PH12021552638A1 - Compositions useful for treatment of pompe disease - Google Patents

Compositions useful for treatment of pompe disease

Info

Publication number
PH12021552638A1
PH12021552638A1 PH1/2021/552638A PH12021552638A PH12021552638A1 PH 12021552638 A1 PH12021552638 A1 PH 12021552638A1 PH 12021552638 A PH12021552638 A PH 12021552638A PH 12021552638 A1 PH12021552638 A1 PH 12021552638A1
Authority
PH
Philippines
Prior art keywords
raav
treatment
compositions useful
disease
pompe disease
Prior art date
Application number
PH1/2021/552638A
Other languages
English (en)
Inventor
Juliette Hordeaux
James M Wilson
Original Assignee
Univ Pennsylvania
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 Univ Pennsylvania filed Critical Univ Pennsylvania
Publication of PH12021552638A1 publication Critical patent/PH12021552638A1/en

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • 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
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • 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
    • A61P3/00—Drugs for disorders of the metabolism
    • 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
    • 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
    • C12N15/625—DNA sequences coding for fusion proteins containing a sequence coding for a signal sequence
    • 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)
    • 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
    • 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)
    • C12N9/2405—Glucanases
    • C12N9/2408—Glucanases acting on alpha -1,4-glucosidic bonds
    • 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)
    • 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
    • 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
    • A01K2267/0306—Animal model for genetic diseases
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • 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
    • 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)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Diabetes (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Endocrinology (AREA)
  • Toxicology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Neurology (AREA)
  • Immunology (AREA)
  • Mycology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
PH1/2021/552638A 2019-04-30 2020-04-29 Compositions useful for treatment of pompe disease PH12021552638A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962840911P 2019-04-30 2019-04-30
US201962913401P 2019-10-10 2019-10-10
PCT/US2020/030493 WO2020223362A1 (en) 2019-04-30 2020-04-29 Compositions useful for treatment of pompe disease

Publications (1)

Publication Number Publication Date
PH12021552638A1 true PH12021552638A1 (en) 2022-08-15

Family

ID=73029176

Family Applications (2)

Application Number Title Priority Date Filing Date
PH1/2021/552638A PH12021552638A1 (en) 2019-04-30 2020-04-29 Compositions useful for treatment of pompe disease
PH1/2021/552637A PH12021552637A1 (en) 2019-04-30 2020-04-29 Compositions useful for treatment of pompe disease

Family Applications After (1)

Application Number Title Priority Date Filing Date
PH1/2021/552637A PH12021552637A1 (en) 2019-04-30 2020-04-29 Compositions useful for treatment of pompe disease

Country Status (16)

Country Link
US (3) US20220193261A1 (enExample)
EP (2) EP3963063A4 (enExample)
JP (2) JP2022530824A (enExample)
KR (2) KR20220008280A (enExample)
CN (2) CN114127275A (enExample)
AU (2) AU2020266829A1 (enExample)
BR (2) BR112021021792A2 (enExample)
CA (2) CA3134485A1 (enExample)
CL (2) CL2021002754A1 (enExample)
CO (2) CO2021016200A2 (enExample)
IL (2) IL287522A (enExample)
MX (2) MX2021013365A (enExample)
PH (2) PH12021552638A1 (enExample)
SG (2) SG11202111380VA (enExample)
TW (1) TW202100541A (enExample)
WO (2) WO2020223362A1 (enExample)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2018281280B2 (en) 2017-06-07 2024-12-19 Regeneron Pharmaceuticals, Inc. Compositions and methods for internalizing enzymes
CN112040985A (zh) 2018-02-07 2020-12-04 瑞泽恩制药公司 用于递送治疗性蛋白质的方法和组合物
EP3793591A1 (en) 2018-05-17 2021-03-24 Regeneron Pharmaceuticals, Inc. Anti-cd63 antibodies, conjugates, and uses thereof
JP2022552254A (ja) * 2019-10-10 2022-12-15 アミカス セラピューティックス インコーポレイテッド バリアントｉｇｆ２構築体
EP4149519A4 (en) * 2020-05-14 2024-06-12 The Trustees of The University of Pennsylvania COMPOSITIONS FOR THE TREATMENT OF POMPE DISEASE
CA3183153A1 (en) 2020-06-17 2021-12-23 Christian HINDERER Compositions and methods for treatment of gene therapy patients
AU2022388800A1 (en) * 2021-11-12 2024-06-27 The Trustees Of The University Of Pennsylvania Gene therapy for treatment of mucopolysaccharidosis iiia
NL2031676B1 (en) * 2022-04-22 2023-11-07 Univ Erasmus Med Ct Rotterdam Gene therapy for Pompe Disease
WO2024003687A1 (en) * 2022-06-28 2024-01-04 Pfizer Inc. Nucleic acids encoding acid alpha-glucosidase (gaa) and vectors for gene therapy
US20260216373A1 (en) * 2023-01-13 2026-07-30 Amicus Therapeutics, Inc. Gene Therapy Constructs for the Treatment of Pompe Disease
CN116693633B (zh) * 2023-02-21 2023-12-22 广州派真生物技术有限公司 腺相关病毒突变体及其应用
WO2025171163A1 (en) * 2024-02-06 2025-08-14 Duke University Compositions for and methods of treating lysosomal storage diseases

