MX2020011514A - Construcciones para terapia genica y metodos para su uso. - Google Patents

Construcciones para terapia genica y metodos para su uso.

Info

Publication number
MX2020011514A
MX2020011514A MX2020011514A MX2020011514A MX2020011514A MX 2020011514 A MX2020011514 A MX 2020011514A MX 2020011514 A MX2020011514 A MX 2020011514A MX 2020011514 A MX2020011514 A MX 2020011514A MX 2020011514 A MX2020011514 A MX 2020011514A
Authority
MX
Mexico
Prior art keywords
methods
gene therapy
therapy constructs
constructs
improved
Prior art date
Application number
MX2020011514A
Other languages
English (en)
Inventor
Hung Do
Russell Gotschall
Steven Tuske
Ce Feng Liu
Original Assignee
Amicus 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 Amicus Therapeutics Inc filed Critical Amicus Therapeutics Inc
Publication of MX2020011514A publication Critical patent/MX2020011514A/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
    • 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
    • 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
    • C07K14/575—Hormones
    • C07K14/65—Insulin-like growth factors, i.e. somatomedins, e.g. IGF-1, IGF-2
    • 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
    • A61K48/0075—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
    • C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/38—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against protease inhibitors of peptide structure
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/40—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against enzymes
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K19/00—Hybrid peptides, i.e. peptides covalently bound to nucleic acids, or non-covalently bound protein-protein complexes
    • 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
    • C12N15/52—Genes encoding for enzymes or proenzymes
    • 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)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • 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)
    • 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)
    • C12Y302/0102—Alpha-glucosidase (3.2.1.20)
    • 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)
    • C12Y302/0105—Alpha-N-acetylglucosaminidase (3.2.1.50)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/90—Immunoglobulins specific features characterized by (pharmaco)kinetic aspects or by stability of the immunoglobulin
    • C07K2317/92—Affinity (KD), association rate (Ka), dissociation rate (Kd) or EC50 value
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/02—Fusion polypeptide containing a localisation/targetting motif containing a signal sequence
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/03—Fusion polypeptide containing a localisation/targetting motif containing a transmembrane segment
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/06—Fusion polypeptide containing a localisation/targetting motif containing a lysosomal/endosomal localisation signal
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/70—Fusion polypeptide containing domain for protein-protein interaction
    • C07K2319/74—Fusion polypeptide containing domain for protein-protein interaction containing a fusion for binding to a cell surface receptor
    • 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
    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/10—Vectors comprising a special translation-regulating system regulates levels of translation
    • C12N2840/105—Vectors comprising a special translation-regulating system regulates levels of translation enhancing translation
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/02—Thioester hydrolases (3.1.2)
    • C12Y301/02022—Palmitoyl-protein hydrolase (3.1.2.22)

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Immunology (AREA)
  • Virology (AREA)
  • Diabetes (AREA)
  • Endocrinology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (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)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

Se proporcionan en la presente vectores mejorados para terapia génica y métodos de uso, en algunas formas de realización, que comprenden secuencias para la expresión mejorada y la selección celular de una proteína terapéutica.
MX2020011514A 2018-04-30 2019-04-30 Construcciones para terapia genica y metodos para su uso. MX2020011514A (es)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201862664741P 2018-04-30 2018-04-30
US201862688640P 2018-06-22 2018-06-22
US201862744068P 2018-10-10 2018-10-10
PCT/US2019/030076 WO2019213180A1 (en) 2018-04-30 2019-04-30 Gene therapy constructs and methods of use

Publications (1)

Publication Number Publication Date
MX2020011514A true MX2020011514A (es) 2020-12-09

Family

ID=66669057

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2020011514A MX2020011514A (es) 2018-04-30 2019-04-30 Construcciones para terapia genica y metodos para su uso.

