KR20200075865A - 신경변성 질환에 대한 유전자 요법 - Google Patents

신경변성 질환에 대한 유전자 요법 Download PDF

Info

Publication number
KR20200075865A
KR20200075865A KR1020207014683A KR20207014683A KR20200075865A KR 20200075865 A KR20200075865 A KR 20200075865A KR 1020207014683 A KR1020207014683 A KR 1020207014683A KR 20207014683 A KR20207014683 A KR 20207014683A KR 20200075865 A KR20200075865 A KR 20200075865A
Authority
KR
South Korea
Prior art keywords
nucleic acid
isolated nucleic
vector
itr
c9orf72
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
KR1020207014683A
Other languages
English (en)
Korean (ko)
Inventor
아사 아벨리오비치
로라 헤크만
허브 린
Original Assignee
프리베일 테라퓨틱스, 인크.
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 프리베일 테라퓨틱스, 인크. filed Critical 프리베일 테라퓨틱스, 인크.
Publication of KR20200075865A publication Critical patent/KR20200075865A/ko
Pending legal-status Critical Current

Links

Images

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7105Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0085Brain, e.g. brain implants; Spinal cord
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/14Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16Anti-Parkinson drugs
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1138Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against receptors or cell surface proteins
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/50Physical structure
    • C12N2310/53Physical structure partially self-complementary or closed
    • C12N2310/531Stem-loop; Hairpin
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/32Special delivery means, e.g. tissue-specific
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2330/00Production
    • C12N2330/50Biochemical production, i.e. in a transformed host cell
    • C12N2330/51Specially adapted vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2710/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011Details
    • C12N2710/14011Baculoviridae
    • C12N2710/14041Use of virus, viral particle or viral elements as a vector
    • C12N2710/14043Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vectore
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14121Viruses as such, e.g. new isolates, mutants or their genomic sequences
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14151Methods of production or purification of viral material
    • C12N2750/14152Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/48Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/50Vector 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)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Neurology (AREA)
  • Medicinal Chemistry (AREA)
  • Neurosurgery (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Psychology (AREA)
  • Epidemiology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Hospice & Palliative Care (AREA)
  • Immunology (AREA)
  • Psychiatry (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
KR1020207014683A 2017-10-23 2018-10-23 신경변성 질환에 대한 유전자 요법 Pending KR20200075865A (ko)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201762575795P 2017-10-23 2017-10-23
US62/575,795 2017-10-23
US201862742723P 2018-10-08 2018-10-08
US62/742,723 2018-10-08
PCT/US2018/057187 WO2019084068A1 (en) 2017-10-23 2018-10-23 GENE THERAPIES FOR NEURODEGENERATIVE DISEASE

Publications (1)

Publication Number Publication Date
KR20200075865A true KR20200075865A (ko) 2020-06-26

Family

ID=66247008

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020207014683A Pending KR20200075865A (ko) 2017-10-23 2018-10-23 신경변성 질환에 대한 유전자 요법

Country Status (11)

Country Link
US (1) US20210261981A1 (https=)
EP (1) EP3701030A4 (https=)
JP (2) JP7413256B2 (https=)
KR (1) KR20200075865A (https=)
CN (2) CN113005123A (https=)
AU (1) AU2018354195A1 (https=)
BR (1) BR112020008033A2 (https=)
CA (2) CA3177979A1 (https=)
IL (2) IL274129A (https=)
MX (1) MX2020004207A (https=)
WO (1) WO2019084068A1 (https=)

Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20220109347A (ko) 2021-01-28 2022-08-04 주식회사 에이브레인 신경 퇴행성 질환의 치료를 위한 유전자 요법
KR20220109346A (ko) 2021-01-28 2022-08-04 주식회사 에이브레인 신경 퇴행성 질환의 치료를 위한 유전자 요법
KR20230061109A (ko) 2021-10-28 2023-05-08 한국해양과학기술원 신규 화합물 및 이를 유효성분으로 포함하는 신경염증성 뇌질환 예방 또는 치료용 조성물

