CN120866359A - 用于治疗神经退行性障碍的重组aav载体 - Google Patents

用于治疗神经退行性障碍的重组aav载体

Info

Publication number
CN120866359A
CN120866359A CN202511044894.9A CN202511044894A CN120866359A CN 120866359 A CN120866359 A CN 120866359A CN 202511044894 A CN202511044894 A CN 202511044894A CN 120866359 A CN120866359 A CN 120866359A
Authority
CN
China
Prior art keywords
aadc
nucleotide sequence
gdnf
sequence
gba1
Prior art date
Legal status (The legal status 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 status listed.)
Pending
Application number
CN202511044894.9A
Other languages
English (en)
Chinese (zh)
Inventor
陶冶铮
田幸雪
赵小平
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Shanghai Tianze Yuntai Biomedical Co ltd
Original Assignee
Shanghai Tianze Yuntai Biomedical 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 Shanghai Tianze Yuntai Biomedical Co ltd filed Critical Shanghai Tianze Yuntai Biomedical Co ltd
Publication of CN120866359A publication Critical patent/CN120866359A/zh
Pending legal-status Critical Current

Links

Classifications

    • 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
    • C12N9/88—Lyases (4.)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • 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
    • A61K48/005—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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • 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
    • C07K14/475—Growth factors; Growth regulators
    • C07K14/4756—Neuregulins, i.e. p185erbB2 ligands, glial growth factor, heregulin, ARIA, neu differentiation factor
    • 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
    • C07K14/475—Growth factors; Growth regulators
    • C07K14/48—Nerve growth factor [NGF]
    • 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
    • C07K14/52—Cytokines; Lymphokines; Interferons
    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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)
    • C12Y302/01045—Glucosylceramidase (3.2.1.45), i.e. beta-glucocerebrosidase
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y401/00—Carbon-carbon lyases (4.1)
    • C12Y401/01—Carboxy-lyases (4.1.1)
    • C12Y401/01028—Aromatic-L-amino-acid decarboxylase (4.1.1.28), i.e. tryptophane-decarboxylase
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • 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
    • C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Psychology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Immunology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
CN202511044894.9A 2022-04-19 2023-04-19 用于治疗神经退行性障碍的重组aav载体 Pending CN120866359A (zh)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
CNPCT/CN2022/087771 2022-04-19
CN2022087771 2022-04-19
CN202380034877.XA CN119487188A (zh) 2022-04-19 2023-04-19 用于治疗神经退行性障碍的重组aav载体
PCT/CN2023/089291 WO2023202637A1 (en) 2022-04-19 2023-04-19 Recombinant aav vectors for treating neurodegenerative disorders

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
CN202380034877.XA Division CN119487188A (zh) 2022-04-19 2023-04-19 用于治疗神经退行性障碍的重组aav载体

Publications (1)

Publication Number Publication Date
CN120866359A true CN120866359A (zh) 2025-10-31

Family

ID=88419256

Family Applications (5)

Application Number Title Priority Date Filing Date
CN202511044894.9A Pending CN120866359A (zh) 2022-04-19 2023-04-19 用于治疗神经退行性障碍的重组aav载体
CN202380034877.XA Pending CN119487188A (zh) 2022-04-19 2023-04-19 用于治疗神经退行性障碍的重组aav载体
CN202511044852.5A Pending CN120866357A (zh) 2022-04-19 2023-04-19 用于治疗神经退行性障碍的重组aav载体
CN202511079772.3A Active CN120866360B (zh) 2022-04-19 2023-04-19 用于治疗神经退行性障碍的重组aav载体
CN202511079819.6A Active CN120866361B (zh) 2022-04-19 2023-04-19 用于治疗神经退行性障碍的重组aav载体

Family Applications After (4)

Application Number Title Priority Date Filing Date
CN202380034877.XA Pending CN119487188A (zh) 2022-04-19 2023-04-19 用于治疗神经退行性障碍的重组aav载体
CN202511044852.5A Pending CN120866357A (zh) 2022-04-19 2023-04-19 用于治疗神经退行性障碍的重组aav载体
CN202511079772.3A Active CN120866360B (zh) 2022-04-19 2023-04-19 用于治疗神经退行性障碍的重组aav载体
CN202511079819.6A Active CN120866361B (zh) 2022-04-19 2023-04-19 用于治疗神经退行性障碍的重组aav载体

