IL327137A - וירוס קשור אדנו טיפולי המשתמש בחומצה גרעינית המקודדת אלפא-גלוקוזידאז (gaa) לטיפול במחלת פומפה, עם פפטיד אות הטרולוגי - Google Patents

וירוס קשור אדנו טיפולי המשתמש בחומצה גרעינית המקודדת אלפא-גלוקוזידאז (gaa) לטיפול במחלת פומפה, עם פפטיד אות הטרולוגי

Info

Publication number
IL327137A
IL327137A IL327137A IL32713726A IL327137A IL 327137 A IL327137 A IL 327137A IL 327137 A IL327137 A IL 327137A IL 32713726 A IL32713726 A IL 32713726A IL 327137 A IL327137 A IL 327137A
Authority
IL
Israel
Prior art keywords
gaa
glucosidase
nucleic acid
acid encoding
signal peptide
Prior art date
Application number
IL327137A
Other languages
English (en)
Original Assignee
Askbio Inc
Shanghai Belief Delivery Biomed Co Ltd
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 Askbio Inc, Shanghai Belief Delivery Biomed Co Ltd filed Critical Askbio Inc
Publication of IL327137A publication Critical patent/IL327137A/he

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
    • 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
    • C12N15/11—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)
    • C12N9/24—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)
    • 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
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Diabetes (AREA)
  • Virology (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Enzymes And Modification Thereof (AREA)
IL327137A 2023-09-18 2024-09-16 וירוס קשור אדנו טיפולי המשתמש בחומצה גרעינית המקודדת אלפא-גלוקוזידאז (gaa) לטיפול במחלת פומפה, עם פפטיד אות הטרולוגי IL327137A (he)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202363539000P 2023-09-18 2023-09-18
PCT/US2024/046897 WO2025064350A1 (en) 2023-09-18 2024-09-16 Therapeutic adeno-associated virus using nucleic acid encoding alpha-glucosidase (gaa) for treating pompe disease, with heterologous signal peptide

Publications (1)

Publication Number Publication Date
IL327137A true IL327137A (he) 2026-05-01

Family

ID=95071992

Family Applications (1)

Application Number Title Priority Date Filing Date
IL327137A IL327137A (he) 2023-09-18 2024-09-16 וירוס קשור אדנו טיפולי המשתמש בחומצה גרעינית המקודדת אלפא-גלוקוזידאז (gaa) לטיפול במחלת פומפה, עם פפטיד אות הטרולוגי

Country Status (4)

Country Link
AU (1) AU2024346724A1 (he)
IL (1) IL327137A (he)
TW (1) TW202526024A (he)
WO (1) WO2025064350A1 (he)

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102747683B1 (ko) * 2015-12-30 2024-12-31 아미쿠스 세라퓨틱스, 인코포레이티드 폼페병 치료용의 강화된 산 알파-글루코시다제
PE20191203A1 (es) * 2016-09-12 2019-09-10 Genethon Variantes de alfa-glucosidasa acida y usos de las mismas
US12534744B2 (en) * 2020-05-06 2026-01-27 Cellectis S.A. Methods to genetically modify cells for delivery of therapeutic proteins
WO2022121860A1 (en) * 2020-12-07 2022-06-16 East China University Of Science And Technology Methods and kits for inducing immunotolerance to gene delivering target vehicle

Also Published As

Publication number Publication date
WO2025064350A1 (en) 2025-03-27
TW202526024A (zh) 2025-07-01
AU2024346724A1 (en) 2026-04-09

Similar Documents

Publication Publication Date Title
CN102741405B (zh) 提高基因表达的系统和保持有该系统的载体
CA2529179A1 (en) Recombinant adeno-associated virus vector for treatment of alzheimer disease
ES2947311T3 (es) Gen UBE3A modificado para un enfoque de terapia génica para el síndrome de Angelman
FI3684423T3 (fi) Adenoassesioidun viruksen kapsidivariantteja ja niiden käyttömenetelmiä
JP2009515529A5 (he)
DK1857122T3 (da) Stabiliseret mRNA med forøget G/C-indhold, kodende for et viralt antigen
HUP0203409A2 (hu) Gamma-interferon-konjugátumok
US12036247B2 (en) Multiple transgene recombinant adenovirus
PH12021552637A1 (en) Compositions useful for treatment of pompe disease
HRP20240184T1 (hr) Polinukleotidi
WO2003088899A3 (en) Methods for the production of chimeric adeno-associated virus (aav) vectors, compositions of chimeric aav vectors, and methods of use thereof
CA3254578A1 (en) Adeno-associated virus therapy involving an alpha-glucosidase (GAA) codon-optimized nucleic acid to treat Pompe disease, with signal peptide modifications
JP2023540599A5 (he)
WO2008040111A8 (en) Novel peptides for treating and preventing immune-related disorders, including treating and preventing infection by modulating innate immunity
MY157947A (en) Novel peptides for treating and preventing immune-related disorders, including treating and preventing infection by modulating innate immunity
CA3305271A1 (en) Therapeutic adeno-associated virus using nucleic acid encoding alpha-glucosidase (gaa) for treating pompe disease, with heterologous signal peptide
JP2022060514A (ja) ガバペンチノイドからの干渉が減少し、ｈｇｆ異型体を発現するｄｎａコンストラクトを用いる神経病症の治療
HK40121746A (en) Therapeutic adeno-associated virus using codon optimized nucleic acid encoding alpha-glucosidase (gaa) for treating pompe disease, with signal peptide modifications
RU2004134331A (ru) Антиген muc1 со сниженным числом повторяющихся vntr-блоков
US20220186252A1 (en) Adeno associated viral vector delivery of antibodies for the treatment of disease mediated by dysregulated plasma kallikrein
RU2017112853A (ru) Аденовирусный вектор, кодирующий гомологог-1 белка atonal человека (hath1)
CN118165126B (zh) 一种重组猪干扰素λ1、猪干扰素γ和猪Fc（KiH）的融合蛋白及其应用
CN118165125B (zh) 一种重组猪干扰素λ1、猪干扰素γ和猪SA的融合蛋白及其应用
KR20240015036A (ko) 인터페론-β 분비를 위한 재조합 발현 벡터 및 이로형질전환된 약독화 살모넬라 균주
CN117045824A (zh) 重编程因子制剂、生物材料及用途