IL292401A - Compositions and methods for treating glycogen storage disorders - Google Patents

Compositions and methods for treating glycogen storage disorders

Info

Publication number
IL292401A
IL292401A IL292401A IL29240122A IL292401A IL 292401 A IL292401 A IL 292401A IL 292401 A IL292401 A IL 292401A IL 29240122 A IL29240122 A IL 29240122A IL 292401 A IL292401 A IL 292401A
Authority
IL
Israel
Prior art keywords
weeks
patient
amount
gaa
aav vector
Prior art date
Application number
IL292401A
Other languages
English (en)
Hebrew (he)
Original Assignee
Audentes 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 Audentes Therapeutics Inc filed Critical Audentes Therapeutics Inc
Publication of IL292401A publication Critical patent/IL292401A/en

Links

Classifications

    • 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
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • 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
    • 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
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with 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
    • A61K48/005—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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • 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
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • 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
    • 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)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Diabetes (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Pulmonology (AREA)
  • Neurology (AREA)
IL292401A 2019-10-25 2020-10-23 Compositions and methods for treating glycogen storage disorders IL292401A (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962926282P 2019-10-25 2019-10-25
US202063083349P 2020-09-25 2020-09-25
PCT/US2020/057081 WO2021081338A1 (en) 2019-10-25 2020-10-23 Compositions and methods for treating glycogen storage disorders

Publications (1)

Publication Number Publication Date
IL292401A true IL292401A (en) 2022-06-01

Family

ID=75620302

Family Applications (1)

Application Number Title Priority Date Filing Date
IL292401A IL292401A (en) 2019-10-25 2020-10-23 Compositions and methods for treating glycogen storage disorders

Country Status (14)

Country Link
US (1) US20220387562A1 (https=)
EP (1) EP4048286A4 (https=)
JP (1) JP2022554141A (https=)
KR (1) KR20220105643A (https=)
CN (1) CN114828858A (https=)
AU (1) AU2020372429A1 (https=)
BR (1) BR112022007674A2 (https=)
CA (1) CA3158281A1 (https=)
CO (1) CO2022006772A2 (https=)
IL (1) IL292401A (https=)
MX (1) MX2022004799A (https=)
PH (1) PH12022550977A1 (https=)
TW (2) TW202600177A (https=)
WO (1) WO2021081338A1 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3250992A1 (en) * 2022-02-03 2023-08-10 Astellas Gene Therapies, Inc. COMPOSITIONS AND METHODS FOR AN IMPROVED TREATMENT OF POMPE DISEASE
JP2025527694A (ja) 2022-08-23 2025-08-22 マストバイオ株式会社 Ｉｌ２変異体及びそれを含むタンパク質複合体

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2330615T3 (es) * 2000-04-28 2009-12-14 Asklepios Biopharmaceutical, Inc. Secuencias de adn que codifican para minigenes de distrofina y metodos de uso de las mismas.
EP1366160B1 (en) * 2000-10-06 2008-07-09 The Regents Of The University Of Michigan Mini-dystrophin nucleic acid and peptide sequences
KR20150020250A (ko) * 2012-06-19 2015-02-25 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 당뇨병을 치료하기 위한 조성물 및 방법
IL258005B2 (en) * 2015-09-17 2025-01-01 Res Inst Nationwide Childrens Hospital Methods and materials for gene therapy for beta-4,1-N-acetylgalactosaminyltransferase 2
EP3293260A1 (en) * 2016-09-12 2018-03-14 Genethon Acid-alpha glucosidase variants and uses thereof
EP3596112A2 (en) * 2017-03-17 2020-01-22 Newcastle University Adeno-associated virus vector delivery of a fragment of micro-dystrophin to treat muscular dystrophy
US11597917B2 (en) * 2017-07-06 2023-03-07 The Medical College Of Wisconsin, Inc. In vitro and in vivo enrichment strategy targeting lymphocytes derived from vector transduced HSCs for therapy of disorders
CN111936621B (zh) * 2018-02-05 2025-05-13 奥登茨治疗公司 转录调控元件和它的用途
CN111902539B (zh) * 2018-02-07 2025-05-16 吉尼松公司 杂合调控元件

Also Published As

Publication number Publication date
CA3158281A1 (en) 2021-04-29
US20220387562A1 (en) 2022-12-08
JP2022554141A (ja) 2022-12-28
CO2022006772A2 (es) 2022-08-09
CN114828858A (zh) 2022-07-29
TW202116359A (zh) 2021-05-01
EP4048286A4 (en) 2023-12-06
WO2021081338A1 (en) 2021-04-29
EP4048286A1 (en) 2022-08-31
KR20220105643A (ko) 2022-07-27
BR112022007674A2 (pt) 2022-08-09
AU2020372429A1 (en) 2022-04-28
MX2022004799A (es) 2022-07-19
TWI885000B (zh) 2025-06-01
TW202600177A (zh) 2026-01-01
PH12022550977A1 (en) 2023-10-09

Similar Documents

Publication Publication Date Title
JP7776477B2 (ja) 転写調節要素及びその使用
AU2020282352B2 (en) Compositions and methods for selective gene regulation
JP2019504888A (ja) 一本鎖アデノ随伴ウイルス９または自己相補型アデノ随伴ウイルス９の脳脊髄液への注射
EP3210632B1 (en) Gene therapy for the treatment of a retinal degeneration disease
US20240216540A1 (en) Compositions and methods for treating sensorineural hearing loss using stereocilin dual vector systems
US20220387562A1 (en) Compositions and methods for treating glycogen storage disorders
CN115029360A (zh) 用于治疗粘多糖贮积症iiia型的转基因表达盒
RU2839698C1 (ru) Композиции и способы лечения нарушений накопления гликогена
US20230089784A1 (en) Methods and compositions for production of genetically modified primary cells
US20260027236A1 (en) Guide rnas, vectors, and virions for targeting mutations in the pln gene
WO2026080685A1 (en) Guide rnas, vectors, and virions for targeting mutations in the pln gene
WO2019228501A1 (en) Lentiviral vector used for treatment of gaucher, lentivirus, and preparation method and application thereof
CA3014704C (en) Gene therapy for the treatment of a retinal degeneration disease
JP2026504367A (ja) グルコシルセラミダーゼβ１欠損に関連する神経障害の治療のための組成物及び方法
WO2020187272A1 (zh) 一种用于基因治疗的融合蛋白及其应用
HK1241759A1 (en) Gene therapy for the treatment of a retinal degeneration disease
HK1241759A (en) Gene therapy for the treatment of a retinal degeneration disease