Country Status (5)

Country Link
US (1) US12605467B2 (https=)
EP (1) EP4511482A4 (https=)
JP (1) JP2025516136A (https=)
CN (5) CN120866359A (https=)
WO (1) WO2023202637A1 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN117018231B (zh) * 2023-08-16 2024-05-10 科辉智药(深圳)新药研究中心有限公司 用于治疗神经病变的基因疗法及其应用
WO2025166048A1 (en) * 2024-01-31 2025-08-07 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Synthetic gba1 genes

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20130224836A1 (en) * 2010-10-27 2013-08-29 Jichi Medical University Adeno-Associated Virus Virion for Gene Transfer to Nervous System Cells
CN108841868A (zh) * 2018-05-31 2018-11-20 康霖生物科技（杭州）有限公司 一种用于中枢神经系统疾病治疗的基因序列构建体
CN112553229A (zh) * 2014-11-05 2021-03-26 沃雅戈治疗公司 用于治疗帕金森病的aadc多核苷酸
WO2021138350A1 (en) * 2019-12-29 2021-07-08 Gloriana Therapeutics Mammalian cells secreting gdnf and their therapeutic use

Family Cites Families (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20030050273A1 (en) * 2001-08-29 2003-03-13 Keiya Ozawa Compositions and methods for treating neurodegenerative diseases
GB201118636D0 (en) * 2011-10-28 2011-12-07 Oxford Biomedica Ltd Nucleotide sequence
LT3137497T (lt) * 2014-05-02 2021-07-26 Genzyme Corporation Aav vektoriai, skirti tinklainės ir cns genų terapijai
EP3294890A4 (en) * 2015-05-08 2018-10-03 Children's Medical Research Institute Promoters for expression of heterologous genes
KR20210059796A (ko) * 2016-04-29 2021-05-25 오르파짐 에이/에스 글루코세레브로시다제 연관 장애를 치료하기 위한 아리모클로몰
WO2017191274A2 (en) * 2016-05-04 2017-11-09 Curevac Ag Rna encoding a therapeutic protein
BR112020006661A2 (pt) * 2017-10-03 2020-10-13 Prevail Therapeutics, Inc. terapias de genes para distúrbios lipossomais
WO2019068854A1 (en) * 2017-10-06 2019-04-11 Ospedale San Raffaele S.R.L. GENE THERAPY OF NEURODEGENERATIVE DISEASES USING VAA VECTORS
BR112020008033A2 (pt) * 2017-10-23 2020-10-27 Prevail Therapeutics, Inc. terapias gênicas para doença neurodegenerativa
CN109266648B (zh) * 2018-09-26 2021-10-19 中国科学技术大学 用于在体基因治疗的基因编辑组合物或试剂盒
KR20220022107A (ko) * 2018-12-05 2022-02-24 아베오나 테라퓨틱스 인코퍼레이티드 유전자 전달을 위한 재조합 아데노-연관 바이러스 벡터
CA3136004A1 (en) * 2019-04-10 2020-10-15 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
JP7664848B2 (ja) * 2019-04-10 2025-04-18 プリベイル セラピューティクス，インコーポレーテッド リソソーム障害のための遺伝子療法
US20220298528A1 (en) * 2019-08-12 2022-09-22 UCB Biopharma SRL Viral particles for use in treating synucleinopathies such as parkinson's diseases by gene therapy
CN113755524B (zh) * 2020-06-02 2023-11-03 舒泰神（北京）生物制药股份有限公司 用于治疗脊髓性肌萎缩的腺相关病毒载体及其用途
WO2022032153A1 (en) * 2020-08-06 2022-02-10 Voyager Therapeutics, Inc. Cell culture medium for use in producing gene therapy products in bioreactors
TW202325850A (zh) * 2021-11-29 2023-07-01 大陸商上海瑞宏迪醫藥有限公司 Aadc、gdnf多核苷酸及其用於治療帕金森病
CN114317610B (zh) * 2022-01-27 2024-07-02 上海本导基因技术有限公司 一种适用于帕金森疾病基因治疗的慢病毒载体