Country Status (16)

Country Link
US (3) US10874750B2 (es)
EP (1) EP3788064A1 (es)
JP (1) JP2021521851A (es)
KR (1) KR20210005154A (es)
CN (1) CN112513071A (es)
AU (1) AU2019261982A1 (es)
BR (1) BR112020021962A2 (es)
CA (1) CA3098674A1 (es)
CL (1) CL2020002809A1 (es)
CO (1) CO2020014729A2 (es)
MX (1) MX2020011514A (es)
PH (1) PH12020551818A1 (es)
SG (1) SG11202010533WA (es)
TW (2) TW202344691A (es)
WO (1) WO2019213180A1 (es)
ZA (1) ZA202006905B (es)

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SG11202010533WA (en) * 2018-04-30 2020-11-27 Amicus Therapeutics Inc Gene therapy constructs and methods of use
CN113631182A (zh) 2018-10-10 2021-11-09 阿米库斯治疗学公司 二硫键稳定的多肽组合物和使用方法
CA3154189A1 (en) * 2019-10-10 2021-04-15 Hung Do Variant igf2 constructs
MX2022005916A (es) * 2019-11-19 2022-08-04 Asklepios Biopharmaceutical Inc Virus adenoasociado terapeutico que comprende promotores especificos del higado para el tratamiento de la enfermedad de pompe y los trastornos liposomales.
EP4149519A4 (en) * 2020-05-14 2024-06-12 The Trustees of The University of Pennsylvania COMPOSITIONS FOR THE TREATMENT OF POMPE DISEASE
BR112023001456A2 (pt) 2020-07-27 2023-04-11 Voyager Therapeutics Inc Composições e métodos para o tratamento de distúrbios neurológicos relacionados à deficiência de beta glicosilceramidase
WO2022026410A2 (en) 2020-07-27 2022-02-03 Voyager Therapeutics, Inc Compositions and methods for the treatment of niemann-pick type c1 disease
WO2022104186A1 (en) * 2020-11-16 2022-05-19 Tavotek Biotherapeutics (Hong Kong) Limited A shielded and homing bispecific antibody that simultaneously inhibits angiogenic pathway targets and her2 family proteins and uses thereof
AU2021392642A1 (en) 2020-12-01 2023-06-22 The Trustees Of The University Of Pennsylvania Compositions and uses thereof for treatment of angelman syndrome
WO2023086939A1 (en) * 2021-11-12 2023-05-19 Amicus Therapeutics, Inc. Compositions and methods for treating mucopolysaccharidosis iiia
WO2023091949A2 (en) 2021-11-17 2023-05-25 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
WO2023240236A1 (en) 2022-06-10 2023-12-14 Voyager Therapeutics, Inc. Compositions and methods for the treatment of spinal muscular atrophy related disorders
WO2024151982A1 (en) 2023-01-13 2024-07-18 Amicus Therapeutics, Inc. Gene therapy constructs for the treatment of pompe disease

