CO2021005083A2 - Distrofinas miniaturizadas y usos de las mismas - Google Patents

Distrofinas miniaturizadas y usos de las mismas

Info

Publication number
CO2021005083A2
CO2021005083A2 CONC2021/0005083A CO2021005083A CO2021005083A2 CO 2021005083 A2 CO2021005083 A2 CO 2021005083A2 CO 2021005083 A CO2021005083 A CO 2021005083A CO 2021005083 A2 CO2021005083 A2 CO 2021005083A2
Authority
CO
Colombia
Prior art keywords
miniaturized
dystrophins
miniaturized dystrophins
methods
miniaturized dystrophin
Prior art date
Application number
CONC2021/0005083A
Other languages
English (en)
Inventor
Glen Banks
Jonathan Harry Davis
Paul Charles Levesque
Original Assignee
Bristol Myers Squibb Co
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 Bristol Myers Squibb Co filed Critical Bristol Myers Squibb Co
Publication of CO2021005083A2 publication Critical patent/CO2021005083A2/es

Links

Classifications

    • 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
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • 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
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/62—DNA sequences coding for fusion proteins
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • 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)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Immunology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)

Abstract

En el presente documento se describen moléculas de ácido nucleico, polipéptidos, células, vectores y composiciones farmacéuticas relacionadas con distrofina miniaturizada. También se describen métodos de producción y métodos de uso terapéutico de la distrofina miniaturizada.
CONC2021/0005083A 2018-10-24 2021-04-21 Distrofinas miniaturizadas y usos de las mismas CO2021005083A2 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862749982P 2018-10-24 2018-10-24
PCT/US2019/057855 WO2020086844A1 (en) 2018-10-24 2019-10-24 Miniaturized dystrophins and uses thereof

Publications (1)

Publication Number Publication Date
CO2021005083A2 true CO2021005083A2 (es) 2021-04-30

Family

ID=68582390

Family Applications (1)

Application Number Title Priority Date Filing Date
CONC2021/0005083A CO2021005083A2 (es) 2018-10-24 2021-04-21 Distrofinas miniaturizadas y usos de las mismas

Country Status (16)

Country Link
US (1) US20210403947A1 (es)
EP (1) EP3870202A1 (es)
JP (1) JP2022505816A (es)
KR (1) KR20210082193A (es)
CN (1) CN113613668A (es)
AU (1) AU2019368307A1 (es)
BR (1) BR112021007480A2 (es)
CA (1) CA3117738A1 (es)
CL (1) CL2021000994A1 (es)
CO (1) CO2021005083A2 (es)
EA (1) EA202191123A1 (es)
IL (1) IL282547A (es)
MX (1) MX2021004688A (es)
PE (1) PE20211110A1 (es)
SG (1) SG11202104051VA (es)
WO (1) WO2020086844A1 (es)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2021222332A1 (en) 2020-04-29 2021-11-04 Bristol-Myers Squibb Company Miniaturized dystrophins having spectrin fusion domains and uses thereof
BR112023001648A2 (pt) 2020-07-27 2023-04-04 Anjarium Biosciences Ag Moléculas de dna de fita dupla, veículo de entrega e método para preparar uma molécula de dna com extremidade em grampo
US20240003898A1 (en) * 2020-10-30 2024-01-04 Pfizer Inc. Methods for measuring dystrophin in tissue samples
EP4337267A1 (en) * 2021-05-11 2024-03-20 RegenxBio Inc. Treatment of duchenne muscular dystrophy and combinations thereof

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4522811A (en) 1982-07-08 1985-06-11 Syntex (U.S.A.) Inc. Serial injection of muramyldipeptides and liposomes enhances the anti-infective activity of muramyldipeptides
US4683195A (en) 1986-01-30 1987-07-28 Cetus Corporation Process for amplifying, detecting, and/or-cloning nucleic acid sequences
EP1366160B1 (en) 2000-10-06 2008-07-09 The Regents Of The University Of Michigan Mini-dystrophin nucleic acid and peptide sequences
MXPA05004860A (es) 2002-11-04 2005-08-18 Advisys Inc Promotores musculares sinteticos con actividades que exceden las secuencias reguladoras de origen natural en las celulas cardiacas.
CA2972811A1 (en) * 2015-01-16 2016-07-21 University Of Washington Novel micro-dystrophins and related methods of use
CA2971303A1 (en) * 2016-06-21 2017-12-21 Bamboo Therapeutics, Inc. Optimized mini-dystrophin genes and expression cassettes and their use

