MX2024000748A - Terapia génica de kcnv2. - Google Patents

Terapia génica de kcnv2.

Info

Publication number
MX2024000748A
MX2024000748A MX2024000748A MX2024000748A MX2024000748A MX 2024000748 A MX2024000748 A MX 2024000748A MX 2024000748 A MX2024000748 A MX 2024000748A MX 2024000748 A MX2024000748 A MX 2024000748A MX 2024000748 A MX2024000748 A MX 2024000748A
Authority
MX
Mexico
Prior art keywords
kcnv2
gene therapy
vectors
disclosed
subject
Prior art date
Application number
MX2024000748A
Other languages
English (en)
Spanish (es)
Inventor
Anastasios Georgiadis
Original Assignee
Meiragtx Uk Ii Ltd
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 Meiragtx Uk Ii Ltd filed Critical Meiragtx Uk Ii Ltd
Publication of MX2024000748A publication Critical patent/MX2024000748A/es

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • A61K48/0058Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/177Receptors; Cell surface antigens; Cell surface determinants
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • A61P27/02Ophthalmic agents
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705Receptors; Cell surface antigens; Cell surface determinants
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00Genetically modified animals
    • A01K2217/07Animals genetically altered by homologous recombination
    • A01K2217/075Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2227/00Animals characterised by species
    • A01K2227/10Mammal
    • A01K2227/105Murine
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00Nucleic acids vectors
    • C12N2800/22Vectors comprising a coding region that has been codon optimised for expression in a respective host
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/008Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/48Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/50Vector 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)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Wood Science & Technology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Cell Biology (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Toxicology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
MX2024000748A 2021-07-14 2022-07-13 Terapia génica de kcnv2. MX2024000748A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202163221879P 2021-07-14 2021-07-14
PCT/IB2022/056457 WO2023285986A1 (en) 2021-07-14 2022-07-13 Kcnv2 gene therapy

Publications (1)

Publication Number Publication Date
MX2024000748A true MX2024000748A (es) 2024-03-15

Family

ID=82932641

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2024000748A MX2024000748A (es) 2021-07-14 2022-07-13 Terapia génica de kcnv2.

Country Status (10)

Country Link
US (1) US20240307559A1 (https=)
EP (1) EP4370538A1 (https=)
JP (1) JP2024525742A (https=)
KR (1) KR20240052746A (https=)
CN (1) CN118019757A (https=)
AU (1) AU2022311329A1 (https=)
CA (1) CA3225080A1 (https=)
IL (1) IL310018A (https=)
MX (1) MX2024000748A (https=)
WO (1) WO2023285986A1 (https=)

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE4345297A1 (de) 1993-11-11 1995-09-28 Nsm Ag Geldspielgerät
US5856152A (en) 1994-10-28 1999-01-05 The Trustees Of The University Of Pennsylvania Hybrid adenovirus-AAV vector and methods of use therefor
CA2346262A1 (en) 1998-09-17 2000-03-23 University Of Florida Methods for treatment of degenerative retinal diseases
MA44546B1 (fr) 2016-06-15 2021-03-31 Univ California Virus adéno-associés variants et procédés d'utilisation
SG11202000840YA (en) * 2017-07-31 2020-02-27 Reflection Biotechnologies Ltd Cellular models of and therapies for ocular diseases

Also Published As

Publication number Publication date
WO2023285986A1 (en) 2023-01-19
EP4370538A1 (en) 2024-05-22
CA3225080A1 (en) 2023-01-19
CN118019757A (zh) 2024-05-10
JP2024525742A (ja) 2024-07-12
IL310018A (en) 2024-03-01
US20240307559A1 (en) 2024-09-19
KR20240052746A (ko) 2024-04-23
AU2022311329A1 (en) 2024-02-29

Similar Documents

Publication Publication Date Title
Hsu et al. CRISPR technologies for stem cell engineering and regenerative medicine
Najafi et al. Therapeutic potentials of CRISPR-Cas genome editing technology in human viral infections
MY209860A (en) Gene therapy vectors for treating heart disease
WO2021163357A3 (en) Gene therapy vectors for treating heart disease
FI3795181T3 (fi) Amd:n hoito aav2-variantilla ja afliberseptillä
CY1119295T1 (el) Γονιδιακη θεραπεια για ασθενειες λυσοσωμικης αποθhκευσης
EP4378487A3 (en) Modified factor ix, and compositions, methods and uses for gene transfer to cells, organs and tissues
PH12020500239A1 (en) Factor viii (fviii) gene therapy methods
WO2022195074A3 (en) Gene therapy composition and treatment of right ventricular arrhythmogenic cardiomyopathy
WO2022035903A3 (en) Gene therapies for lysosomal disorders
WO2020142740A8 (en) Treatment of sjogren's disease with nuclease fusion proteins
MXPA02007448A (es) Sistema de suministro y metodo para terapia genetica.
MX2024000748A (es) Terapia génica de kcnv2.
Deng et al. Combination of ibrutinib and chemotherapy produced a durable remission in multiply relapsed diffuse large B-cell lymphoma leg type with mutant MYD88 and wildtype CD79
MX2023000945A (es) 55cepa c de prevotella histicola como terapia oral para enfermedades inflamatorias.
Chiam et al. Scedosporium prolificans endogenous endophthalmitis
MX2024000747A (es) Terapia génica con retgc.
MX2021015292A (es) Interacciones terapeuticas de leucometiltioninio.
RU2013130028A (ru) Новые производные оксадиазола в качестве модуляторов рецептора сфингозин 1-фосфата (s1p)
Ranzenigo et al. HIV cure-related research: where we stand and where we go
MX2023007029A (es) Masitinib para el tratamiento de la enfermedad de alzheimer.
Tae CRISPR, OMEGA, and Fanzor: Mixed Blessings of Genome Editing Technology
MX2026002137A (es) Proteina hibrida, secuencia nucleotidica hibrida, molecula de acido nucleico, vector para la expresion de proteina hibrida, uso de la proteina hibrida o secuencia nucleotidica, formulacion, proceso para la produccion de una formulacion y metodo para el tratamiento de lesiones y/o cancer causados por la infeccion por el virus del papiloma humano (hpv)
EA202192739A1 (ru) Генная терапия лизосомальных нарушений
Dau et al. CONCURRENT REPROGRAMMING AND GENE EDITING OF HUMAN FIBROBLASTS INTO INDUCED PLURIPOTENT STEM CELLS USING DUAL COLOR SINGLE-CYCLE MEASLES VECTORS