[go: up one dir, main page]

MX2021010301A - Composiciones y métodos para el tratamiento de laminopatías. - Google Patents

Composiciones y métodos para el tratamiento de laminopatías.

Info

Publication number
MX2021010301A
MX2021010301A MX2021010301A MX2021010301A MX2021010301A MX 2021010301 A MX2021010301 A MX 2021010301A MX 2021010301 A MX2021010301 A MX 2021010301A MX 2021010301 A MX2021010301 A MX 2021010301A MX 2021010301 A MX2021010301 A MX 2021010301A
Authority
MX
Mexico
Prior art keywords
laminopathies
methods
compositions
treatment
disclosure provides
Prior art date
Application number
MX2021010301A
Other languages
English (en)
Inventor
Kartik Ramamoorthi
Stephanie Tagliatela
Anne Tanenhaus
Sirika Wood
Original Assignee
Encoded Therapeutics Inc
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 Encoded Therapeutics Inc filed Critical Encoded Therapeutics Inc
Publication of MX2021010301A publication Critical patent/MX2021010301A/es

Links

Classifications

    • 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
    • A01K67/00—Rearing or breeding animals, not otherwise provided for; New or modified breeds of animals
    • A01K67/027—New or modified breeds of vertebrates
    • A01K67/0275—Genetically modified vertebrates, e.g. transgenic
    • A01K67/0276—Knock-out vertebrates
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • 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
    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • 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
    • 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
    • C07K14/78—Connective tissue peptides, e.g. collagen, elastin, laminin, fibronectin, vitronectin or cold insoluble globulin [CIG]
    • 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
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • A01K2217/077—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out heterozygous knock out animals displaying phenotype
    • 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
    • A01K2267/0375—Animal model for cardiovascular 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
    • 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

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)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biophysics (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Toxicology (AREA)
  • Plant Pathology (AREA)
  • Environmental Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Animal Husbandry (AREA)
  • Biodiversity & Conservation Biology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Neurology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Immunology (AREA)

Abstract

La presente divulgación proporciona composiciones y métodos para tratar, prevenir o inhibir laminopatías. En un aspecto, la divulgación proporciona constructos de ácidos nucleicos y/o vectores que comprenden una secuencia de nucleótidos que codifica la lámina A y/o la lámina C.
MX2021010301A 2019-02-28 2020-02-28 Composiciones y métodos para el tratamiento de laminopatías. MX2021010301A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962812021P 2019-02-28 2019-02-28
PCT/US2020/020520 WO2020176896A1 (en) 2019-02-28 2020-02-28 Compositions and methods for treating laminopathies

Publications (1)

Publication Number Publication Date
MX2021010301A true MX2021010301A (es) 2021-11-12

Family

ID=72238989

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2021010301A MX2021010301A (es) 2019-02-28 2020-02-28 Composiciones y métodos para el tratamiento de laminopatías.

Country Status (17)

Country Link
US (1) US12357707B2 (es)
EP (1) EP3931336A4 (es)
JP (1) JP2022522196A (es)
KR (1) KR20210144696A (es)
CN (1) CN113840927A (es)
AU (1) AU2020229382A1 (es)
BR (1) BR112021016783A2 (es)
CA (1) CA3131776A1 (es)
CL (1) CL2021002262A1 (es)
CO (1) CO2021012310A2 (es)
EA (1) EA202192376A1 (es)
IL (1) IL285886A (es)
MA (1) MA55087A (es)
MX (1) MX2021010301A (es)
SG (1) SG11202109362XA (es)
TW (1) TW202045725A (es)
WO (1) WO2020176896A1 (es)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018213786A1 (en) * 2017-05-19 2018-11-22 Encoded Therapeutics, Inc. High activity regulatory elements
BR112020010883A2 (pt) 2017-12-01 2020-11-10 Encoded Therapeutics, Inc. proteínas de ligação a dna engenheiradas
KR20230124979A (ko) * 2020-12-23 2023-08-28 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 심장 유전자 요법을 위한 벡터의 증가된 패키징 효율
GB202100311D0 (en) * 2021-01-11 2021-02-24 Univ Court Of The Univ Of Aberdeen Treatment for Lipodystrophy
US20220362319A1 (en) * 2021-05-12 2022-11-17 PPM Labs LLC Cannabinoid Concentration Process
EP4198053A1 (en) * 2021-12-15 2023-06-21 Universitätsklinikum Hamburg-Eppendorf Lmna gene expression for treatment of laminopathies
WO2025075551A1 (en) * 2023-10-06 2025-04-10 National University Of Singapore Method of treating lmna dilated cardiomyopathy

