AR129122A1 - Terapia génica del gen de la proteína 2 del linfoma de células b asociado a atanogén 3 (bag3) mediante un vector aav - Google Patents

Terapia génica del gen de la proteína 2 del linfoma de células b asociado a atanogén 3 (bag3) mediante un vector aav

Info

Publication number
AR129122A1
AR129122A1 ARP230100975A ARP230100975A AR129122A1 AR 129122 A1 AR129122 A1 AR 129122A1 AR P230100975 A ARP230100975 A AR P230100975A AR P230100975 A ARP230100975 A AR P230100975A AR 129122 A1 AR129122 A1 AR 129122A1
Authority
AR
Argentina
Prior art keywords
promoter
gene
vector
bag3
capsid
Prior art date
Application number
ARP230100975A
Other languages
English (en)
Spanish (es)
Inventor
Christopher Dean Herzog
Chester Bittencort Sacramento
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 AR129122A1 publication Critical patent/AR129122A1/es

Links

Classifications

    • 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
    • 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
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/06—Antiarrhythmics
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • 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
    • A01K2217/075—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
    • A01K2217/00—Genetically modified animals
    • A01K2217/20—Animal model comprising regulated expression system
    • A01K2217/206—Animal model comprising tissue-specific expression system, e.g. tissue specific expression of transgene, of Cre recombinase
    • 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
    • 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
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

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)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Cardiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Virology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Heart & Thoracic Surgery (AREA)
  • General Chemical & Material Sciences (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Toxicology (AREA)
  • Hospice & Palliative Care (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Immunology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
ARP230100975A 2022-04-22 2023-04-21 Terapia génica del gen de la proteína 2 del linfoma de células b asociado a atanogén 3 (bag3) mediante un vector aav AR129122A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US202263333798P 2022-04-22 2022-04-22

Publications (1)

Publication Number Publication Date
AR129122A1 true AR129122A1 (es) 2024-07-17

Family

ID=88420755

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP230100975A AR129122A1 (es) 2022-04-22 2023-04-21 Terapia génica del gen de la proteína 2 del linfoma de células b asociado a atanogén 3 (bag3) mediante un vector aav

Country Status (13)

Country Link
US (1) US20250269065A1 (OSRAM)
EP (1) EP4511048A2 (OSRAM)
JP (1) JP2025512574A (OSRAM)
KR (1) KR20250012749A (OSRAM)
CN (1) CN119072323A (OSRAM)
AR (1) AR129122A1 (OSRAM)
AU (1) AU2023258039A1 (OSRAM)
CA (1) CA3248774A1 (OSRAM)
CL (1) CL2024003182A1 (OSRAM)
IL (1) IL316183A (OSRAM)
MX (1) MX2024012761A (OSRAM)
TW (1) TW202409285A (OSRAM)
WO (1) WO2023205767A2 (OSRAM)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN109641063B (zh) 2016-04-20 2022-11-15 能源环境和技术研究中心O.A., M.P. 用于增强pklr的基因表达的组合物和方法
SG11202002263TA (en) 2017-10-16 2020-04-29 Centro De Investigaciones Energeticas Medioambientales Y Tecnologicas Lentiviral vectors for delivery of pklr to treat pyruvate kinase deficiency
JP7576754B2 (ja) 2018-04-11 2024-11-01 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 幹細胞移植のための組成物および方法
WO2024215655A1 (en) * 2023-04-10 2024-10-17 Tenaya Therapeutics, Inc. Cardioprotective bag3 therapies
EP4705326A1 (en) * 2023-05-02 2026-03-11 Research Institute at Nationwide Children's Hospital Gene therapy for treatment of protein misfolding diseases
WO2025235491A1 (en) * 2024-05-07 2025-11-13 Affinia Therapeutics Inc. Recombinant aav for treatment of cardiac diseases

Family Cites Families (3)

* 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
CA3228845A1 (en) * 2014-01-31 2015-08-06 Temple University Of The Commonwealth System Of Higher Education Bag3 and uses thereof in the treatment of heart failure with reduced ejection fraction
AU2020412375A1 (en) * 2019-12-24 2022-06-30 AskBio Inc. Regulatory nucleic acid sequences

Also Published As

Publication number Publication date
MX2024012761A (es) 2024-11-08
CA3248774A1 (en) 2023-10-26
CN119072323A (zh) 2024-12-03
JP2025512574A (ja) 2025-04-17
EP4511048A2 (en) 2025-02-26
AU2023258039A1 (en) 2024-11-21
IL316183A (en) 2024-12-01
CL2024003182A1 (es) 2025-03-14
TW202409285A (zh) 2024-03-01
KR20250012749A (ko) 2025-01-24
WO2023205767A3 (en) 2023-11-30
WO2023205767A2 (en) 2023-10-26
US20250269065A1 (en) 2025-08-28

Similar Documents

Publication Publication Date Title
CL2024003182A1 (es) Terapia génica con el atanógeno 3 asociado a linfoma de células b 2 (bag3) utilizando
MX2023001615A (es) Genoterapia con placofilina-2 (pkp2) mediante el uso de vector de aav.
MX2023000994A (es) Genoterapia con csrp3 (proteina 3 rica en cisteina y glicina).
JP2020063263A5 (OSRAM)
PE20250736A1 (es) Terapia genica para tratar la hemofilia a
IL275880B1 (en) Gene therapy for limb-girdle muscular dystrophy type 2c
JP2019513389A5 (OSRAM)
CO2022017959A2 (es) Composiciones para la reducción específica de la expresión transgénica en drg.
BR112023001418A2 (pt) Vetor viral adeno-associado para expressão de glut1 e seus usos
CO2024001148A2 (es) Casetes de expresión optimizada para genoterapia
CL2021002236A1 (es) Inserción de vectores de virus adeno-asociados de b-sarcoglicano y el tratamiento de distrofia muscular
CL2023001876A1 (es) Proteínas de la cápside viral con especificidad para las células del tejido cardiaco
CL2024002376A1 (es) Vector adenoasociado para la expresión de glut1 y los usos del mismo
WO2023108029A3 (en) Junctophilin-2 (jph2) gene therapy using aav vector
CL2021002172A1 (es) Virus adenoasociado recombinante para el tratamiento de la neurodegeneración de inicio en adultos asociada a grn.
WO2021243085A3 (en) Adeno-associated virus virions with variant capsids and methods of use thereof
AR122289A1 (es) Terapia genética cardíaca con aav para la cardiomiopatía en humanos
CO2022015313A2 (es) Variantes de cápsides de aav y usos de los mismos
AR129033A1 (es) Cápsides para terapia con el gen de placofilina-2
Phillips Gene, stem cell, and future therapies for orphan diseases
WO2023183583A3 (en) Adeno-associated virus compositions having increased heart enrichment
JP2024545183A5 (OSRAM)
AR125143A1 (es) Terapias génicas para la deficiencia de la 21-hidroxilasa
CO2021011090A2 (es) Composiciones útiles en el tratamiento de la enfermedad de krabbe
MX2025011409A (es) Plataforma de complejo modificado de virus adeno-asociado con tasa de expresión de los genes cargados mejorada y genotoxicidad reducida