Families Citing this family (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3692075A4 (en) 2017-10-03 2022-02-09 Prevail Therapeutics, Inc. GENE THERAPIES FOR LYSOSOMAL DISEASES
BR112020006671A2 (pt) 2017-10-03 2020-12-01 Prevail Therapeutics, Inc. terapias genéticas para distúrbios lisossômicos
KR102709597B1 (ko) 2017-10-03 2024-09-26 프리베일 테라퓨틱스, 인크. 리소좀 장애를 위한 유전자 요법
MA54792A (fr) * 2018-03-09 2021-12-01 Avrobio Inc Compositions et méthodes pour le traitement de la maladie de parkinson
PL3953377T3 (pl) 2019-04-10 2026-02-16 Prevail Therapeutics, Inc. Terapie genowe zaburzeń lizosomalnych
CA3136117A1 (en) 2019-04-10 2020-10-15 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
MX2022004771A (es) * 2019-10-22 2022-10-07 Applied Genetic Tech Corporation Vectores de virus adeno-asociados (aav) de triple funcion para el tratamiento de enfermedades asociadas a c9orf72.
US20240294906A1 (en) * 2020-07-29 2024-09-05 Alnylam Pharmaceuticals, Inc. Atxn2 irna compositions and methods of use thereof for treating or preventing atxn2-associated neurodegenerative diseases
KR20230088393A (ko) 2020-10-15 2023-06-19 프리베일 테라퓨틱스, 인크. 재조합 아데노 연관 바이러스 조성물 및 이의 제조 방법
US20240044869A1 (en) 2020-10-15 2024-02-08 Prevail Therapeutics, Inc. Assay for measuring potency of gene therapy drug product
EP4252846A4 (en) 2020-11-06 2025-12-03 Sumitomo Pharma Co Ltd REGULATOR FOR THE EXPRESSION AND/OR FUNCTION OF THE RPS25 GENE
AR128239A1 (es) * 2022-01-10 2024-04-10 Univ Pennsylvania Composiciones y métodos útiles para el tratamiento de trastornos mediados por c9orf72
WO2023202637A1 (en) 2022-04-19 2023-10-26 Shanghai Vitalgen Biopharma Co., Ltd. Recombinant aav vectors for treating neurodegenerative disorders
TW202511485A (zh) 2023-06-16 2025-03-16 日商住友製藥股份有限公司 調節rps25基因之表現及/或其功能的反義寡核苷酸