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2004035753A2 (en) * 2002-10-18 2004-04-29 The Government Of The United States Of America As Represented By The Secretary Of The Department Of Health And Human Services Lmna gene and its involvement in hutchinson-gilford progeria syndrome (hgps) and arteriosclerosis
US7838531B2 (en) * 2002-10-18 2010-11-23 The United States Of America As Represented By The Department Of Health And Human Services Farnesyltransferase inhibitors for treatment of laminopathies, cellular aging and atherosclerosis
WO2007047913A2 (en) 2005-10-20 2007-04-26 Isis Pharmaceuticals, Inc Compositions and methods for modulation of lmna expression
SG11201404956PA (en) 2012-02-17 2014-09-26 Philadelphia Children Hospital Aav vector compositions and methods for gene transfer to cells, organs and tissues
ES2953614T3 (es) * 2018-01-19 2023-11-14 Agency Science Tech & Res Alteración del complejo linc para tratar la laminopatía
GB201902068D0 (en) * 2019-02-14 2019-04-03 Cambridge Entpr Ltd Methods of reactivating proliferative capacity non-proliferative tissues

Also Published As

Publication number Publication date
BR112021016783A2 (pt) 2021-11-09
SG11202109362XA (en) 2021-09-29
JP2022522196A (ja) 2022-04-14
MA55087A (fr) 2022-01-05
EP3931336A1 (en) 2022-01-05
CN113840927A (zh) 2021-12-24
AU2020229382A1 (en) 2021-09-16
CL2021002262A1 (es) 2022-05-27
CA3131776A1 (en) 2020-09-03
CO2021012310A2 (es) 2021-09-30
US12357707B2 (en) 2025-07-15
KR20210144696A (ko) 2021-11-30
EA202192376A1 (ru) 2021-12-27
EP3931336A4 (en) 2023-01-04
TW202045725A (zh) 2020-12-16
US20220193264A1 (en) 2022-06-23
WO2020176896A1 (en) 2020-09-03
IL285886A (en) 2021-10-31

Similar Documents

Publication Publication Date Title
MX2021010301A (es) Composiciones y métodos para el tratamiento de laminopatías.
CY1123697T1 (el) Θεραπεια της amd με χρηση της παραλλαγης aav2 με αφλιβερσεπτη
MX2025007819A (es) Composiciones para usarse en el tratamiento de un sujeto que tiene hemofilia
CL2023002870A1 (es) Composiciones de arni contra el angiotensinógeno (agt) y métodos para su uso
CY1125152T1 (el) Συνθεσεις και μεθοδοι για τη μειωση της νεοαγγειωσης του οφθαλμου
BR112018008326A2 (pt) composições e métodos para o tratamento de câncer
WO2016106401A3 (en) Rna agents for p21 gene modulation
MY192888A (en) Dna alkylating agents
BR112019017403A2 (pt) composições e métodos para o tratamento de câncer
BR112016022855A2 (pt) composições e métodos para modular a expressão de pkk
NZ733882A (en) Compositions for modulating c9orf72 expression
MX2018009325A (es) Compuestos y metodos para tratar enfermedades mediadas por el acido ribonucleico (arn).
EA201792080A1 (ru) Гуманизированные анти-c1s антитела и способы их применения
BR112017015627A2 (pt) Compostos de piperidina ureia substituída com 4- metilsulfonila para o tratamento de cardiomiopatia dilatada (dcm)
BR112016020472A2 (pt) composições para modulação de expressão de ata-xina 2
MX394221B (es) Metodos de reduccion de olor.
MX378051B (es) Composición de cemento compatibilizada para el tratamiento de una formación subterránea.
BR112018006986A2 (pt) métodos e composições para contrações musculares indesejadas ou anormais
MX388737B (es) Oligonucleótidos terapéuticos.
MX375277B (es) Métodos para tratar el síndrome de prader-willi
WO2018035199A8 (en) Sea lice vaccine
MX2020003806A (es) Anticuerpos monoclonales de adn que se dirigen a ctla-4 para el tratamiento y prevención del cáncer.
AR101146A1 (es) Proteína con actividad xilanasa
AR107725A1 (es) Composiciones, organismos, sistemas y métodos de cítricos resistentes a patógenos
MX2018012545A (es) Reactivos para el tratamiento de distrofia muscular oculofaríngea (opmd) y su uso.