BR112022021468A2 - Genes gla otimizados por códon e usos dos mesmos - Google Patents

Genes gla otimizados por códon e usos dos mesmos

Info

Publication number
BR112022021468A2
BR112022021468A2 BR112022021468A BR112022021468A BR112022021468A2 BR 112022021468 A2 BR112022021468 A2 BR 112022021468A2 BR 112022021468 A BR112022021468 A BR 112022021468A BR 112022021468 A BR112022021468 A BR 112022021468A BR 112022021468 A2 BR112022021468 A2 BR 112022021468A2
Authority
BR
Brazil
Prior art keywords
codon
optimized
genes
galactosidase
gla
Prior art date
Application number
BR112022021468A
Other languages
English (en)
Inventor
H Kirn David
Kotterman Melissa
Francis Peter
Schaffer David
Szymanski Paul
Whittlesey Kevin
Original Assignee
4D Molecular 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 4D Molecular Therapeutics Inc filed Critical 4D Molecular Therapeutics Inc
Publication of BR112022021468A2 publication Critical patent/BR112022021468A2/pt

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
    • 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
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • 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
    • 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
    • A61K48/0083—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 administration regime
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • 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
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • C12N9/2465—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1) acting on alpha-galactose-glycoside bonds, e.g. alpha-galactosidase (3.2.1.22)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01022—Alpha-galactosidase (3.2.1.22)
    • 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
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • 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
    • C12N2840/00—Vectors comprising a special translation-regulating system

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Dermatology (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)

Abstract

GENES GLA OTIMIZADOS POR CÓDON E USOS DOS MESMOS. A presente revelação fornece sequências nucleotídicas otimizadas de códon que codificam alfa-galactosidase A humana, vetores, e células hospedeiras compreendendo sequências de alfa-galactosidase A otimizadas de códon, e métodos de tratamento de transtornos tal como doença de Fabry compreendendo administrar ao indivíduo uma sequência otimizada de códon que codifica alfa-galactosidase A humana.
BR112022021468A 2020-04-27 2021-04-26 Genes gla otimizados por códon e usos dos mesmos BR112022021468A2 (pt)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063016207P 2020-04-27 2020-04-27
US202063114195P 2020-11-16 2020-11-16
PCT/US2021/029146 WO2021222094A1 (en) 2020-04-27 2021-04-26 Codon optimized gla genes and uses thereof

Publications (1)

Publication Number Publication Date
BR112022021468A2 true BR112022021468A2 (pt) 2022-12-13

Family

ID=78221860

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112022021468A BR112022021468A2 (pt) 2020-04-27 2021-04-26 Genes gla otimizados por códon e usos dos mesmos

Country Status (13)

Country Link
US (3) US11198858B2 (pt)
EP (1) EP4103211A4 (pt)
JP (2) JP7408838B2 (pt)
KR (1) KR20230026986A (pt)
CN (1) CN115515613A (pt)
AU (2) AU2021263563B2 (pt)
BR (1) BR112022021468A2 (pt)
CA (1) CA3175905A1 (pt)
IL (1) IL297404A (pt)
MX (1) MX2022013411A (pt)
TW (1) TWI769795B (pt)
WO (1) WO2021222094A1 (pt)
ZA (1) ZA202210127B (pt)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CR20200165A (es) * 2017-09-20 2020-06-05 4D Molecular Therapeutics Inc Variantes de cápsides de virus adenoasociados y métodos de uso de estas
WO2023102517A1 (en) * 2021-12-02 2023-06-08 The Trustees Of The University Of Pennsylvania Compositions and methods for treatment of fabry disease
CN116622750B (zh) * 2023-06-01 2024-05-31 上海勉亦生物科技有限公司 优化的人法布里转基因表达盒及其用途

Family Cites Families (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2002064799A2 (en) 1999-09-28 2002-08-22 Transkaryotic Therapies, Inc. Optimized messenger rna
WO2004083441A2 (en) 2003-03-19 2004-09-30 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Random peptide library displayed on aav vectors
US9233131B2 (en) 2003-06-30 2016-01-12 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
EP2016174A2 (en) 2006-04-28 2009-01-21 The Trustees of the University of Pennsylvania Modified aav vectors having reduced capsid immunogenicity and use thereof
WO2008143354A1 (ja) * 2007-05-18 2008-11-27 Tokyo Metropolitan Organization For Medical Research 酵素補充療法用医薬組成物
WO2012145601A2 (en) 2011-04-22 2012-10-26 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
EP3003391B1 (en) 2013-05-31 2021-09-22 The Regents of The University of California Adeno-associated virus variants and methods of use thereof
EP3062884B1 (en) 2013-10-29 2020-12-02 President and Fellows of Harvard College Compositions for use in the treatment of retinitis pigmentosa
SG11201607738WA (en) 2014-03-17 2016-10-28 Adverum Biotechnologies Inc Compositions and methods for enhanced gene expression in cone cells
BR112017018846A2 (pt) 2015-03-02 2018-07-31 Adverum Biotechnologies, Inc. composições e métodos para entrega intravítrea de polinucleotídeos a cones retinianos.
US10883117B2 (en) 2015-03-24 2021-01-05 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
GB201508025D0 (en) * 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
EP3384034B1 (en) 2015-12-02 2020-07-08 The Board of Trustees of the Leland Stanford Junior University Novel recombinant adeno-associated virus capsids with enhanced human skeletal muscle tropism
EP3452101A2 (en) 2016-05-04 2019-03-13 CureVac AG Rna encoding a therapeutic protein
CR20180589A (es) 2016-05-13 2019-02-27 4D Molecular Therapeutics Inc Cápsulas variantes de virus adenoasociados y métodos de uso de estas
JP7114485B2 (ja) * 2016-05-18 2022-08-08 モデルナティエックス インコーポレイテッド ファブリー病の治療のためのα－ガラクトシダーゼＡをコードするポリヌクレオチド
CR20200165A (es) 2017-09-20 2020-06-05 4D Molecular Therapeutics Inc Variantes de cápsides de virus adenoasociados y métodos de uso de estas
AU2019391042A1 (en) * 2018-12-05 2021-06-10 Abeona Therapeutics Inc. Recombinant adeno-associated viral vector for gene delivery
AU2020248099A1 (en) * 2019-03-27 2021-10-07 Sigilon Therapeutics, Inc. Compositions, devices and methods for treating Fabry disease