Family Cites Families (33)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6087164A (en) * 1997-10-03 2000-07-11 Yissum Research Development Company Of The Hebrew University Of Jerusalem Methods and compositions for inducing tumor-specific cytotoxicity
AU1086501A (en) 1999-10-15 2001-04-30 Carnegie Institution Of Washington Rna interference pathway genes as tools for targeted genetic interference
US6326193B1 (en) 1999-11-05 2001-12-04 Cambria Biosciences, Llc Insect control agent
EP1118334A1 (en) 2000-01-11 2001-07-25 Aventis Behring Gesellschaft mit beschränkter Haftung Method for the production of conjugates and uses thereof for the prevention and treatment of allergic reactions and autoimmune diseases
WO2001096584A2 (en) 2000-06-12 2001-12-20 Akkadix Corporation Materials and methods for the control of nematodes
IL158623A0 (en) 2001-04-30 2004-05-12 Symbiontics Inc Subcellular targeting of therapeutic proteins
US7629309B2 (en) 2002-05-29 2009-12-08 Zystor Therapeutics, Inc. Targeted therapeutic proteins
US7560424B2 (en) 2001-04-30 2009-07-14 Zystor Therapeutics, Inc. Targeted therapeutic proteins
US20030072761A1 (en) 2001-10-16 2003-04-17 Lebowitz Jonathan Methods and compositions for targeting proteins across the blood brain barrier
WO2003032727A1 (en) * 2001-10-16 2003-04-24 Symbiontics Inc. Methods and compositions for targeting underglycosylated proteins across the blood brain barrier
DE10237639A1 (de) * 2002-08-13 2004-02-26 Martin-Luther-Universität Halle-Wittenberg Verfahren zur Herstellung von virusanalogen Partikeln durch in vitro-Assemblierung von Kapsomeren oder von Kapsomeren, an die biologisch aktive Makromoleküle gebunden sind, bei gleichzeitiger Verpackung der Makromoleküle
EP1587923B1 (en) 2003-01-22 2011-08-24 Duke University Improved constructs for expressing lysosomal polypeptides
WO2004098648A1 (en) 2003-05-01 2004-11-18 Genzyme Corporation Gene therapy for neurometabolic disorders
GB0314856D0 (en) * 2003-06-25 2003-07-30 Unitargeting Res As Protein expression system
WO2005033134A2 (en) 2003-09-30 2005-04-14 Regeneron Pharmaceuticals, Inc. Secreted protein therapeutics and uses thereof
US20050244400A1 (en) 2004-02-10 2005-11-03 Zystor Therapeutics, Inc. Acid alpha-glucosidase and fragments thereof
ES2242542B1 (es) * 2004-04-30 2006-12-16 Consejo Superior De Investigaciones Cientificas Procedimiento para la produccion en levaduras de capsidas virales vacias compuestas por proteinas derivadas de pvp2 del virus causante de la enfermedad de la bursitis infecciosa (ibdv).
WO2006007560A2 (en) 2004-07-01 2006-01-19 University Of Pennsylvania Targeted protein replacement for the treatment of lysosomal storage disorders
CN1903374A (zh) * 2005-07-27 2007-01-31 中国人民解放军军事医学科学院生物医学分析中心 大肠杆菌在制备肿瘤诊断或治疗药物中的用途
CA2669347A1 (en) 2006-11-13 2008-05-29 Zystor Therapeutics, Inc. Methods for treating pompe disease
HUE034850T2 (en) 2008-05-07 2018-03-28 Biomarin Pharm Inc Lysosomal targeting peptides and their use
PT3912643T (pt) 2009-02-13 2023-01-12 Immunomedics Inc Imunoconjugados com uma ligação intracelular clivável
US9279007B2 (en) * 2010-11-22 2016-03-08 Amicus Therapeutics, Inc. Signal sequences to improve protein expressions and secretion of recombinant enzymes and other proteins
PL2646044T3 (pl) * 2010-11-30 2020-03-31 Orphazyme A/S Sposoby zwiększenia aktywności wewnątrzkomórkowej Hsp70
US8580922B2 (en) 2011-03-04 2013-11-12 Shire Human Genetic Therapies, Inc. Peptide linkers for polypeptide compositions and methods for using same
EP2687597B1 (en) 2011-03-16 2018-12-26 Amano Enzyme Inc. Modified alpha-glucosidase and applications of same
DK2714752T3 (en) 2011-05-27 2018-02-26 Amicus Therapeutics Inc PROCEDURES FOR THE CONNECTION OF TARGETING PEPTIDES TO RECOMBINANT LYSOSOMAL ENZYMES FOR IMPROVED TREATMENT OF LYSOSOMAL DISPOSAL DISEASES
US9387236B2 (en) * 2011-06-10 2016-07-12 Prothera Inc. Pharmaceutical compositions containing protease and methods for the treatment of lysosomal storage diseases
LT3115372T (lt) 2012-11-27 2019-06-25 Biomarin Pharmaceutical Inc. Tiksliniai terapiniai lizosominio fermento sulieti baltymai ir jų panaudojimas
ES2804594T3 (es) * 2013-03-15 2021-02-08 Amicus Therapeutics Inc Reticuladores químicos
WO2015060722A1 (en) * 2013-10-24 2015-04-30 Uniqure Ip B.V. Aav-5 pseudotyped vector for gene therapy for neurological diseases
WO2015085238A1 (en) 2013-12-05 2015-06-11 The Regents Of The University Of California, A California Corporation Inhibitors of lpxc
SG11202010533WA (en) 2018-04-30 2020-11-27 Amicus Therapeutics Inc Gene therapy constructs and methods of use

