidemiology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Immunology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
IL300187A 2020-08-05 2021-08-03 Gene therapy CSRP3 (protein 3 rich in cysteine and glycine) IL300187A (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202063061727P 2020-08-05 2020-08-05
PCT/US2021/044412 WO2022031756A1 (en) 2020-08-05 2021-08-03 Csrp3 (cysteine and glycine rich protein 3) gene therapy

Publications (1)

Publication Number Publication Date
IL300187A true IL300187A (en) 2023-03-01

Family

ID=80118758

Family Applications (1)

Application Number Title Priority Date Filing Date
IL300187A IL300187A (en) 2020-08-05 2021-08-03 Gene therapy CSRP3 (protein 3 rich in cysteine and glycine)

Country Status (11)

Country Link
US (1) US20230257431A1 (enExample)
EP (1) EP4192962A4 (enExample)
JP (1) JP2023536618A (enExample)
KR (1) KR20230042468A (enExample)
CN (1) CN116234916A (enExample)
AU (1) AU2021321410A1 (enExample)
BR (1) BR112023001336A2 (enExample)
CA (1) CA3184983A1 (enExample)
IL (1) IL300187A (enExample)
MX (1) MX2023000994A (enExample)
WO (1) WO2022031756A1 (enExample)

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102673203B1 (ko) 2016-04-20 2024-06-10 센트로 데 인베스띠가씨오네스 에너제티까스 메디오암비엔딸레스 와이 테크놀로지까스, 오.에이., 엠.피. Pklr의 유전자 발현 증강을 위한 조성물 및 방법
AU2018352583B2 (en) 2017-10-16 2025-04-17 Centro de Investigaciones Energeticas, Medioambientales Y Tecnologicas, O.A., M.P. Lentiviral vectors for delivery of PKLR to treat Pyruvate Kinase Deficiency
US12539316B2 (en) 2018-04-11 2026-02-03 Spacecraft Seven, Llc Compositions and methods for stem cell transplant
IL278213B2 (en) 2018-04-27 2026-04-01 Rocket Pharmaceuticals Ltd A gene therapy vector encoding an e3 ubiquitin protein ligase (park2) and use thereof in treating or inhibiting the onset of parkinson’s disease (pd)
IL280465B2 (en) 2018-07-30 2025-03-01 Centro De Investig Energeticas Medioambientales Y Tecnologicas O A M P Methods for gene modification of hematopoietic cells
FI4103214T3 (fi) 2020-02-13 2026-07-03 Tenaya Therapeutics Inc Geeniterapian vektorit sydänsairauden hoitoon
WO2022032226A1 (en) 2020-08-07 2022-02-10 Spacecraft Seven, Llc Plakophilin-2 (pkp2) gene therapy using aav vector
WO2023159190A1 (en) * 2022-02-18 2023-08-24 Ginkgo Bioworks, Inc. Gene therapy for arrhythmogenic cardiomyopathy
IL315695A (en) * 2022-03-18 2024-11-01 Univ Florida Methods and compositions for treating RBM20-associated cardiomyopathy with a viral vector
JP2025512574A (ja) * 2022-04-22 2025-04-17 スペースクラフト セブン リミテッド ライアビリティ カンパニー ＡＡＶベクターを使用するＢ細胞リンパ腫２関連Ａｎｔｈａｎｏｇｅｎｅ ３ （ＢＡＧ３）遺伝子治療

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE10004857A1 (de) * 2000-02-03 2001-08-16 Schering Ag MLP-Gen, Nukleinsäuren, Polypeptide und deren Verwendung
CA2693712C (en) 2007-07-14 2018-05-29 The University Of Iowa Research Foundation Methods and compositions for treating brain diseases
EP2792742A1 (en) * 2013-04-17 2014-10-22 Universitätsklinikum Hamburg-Eppendorf (UKE) Gene-therapy vectors for treating cardiomyopathy
EP3564379A1 (en) 2013-09-13 2019-11-06 California Institute of Technology Selective recovery
LT3137497T (lt) 2014-05-02 2021-07-26 Genzyme Corporation Aav vektoriai, skirti tinklainės ir cns genų terapijai
AU2016366549B2 (en) 2015-12-11 2022-11-10 California Institute Of Technology Targeting peptides for directing adeno-associated viruses (AAVs)
CN110997923B (zh) * 2017-03-17 2024-01-02 全国儿童医院研究所 腺相关病毒载体递送肌肉特异性微肌营养不良蛋白以治疗肌营养不良症
DK3684423T5 (da) * 2017-09-20 2024-08-26 4D Molecular Therapeutics Inc Adeno-associerede virusvariantcapsider og fremgangsmåder til anvendelse deraf
CA3097375A1 (en) * 2018-04-27 2019-10-31 Universitat Heidelberg Modified aav capsid polypeptides for treatment of muscular diseases
CN112272705A (zh) * 2018-06-08 2021-01-26 佛罗里达大学研究基金会有限公司 用于心肌病的aav心脏基因治疗
CN114450411A (zh) * 2019-04-01 2022-05-06 特纳亚治疗股份有限公司 具有工程化衣壳的腺相关病毒

Also Published As

Publication number Publication date
BR112023001336A2 (pt) 2023-02-14
WO2022031756A1 (en) 2022-02-10
AU2021321410A1 (en) 2023-04-06
CA3184983A1 (en) 2022-02-10
EP4192962A4 (en) 2025-04-23
KR20230042468A (ko) 2023-03-28
CN116234916A (zh) 2023-06-06
MX2023000994A (es) 2023-03-01
JP2023536618A (ja) 2023-08-28
EP4192962A1 (en) 2023-06-14
US20230257431A1 (en) 2023-08-17

Similar Documents

Publication Publication Date Title
SG11202106771QA (en) Single-molecule protein and peptide sequencing
PH12021550789A1 (en) Disulfide bond stabilized polypeptide compositions and methods of use
EP3946410A4 (en) Therapeutic interferon alpha 1 proteins
EP1751179A4 (en) PEPTIDES DERIVED FROM THE CASEIN AND THEIR THERAPEUTIC USES
IL305809A (en) Cancer neoantigenic peptides and their use
MX2021008207A (es) Proteínas de fusión multifuncionales y usos de las mismas.
GB202006974D0 (en) Chimaeric proteins and therapeutic agents
IL308790A (en) Clamped peptides and their methods
IL299907A (en) Paired peptides and their methods
IL319851A (en) LRRC-15 binding protein structures and uses thereof
IL292047A (en) Proteins containing various, multiple unnatural amino acids and methods for preparing and using such proteins
IL327193A (en) CAS12 protein and its uses
WO2004074486A3 (en) Fusion proteins of interferon alpha muteins with improved properties
IL316176A (en) SIGLEC-8 binding proteins and their uses
GB0216972D0 (en) Peptides and peptide derivaties for the treatment of &-synuclein-related diseases
IL273546B (en) Textured protein packaging and its uses
EP4279507A4 (en) CD73-BINDING PROTEIN AND USE THEREOF
EP4269428A4 (en) NEW PEPTIDE AND ITS USE
IL311185A (en) MOG-binding proteins and uses thereof
HK40094212A (en) Csrp3 (cysteine and glycine rich protein 3) gene therapy
IL318460A (en) RABIES G protein and its uses
GB202403250D0 (en) Fgf21 mutant protein and use thereof
GB202209228D0 (en) Modified amino acids and uses thereof
WO2006122162A3 (en) Use of peptides derived from the growth factor amp-18 for the treatment of mucositis
NZ597470A (en) Therapeutic peptides, polypeptides ans nucleic acid sequences