Also Published As

Publication number Publication date
TW202206595A (zh) 2022-02-16
US11198858B2 (en) 2021-12-14
EP4103211A4 (en) 2023-08-23
CA3175905A1 (en) 2021-11-04
TWI769795B (zh) 2022-07-01
EP4103211A1 (en) 2022-12-21
KR20230026986A (ko) 2023-02-27
JP7408838B2 (ja) 2024-01-05
AU2023200818A1 (en) 2023-03-09
US20230190964A1 (en) 2023-06-22
ZA202210127B (en) 2024-01-31
AU2021263563B2 (en) 2023-02-02
AU2021263563A1 (en) 2022-10-20
US20240018498A1 (en) 2024-01-18
US11802278B2 (en) 2023-10-31
CN115515613A (zh) 2022-12-23
US20210332341A1 (en) 2021-10-28
IL297404A (en) 2022-12-01
JP2023156511A (ja) 2023-10-24
JP2023516224A (ja) 2023-04-18
MX2022013411A (es) 2022-11-14
WO2021222094A1 (en) 2021-11-04

Similar Documents

Publication Publication Date Title
BR112022021468A2 (pt) Genes gla otimizados por códon e usos dos mesmos
MX2020003965A (es) Terapias genicas para los trastornos lisosomales.
SA519401435B1 (ar) مركبات حلقية ثنائية النيوكليوتيد
BR112018013808A2 (pt) dinucleotídeos cíclicos para o tratamento de condições associadas à atividade de sting, tal como câncer
BR112018015659A2 (pt) genes otimizados do fator viii
BR112018068391A2 (pt) sistema e métodos para produzir uma proteína, célula inoculada, construção de rna para expressar a replicase de alfavírus e dna
BRPI0808104A8 (pt) Métodos para preparar e para produzir um polipeptídeo, polipeptídeo, sequência de ácido nucleico isolada, construção de ácido nucleico, vetor de expressão recombinante, e, célula hospedeira recombinante
MX2020004005A (es) Terapias genicas para los trastornos lisosomales.
MX2020003557A (es) Terapias genicas para los trastornos lisosomales.
AR100560A1 (es) Inhibición o regulación por disminución de la glucógeno sintasa mediante la creación de codones de terminación prematuros utilizando oligonucléotidos antisentido
BR112017020445A2 (pt) polipeptídeo de fusão, molécula de ácido nucleico, célula hospedeira, método de produção de um polipeptídeo de fusão, uso do polipeptídeo de fusão, método para ativar as vias de sinalização, método para coestimular células, método para induzir a proliferação de linfócitos e método para direcionar o agrupamento de cd137
AR088746A1 (es) Proteinas variantes de la toxina activa contra las larvas del gusano de la raiz del maiz del oeste (axmi205)
CO2022003552A2 (es) Proteínas de fijación multiespecíficas para tratamiento contra el cáncer
MX2018012695A (es) Oligomeros antisentido y metodos de uso de los mismos para tratar enfermedades relacionadas con el gen alpha-glucosidasa acido.
BR112014002027A8 (pt) Gene pesticida axmi279 e métodos para a sua utilização
BR112013032605A2 (pt) variantes do promotor do gene gap codificador de glicerinaldeido-3-fosfato-de-hidrogenase
MX2020007011A (es) Vectores de vaccinia modificados.
CL2021001490A1 (es) Constructos de iarn modificados químicamente y usos de estos
BR112023003834A2 (pt) Genes rpgrorf15 otimizados por códon e usos dos mesmos
EA201170917A1 (ru) Ген, кодирующий мутантную глюкокиназу человека, кодируемый им фермент, рекомбинантные векторы и хозяева, фармацевтические композиции и их применения, способы лечения и предупреждения болезней
CL2020001810A1 (es) Oligonucleótidos antisentido para alfa-sinucleína y usos de los mismos.
AR092152A1 (es) Uso de una region no traducida de maiz para expresion transgenica en plantas
CO2023005720A2 (es) Métodos y composiciones para terapia génica con placofilina-2
MX2021006253A (es) Terapias génicas para enfermedad neurodegenerativa.
GB201202561D0 (en) Treatment of skin disorders