Also Published As

Publication number Publication date
TW202344691A (zh) 2023-11-16
TWI808169B (zh) 2023-07-11
CN112513071A (zh) 2021-03-16
SG11202010533WA (en) 2020-11-27
ZA202006905B (en) 2022-03-30
PH12020551818A1 (en) 2021-07-26
CO2020014729A2 (es) 2020-12-21
EP3788064A1 (en) 2021-03-10
US20190343968A1 (en) 2019-11-14
CL2020002809A1 (es) 2021-04-30
CA3098674A1 (en) 2019-11-07
TW202003053A (zh) 2020-01-16
AU2019261982A1 (en) 2020-10-15
US10874750B2 (en) 2020-12-29
US11491243B2 (en) 2022-11-08
KR20210005154A (ko) 2021-01-13
US20210162075A1 (en) 2021-06-03
WO2019213180A1 (en) 2019-11-07
US20230233711A1 (en) 2023-07-27
JP2021521851A (ja) 2021-08-30
BR112020021962A2 (pt) 2021-01-26

Similar Documents

Publication Publication Date Title
PH12020551818A1 (en) Gene therapy constructs and methods of use
MX2021006646A (es) Vector viral adenoasociado recombinante para el suministro de genes.
MX2020010458A (es) Conjugados de camptotecina-peptido.
PH12018500964A1 (en) Modified friedreich ataxia genes and vectors for gene therapy
EA202091486A1 (ru) Способы и композиции для поляризации макрофагов
CY1124588T1 (el) Γενετικο κατασκευασμα
MX2019010735A (es) Sistemas y metodos para la produccion dirigida de una proteina terapeutica dentro de una celula objetivo.
MX2019008755A (es) Variantes de hsd17b13 y usos de las mismas.
SG10201908086SA (en) Listeria-based compositions comprising a peptide minigene expression system and methods of use thereof
MX2017009044A (es) Metodo para el tratamiento de malignidades.
MX2020000713A (es) Metodos de aferesis y sus usos.
BR112021024463A2 (pt) Oligonucleotídeos e métodos de uso para o tratamento de doenças neurológicas
MX2019004487A (es) Metodos y composiciones para el tratamiento de la enfermedad de fabry.
MX2021002041A (es) Terapia génica para el tratamiento de galactosemia.
WO2020079034A3 (en) Intein proteins and uses thereof
PH12021551135A1 (en) Rna encoding a protein
MX2021007221A (es) Factor i del complemento y cofactor del factor i del complemento, vectores que realizan codificacion para estos y uso terapeutico.
CR20220076A (es) Anticuerpos anti-cd96 y sus métodos de uso
AR113450A1 (es) Vectores adenovirales competentes en la replicación
IL268912A (en) Buildings for genetic therapy and methods for treating hearing loss
IL275951A (en) Vectors that evade the immune system and their use for gene therapy
MX2022004345A (es) Construcciones de igf2 variantes.
CL2018002900A1 (es) Construcciones de adn para la fabricación de polipéptidos bioterapéuticos para su uso en vacunas animales y agentes terapéuticos.
EA202092493A1 (ru) Конструкции для генной терапии и способы применения
MX2022015820A (es) Vacunas de cancer dirigidas a muc16 y sus usos.