Family Cites Families (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PT1587923E (pt) * 2003-01-22 2011-12-07 Univ Duke Construções melhoradas para a expressão de polipéptidos lisossomais
DE602005020745D1 (de) * 2004-02-10 2010-06-02 Zystor Therapeutics Inc Saure alpha-glukosidase und fragmente davon
AU2007322123A1 (en) * 2006-11-13 2008-05-29 Biomarin Pharmaceutical Inc. Methods for treating Pompe disease
DK2205737T3 (da) * 2007-10-04 2013-05-21 Santaris Pharma As Mikromirer
WO2009137721A2 (en) * 2008-05-07 2009-11-12 Zystor Therapeutics, Inc. Lysosomal targeting peptides and uses thereof
WO2010096369A1 (en) * 2009-02-18 2010-08-26 Amicus Therapeutics, Inc. Mouse model for pompe disease and methods of use thereof
EP2628803A3 (en) 2009-02-25 2014-01-01 Cepheid Methods of detecting lung cancer
US8785168B2 (en) * 2009-06-17 2014-07-22 Biomarin Pharmaceutical Inc. Formulations for lysosomal enzymes
HRP20181315T1 (hr) 2010-11-22 2018-10-19 Amicus Therapeutics, Inc. Nove signalne sekvence za poboljšanje ekspresije proteina i izlučivanja rekombinantnih enzima i drugih proteina
WO2012070014A2 (en) * 2010-11-26 2012-05-31 Yissum Research Development Company Of The Hebrew University Of Jerusalem Ltd. Identification of novel cell surface markers for pancreatic progenitor cells and definite endodermal cells
GB201103062D0 (en) * 2011-02-22 2011-04-06 Isis Innovation Method
LT2714752T (lt) * 2011-05-27 2018-03-12 Amicus Therapeutics, Inc. Nukreipiančių peptidų kopuliavimo ant rekombinantinių lizosominių fermentų būdai, skirti lizosominio kaupimo ligų gydymo pagerinimui
WO2013013019A2 (en) * 2011-07-21 2013-01-24 Alnylam Pharmaceuticals, Inc. Lysosomal polypeptides, methods of making and using
CA2947466A1 (en) * 2014-05-01 2015-11-05 University Of Washington In vivo gene engineering with adenoviral vectors
AU2016366549B2 (en) * 2015-12-11 2022-11-10 California Institute Of Technology Targeting peptides for directing adeno-associated viruses (AAVs)
KR102618519B1 (ko) * 2016-03-30 2023-12-28 아미쿠스 세라퓨틱스, 인코포레이티드 재조합 산 알파-글루코시다제를 포함하는 제형
EP3293260A1 (en) * 2016-09-12 2018-03-14 Genethon Acid-alpha glucosidase variants and uses thereof
KR102617502B1 (ko) * 2016-09-12 2023-12-26 제네똥 산-알파 글루코시다제 변이체 및 이의 용도
EP3293259A1 (en) 2016-09-12 2018-03-14 Genethon Acid-alpha glucosidase variants and uses thereof
WO2018178067A1 (en) * 2017-03-27 2018-10-04 Vrije Universiteit Brussel Diaphragm-specific nucleic acid regulatory elements and methods and use thereof
JP2018198611A (ja) 2018-08-03 2018-12-20 ウニベルシダッド アウトノマ デ バルセロナ 脂肪組織に形質導入するのに有用なアデノ随伴ウイルスベクター