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20130224836A1 (en) * 2010-10-27 2013-08-29 Jichi Medical University Adeno-Associated Virus Virion for Gene Transfer to Nervous System Cells
CN112553229A (zh) * 2014-11-05 2021-03-26 沃雅戈治疗公司 用于治疗帕金森病的aadc多核苷酸
CN108841868A (zh) * 2018-05-31 2018-11-20 康霖生物科技（杭州）有限公司 一种用于中枢神经系统疾病治疗的基因序列构建体
WO2021138350A1 (en) * 2019-12-29 2021-07-08 Gloriana Therapeutics Mammalian cells secreting gdnf and their therapeutic use

Non-Patent Citations (5)

* Cited by examiner, † Cited by third party
Title
BRIAN P. MEAD 等: "Novel Focused Ultrasound Gene Therapy Approach Noninvasively Restores Dopaminergic Neuron Function in a Rat Parkinson’s Disease Model", NANO LETT, 14 June 2018 (2018-06-14), pages 4 *
S FURLER 等: "Recombinant AAV vectors containing the foot and mouth disease virus 2A sequence confer efficient bicistronic gene expression in cultured cells and rat substantia nigra neurons", GENE THERAPY, vol. 8, 31 December 2001 (2001-12-31), pages 864 *
YALAN WANG 等: "Codon Usage in Signal Sequences Affects Protein Expression and Secretion Using Baculovirus/Insect Cell Expression System", PLOS ONE, vol. 10, no. 12, 23 December 2015 (2015-12-23) *
无: "Accession No：BC069119.1, Homo sapiens glial cell derived neurotrophic factor, mRNA (cDNA clone MGC:95398 IMAGE:7216973），complete cds", NCBI, 15 July 2006 (2006-07-15) *
无: "Accession No:NM_000790.4 Homo sapiens dopa decarboxylase (DDC), transcript variant 2, mRNA", NCBI, 19 September 2021 (2021-09-19) *

Also Published As

Publication number Publication date
CN120866360A (zh) 2025-10-31
US12605467B2 (en) 2026-04-21
JP2025516136A (ja) 2025-05-27
US20250041456A1 (en) 2025-02-06
CN120866360B (zh) 2026-04-24
CN120866357A (zh) 2025-10-31
CN120866361B (zh) 2026-04-24
CN120866361A (zh) 2025-10-31
EP4511482A4 (en) 2026-03-18
EP4511482A1 (en) 2025-02-26
WO2023202637A1 (en) 2023-10-26
CN119487188A (zh) 2025-02-18

Similar Documents

Publication Publication Date Title
US12281321B2 (en) Frataxin expression constructs having engineered promoters and methods of use thereof
CA3192600A1 (en) Adeno-associated virus compositions having preferred expression levels
US12605467B2 (en) Recombinant AAV vectors for treating neurodegenerative disorders
US20230227802A1 (en) Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
KR20240028976A (ko) 뇌에서 높은 발현 수준을 갖는 aav 조성물
CN119095867A (zh) 具有优选的脑富集的所选aav组合物
JP2025535374A (ja) 好ましい脳エンリッチメントおよび低い肝臓エンリッチメントを有するアデノ随伴ウイルス組成物
US20250269058A1 (en) Gene therapy for treating neurodegenerative diseases
US20250345464A1 (en) Gene therapy for treating neurodegenerative diseases
CN121844054A (zh) 具有增加的脑富集的腺相关病毒组合物
WO2024245120A1 (en) Recombinant aav vectors for treating proteinopathies in central nervous system
IL322184A (en) Compositions and methods for treating neurological disorders associated with glucosylceramide beta 1 deficiency

Legal Events

Date Code Title Description
PB01 Publication
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination
CB02 Change of applicant information
CB02 Change of applicant information

Country or region after: China

Address after: 201210 Shanghai Pudong New Area Galileo Road No. 11 Building 2

Applicant after: Shanghai Tianze Yuntai Biopharmaceutical Co., Ltd.

Address before: 201210 Galileo Road No. 11, Building 2, Pudong New Area, Shanghai

Applicant before: Shanghai Tianze Yuntai biomedical Co.,Ltd.

Country or region before: China