Also Published As

Publication number Publication date
EP3870202A1 (en) 2021-09-01
KR20210082193A (ko) 2021-07-02
IL282547A (en) 2021-06-30
SG11202104051VA (en) 2021-05-28
CN113613668A (zh) 2021-11-05
JP2022505816A (ja) 2022-01-14
MX2021004688A (es) 2021-06-04
CL2021000994A1 (es) 2021-09-20
AU2019368307A1 (en) 2021-06-10
US20210403947A1 (en) 2021-12-30
CA3117738A1 (en) 2020-04-30
PE20211110A1 (es) 2021-06-17
EA202191123A1 (ru) 2022-03-29
WO2020086844A1 (en) 2020-04-30
BR112021007480A2 (pt) 2021-08-10

Similar Documents

Publication Publication Date Title
CO2021005083A2 (es) Distrofinas miniaturizadas y usos de las mismas
ECSP19029982A (es) Moduladores de calpaínas y usos terapeuticos de los mismos
PE20190563A1 (es) Genes de mini-distrofina optimizados y casetes de expresion y su uso
CL2022000300A1 (es) Muteínas de interleucina 21 y sus usos para tratar tumores sólidos (divisional de la solicitud no. 202000252)
CL2018003153A1 (es) Moléculas de anticuerpo para el tratamiento del cáncer.
BR112017024384A2 (pt) bactérias modificadas para reduzir hiperfenilalaninemia
CO2018003477A2 (es) Anticuerpos anti-pd1 y métodos de uso
CL2018002852A1 (es) Producción celular de ácido ribonucleico
CL2018000622A1 (es) Acetamida tienodiazepinas de triazol y sus usos relacionados.
BR112016022814A8 (pt) composição farmacêutica e seu método de preparação, ácido nucleico, e dispositivo médico ou kit médico
BR112018070934A2 (pt) proteínas imunomoduladoras variantes ligantes de icos e usos das mesmas
EA201792420A1 (ru) Композиции и способы перепрограммирования tcr с помощью гибридных белков
BR112017023975A2 (pt) variantes de alfa-amilase e polinucleotídeos codificando as mesmas
MX2021010668A (es) Proteinas de fusion de citoquinas.
EA201792500A1 (ru) Aav-опосредованная экспрессия антител против гриппа и способы их использования
CR20140200A (es) Formas solidas de 3-(5-amino-2-metil-4-oxo-ah-quinazolin-3-il)-piperidin-2,6-diona, y sus composiciones farmaceuticas y sus usos
UY36176A (es) Formas cristalinas de (2r, 5s, 13ar)-8-hidroxi-7,9- dioxo- n-(2,4,6 - trifluorobencil)-2,3,4,5,7,9,13,13a -octahidro -2,5- metanopirido[1?,2?,4,5]pirazino[2,1-b][1,3]oxazapina- 10-carboxamida
BR112018012262A2 (pt) composições de interleucina-15 e seus usos
WO2015195769A3 (en) Optogenetic probes for measuring membrane potential
MX2023010365A (es) Proteina liofilizada estable a temperatura ambiente.
UY34663A (es) Acido nucleico y proteina recombinante del gen de la endotoxina axmi345, vectores, células, composiciones y métodos para proteger plantas
BR112017003219A2 (pt) formulação de acetato de abiraterona e métodos de uso
EA201791500A1 (ru) Циклопропанкарбоксамидные модуляторы муковисцидозного трансмембранного регулятора проводимости
EA202090014A1 (ru) Гетероароматические соединения в качестве ингибиторов ванина
MX2020004939A (es) Fabricacion de composiciones de celulas islote y metodos de uso de las mismas.