Also Published As

Publication number Publication date
CN114072515A (zh) 2022-02-18
MX2021013365A (es) 2022-01-26
TW202100541A (zh) 2021-01-01
CA3134523A1 (en) 2020-11-05
PH12021552637A1 (en) 2022-07-18
CO2021016198A2 (es) 2022-01-17
US20220193207A1 (en) 2022-06-23
KR20220004696A (ko) 2022-01-11
BR112021021720A2 (pt) 2021-12-28
SG11202111380VA (en) 2021-11-29
EP3963063A1 (en) 2022-03-09
EP3980548A4 (en) 2023-09-06
WO2020223362A8 (en) 2021-01-14
IL287523A (en) 2021-12-01
EP3980548A1 (en) 2022-04-13
AU2020266552A1 (en) 2021-11-11
CN114127275A (zh) 2022-03-01
CA3134485A1 (en) 2020-11-05
AU2020266829A1 (en) 2021-11-11
CL2021002754A1 (es) 2022-05-27
WO2020223356A1 (en) 2020-11-05
JP2022530824A (ja) 2022-07-01
BR112021021792A2 (pt) 2022-01-04
US20260191993A1 (en) 2026-07-09
MX2021013364A (es) 2022-01-26
KR20220008280A (ko) 2022-01-20
JP2022530833A (ja) 2022-07-01
CL2021002755A1 (es) 2022-05-27
CO2021016200A2 (es) 2022-01-17
IL287522A (en) 2021-12-01
JP7585233B2 (ja) 2024-11-18
SG11202111400TA (en) 2021-11-29
US20220193261A1 (en) 2022-06-23
WO2020223362A1 (en) 2020-11-05
EP3963063A4 (en) 2023-09-27

Similar Documents

Publication Publication Date Title
WO2020223362A8 (en) Compositions useful for treatment of pompe disease
PH12020550094A1 (en) Adeno-associated virus variant capsids and methods of use thereof
PH12020550706A1 (en) Adeno-associated virus variant capsids and use for inhibiting angiogenesis
SA519402566B1 (ar) تركيبات نافعة في معالجة ضمور العضل النخاعي
PH12018502387A1 (en) Adeno-associated virus variant capsids and methods of use thereof
JP2020510428A5 (enExample)
MX2023006694A (es) Tratamiento de la enfermedad de danon.
EA202092362A1 (ru) Вирусные векторы для нацеливания на ткани глаза
RU2019100525A (ru) Векторная система на основе аденоассоциированного вируса
MX2022004352A (es) Vectores virales adenoasociados para el tratamiento de la enfermedad de niemann-pick tipo c.
JP2017505626A5 (enExample)
RU2019126509A (ru) Выделенный модифицированный белок VP1 капсида аденоассоциированного вируса 5 серотипа (AAV5), капсид и вектор на его основе
BR112023019056A2 (pt) Métodos para tratar doenças oculares usando variantes de aav2 que codificam aflibercepte
PH12022550109A1 (en) Modified aav capsid proteins for treatment of arthritic disease
WO2022076803A8 (en) Compositions and methods for treatment of fabry disease
UY38881A (es) Métodos de tratamiento de enfermedades neovasculares oculares utilizando variantes de aav2 que codifican aflibercept
MX2023007474A (es) Proteinas de la capside viral con especificidad para las celulas del tejido cardiaco.
BR112022003206A2 (pt) Métodos para tratar doenças neovasculares oculares usando variantes de aav2 que codificam aflibercepte
FI3177313T3 (fi) Telomeraasi-käänteistranskriptaasipohjaisia hoitoja sydäninfarktiin liittyvien tilojen hoitamiseksi
MX2025000757A (es) Casetes reguladores específicos del músculo
EA202192973A1 (ru) Композиции, полезные для лечения болезни помпе
WO2020132115A8 (en) Methods and compositions for treating glycogen storage diseases
AR122260A1 (es) Composiciones útiles para el tratamiento de la enfermedad de pompe
EA202192346A1 (ru) Доставка -саркогликана с помощью вектора на основе аденоассоциированного вируса и лечение мышечной дистрофии
RU2021105822A (ru) Недеструктивная генная терапия для лечения mma