CO2020010270A2 - Vector recombinante para terapia génica de la distrofia muscular de cinturas tipo 2c - Google Patents

Vector recombinante para terapia génica de la distrofia muscular de cinturas tipo 2c

Info

Publication number
CO2020010270A2
CO2020010270A2 CONC2020/0010270A CO2020010270A CO2020010270A2 CO 2020010270 A2 CO2020010270 A2 CO 2020010270A2 CO 2020010270 A CO2020010270 A CO 2020010270A CO 2020010270 A2 CO2020010270 A2 CO 2020010270A2
Authority
CO
Colombia
Prior art keywords
gene therapy
muscular dystrophy
recombinant vector
dystrophy type
girdle muscular
Prior art date
Application number
CONC2020/0010270A
Other languages
English (en)
Inventor
Louise R Rodino-Klapac
Original Assignee
Res Inst Nationwide Childrens Hospital
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 Res Inst Nationwide Childrens Hospital filed Critical Res Inst Nationwide Childrens Hospital
Publication of CO2020010270A2 publication Critical patent/CO2020010270A2/es

Links

Classifications

    • 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
    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • 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
    • C07K14/46—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
    • C07K14/4707—Muscular dystrophy
    • 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; CARE OF BIRDS, FISHES, INSECTS; 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; CARE OF BIRDS, FISHES, INSECTS; 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; CARE OF BIRDS, FISHES, INSECTS; 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
    • 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
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
    • 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
    • C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA

Abstract

La divulgación se relaciona con vectores de terapia de gen, tal como vectores AAV, que comprenden un polinucleótido que codifica γ-sarcoglicano (SGCG) y métodos para usar dichos vectores de terapia de gen para tratar sujetos que sufren de una distrofia muscular, por ejemplo, distrofia de cinturas tipo 2C (LGMD2C).
CONC2020/0010270A 2018-01-31 2020-08-31 Vector recombinante para terapia génica de la distrofia muscular de cinturas tipo 2c CO2020010270A2 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862624616P 2018-01-31 2018-01-31
PCT/US2019/015779 WO2019152474A1 (en) 2018-01-31 2019-01-30 Gene therapy for limb-girdle muscular dystrophy type 2c

Publications (1)

Publication Number Publication Date
CO2020010270A2 true CO2020010270A2 (es) 2020-11-20

Family

ID=67478478

Family Applications (1)

Application Number Title Priority Date Filing Date
CONC2020/0010270A CO2020010270A2 (es) 2018-01-31 2020-08-31 Vector recombinante para terapia génica de la distrofia muscular de cinturas tipo 2c

Country Status (17)

Country Link
US (1) US20200360534A1 (es)
EP (1) EP3746100A4 (es)
JP (2) JP7361701B2 (es)
KR (1) KR20200115585A (es)
CN (1) CN111818946A (es)
AR (1) AR114350A1 (es)
AU (1) AU2019216257A1 (es)
BR (1) BR112020015173A2 (es)
CA (1) CA3089080A1 (es)
CL (2) CL2020001948A1 (es)
CO (1) CO2020010270A2 (es)
EA (1) EA202091739A1 (es)
IL (1) IL275880A (es)
MX (1) MX2020007876A (es)
SG (1) SG11202006722RA (es)
TW (1) TWI815856B (es)
WO (1) WO2019152474A1 (es)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX2020008086A (es) 2018-01-30 2021-02-15 Apnimed Inc Delaware Metodos y composiciones para tratar la apnea del sue?o.
US20220411821A1 (en) * 2019-10-28 2022-12-29 University Of Florida Research Foundation, Incorporated Gene therapy vectors
JP2023506030A (ja) * 2019-12-16 2023-02-14 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル ジストロフィン関連タンパク質複合体（ｄａｐｃ）を回復および維持するための組成物および方法
CN116348149A (zh) * 2020-06-15 2023-06-27 全国儿童医院研究所 用于肌营养不良症的腺相关病毒载体递送
CN115996758A (zh) 2020-06-19 2023-04-21 吉尼松公司 使sgcg在肌肉和心脏中充分表达的基因治疗表达系统
TW202227634A (zh) 2020-09-08 2022-07-16 美商薩羅塔治療公司 表現γ—肌聚醣之腺相關病毒載體之全身性遞送及肌肉失養症之治療
JP2023081369A (ja) * 2021-11-30 2023-06-09 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル 自己相補的アデノ随伴ウイルスベクター及び筋ジストロフィーの治療におけるその使用
EP4198134A1 (en) 2021-12-16 2023-06-21 Genethon Gamma-sarcoglycan gene transfer increase using modified itr sequences

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6262035B1 (en) * 1998-10-01 2001-07-17 University Of Iowa Research Foundation Gene replacement therapy for muscular dystrophy
US20100247495A1 (en) * 2009-03-30 2010-09-30 Tom Ichim Treatment of Muscular Dystrophy
US9303079B2 (en) * 2012-04-02 2016-04-05 Moderna Therapeutics, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
AU2013243948A1 (en) * 2012-04-02 2014-10-30 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins associated with human disease
WO2017191274A2 (en) 2016-05-04 2017-11-09 Curevac Ag Rna encoding a therapeutic protein

Also Published As

Publication number Publication date
EP3746100A4 (en) 2021-11-17
SG11202006722RA (en) 2020-08-28
AU2019216257A1 (en) 2020-07-23
TWI815856B (zh) 2023-09-21
JP2021512072A (ja) 2021-05-13
CL2020001948A1 (es) 2020-10-09
BR112020015173A2 (pt) 2020-12-29
AR114350A1 (es) 2020-08-26
EP3746100A1 (en) 2020-12-09
MX2020007876A (es) 2020-12-03
JP7361701B2 (ja) 2023-10-16
WO2019152474A9 (en) 2020-08-27
IL275880A (en) 2020-08-31
CL2021003097A1 (es) 2022-07-15
US20200360534A1 (en) 2020-11-19
CN111818946A (zh) 2020-10-23
WO2019152474A1 (en) 2019-08-08
TW201934751A (zh) 2019-09-01
KR20200115585A (ko) 2020-10-07
JP2023171900A (ja) 2023-12-05
EA202091739A1 (ru) 2021-01-14
CA3089080A1 (en) 2019-08-08

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