Family Cites Families (22)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6204059B1 (en) * 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
US20040053876A1 (en) * 2002-03-26 2004-03-18 The Regents Of The University Of Michigan siRNAs and uses therof
US7829694B2 (en) * 2002-11-26 2010-11-09 Medtronic, Inc. Treatment of neurodegenerative disease through intracranial delivery of siRNA
WO2004069173A2 (en) * 2003-01-31 2004-08-19 The Trustees Of The University Of Pennsylvania Methods for modulating an inflammatory response
EP1844148A2 (en) * 2005-01-31 2007-10-17 University of Iowa Research Foundation Nucleic acid silencing of huntington's disease gene
JP5559185B2 (ja) * 2008-11-11 2014-07-23 オックスフォード バイオメディカ(ユーケー)リミテッド 方法
US20110142789A1 (en) * 2009-12-10 2011-06-16 The Trustees Of The University Of Pennsylvania Compositions and Methods for the Diagnosis and Treatment of Amyotrophic Lateral Sclerosis
US8865881B2 (en) * 2011-02-22 2014-10-21 California Institute Of Technology Delivery of proteins using adeno-associated virus (AAV) vectors
SMT201700339T1 (it) * 2012-04-23 2017-09-07 Biomarin Tech Bv Oligonucleotidi di modulazione dell'rna con caratteristiche migliorate per il trattamento dei disturbi neuromuscolari
JP2016503405A (ja) * 2012-11-05 2016-02-04 ジェンザイム・コーポレーション タンパク質症を処置するための組成物および方法
US20140271550A1 (en) * 2013-03-14 2014-09-18 The Trustees Of The University Of Pennsylvania Constructs and Methods for Delivering Molecules via Viral Vectors with Blunted Innate Immune Responses
EP4166667A3 (en) * 2013-10-11 2023-08-02 Ionis Pharmaceuticals, Inc. Compositions for modulating c9orf72 expression
EP4410805A3 (en) * 2014-03-18 2024-11-27 University of Massachusetts Raav-based compositions and methods for treating amyotrophic lateral sclerosis
CN106103717A (zh) * 2014-03-19 2016-11-09 Ionis制药公司 用于调节共济失调蛋白2表达的组合物
WO2015153760A2 (en) * 2014-04-01 2015-10-08 Sangamo Biosciences, Inc. Methods and compositions for prevention or treatment of a nervous system disorder
WO2016024205A1 (en) * 2014-08-15 2016-02-18 Pfizer Inc. Oligomers targeting hexanucleotide repeat expansion in human c9orf72 gene
CA3193811A1 (en) * 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
CA3019315A1 (en) * 2015-04-23 2016-10-27 University Of Massachusetts Modulation of aav vector transgene expression
EP3292206B8 (en) * 2015-05-07 2022-02-09 Takeda Pharmaceutical Company Limited Glucocerebrosidase gene therapy for parkinson's disease
WO2017151884A1 (en) * 2016-03-02 2017-09-08 The Children's Hospital Of Philadelphia Therapy for frontotemporal dementia
JP7026678B2 (ja) * 2016-09-30 2022-02-28 リジェネロン・ファーマシューティカルズ・インコーポレイテッド C9orf72座位中にヘキサヌクレオチドリピート伸長を有する非ヒト動物
EP3850098A1 (en) * 2018-09-12 2021-07-21 uniQure IP B.V. Rnai induced c9orf72 suppression for the treatment of als/ftd

Cited By (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20220109347A (ko) 2021-01-28 2022-08-04 주식회사 에이브레인 신경 퇴행성 질환의 치료를 위한 유전자 요법
WO2022164260A1 (ko) 2021-01-28 2022-08-04 주식회사 에이브레인 신경 퇴행성 질환의 치료를 위한 유전자 요법
KR20220109346A (ko) 2021-01-28 2022-08-04 주식회사 에이브레인 신경 퇴행성 질환의 치료를 위한 유전자 요법
WO2022164259A1 (ko) 2021-01-28 2022-08-04 주식회사 에이브레인 신경 퇴행성 질환의 치료를 위한 유전자 요법
KR20230061109A (ko) 2021-10-28 2023-05-08 한국해양과학기술원 신규 화합물 및 이를 유효성분으로 포함하는 신경염증성 뇌질환 예방 또는 치료용 조성물

Also Published As

Publication number Publication date
BR112020008033A2 (pt) 2020-10-27
CA3083582A1 (en) 2019-05-02
EP3701030A4 (en) 2022-04-20
JP2021500049A (ja) 2021-01-07
US20210261981A1 (en) 2021-08-26
CN111819281B (zh) 2024-12-31
IL281057A (en) 2021-04-29
CN111819281A (zh) 2020-10-23
CN113005123A (zh) 2021-06-22
MX2020004207A (es) 2020-11-11
JP7413256B2 (ja) 2024-01-15
WO2019084068A1 (en) 2019-05-02
JP2024045131A (ja) 2024-04-02
IL274129A (en) 2020-06-30
AU2018354195A1 (en) 2020-06-04
CA3177979A1 (en) 2019-05-02
EP3701030A1 (en) 2020-09-02

Similar Documents

Publication Publication Date Title
CN111819281B (zh) 用于神经变性疾病的基因疗法
AU2023214366B2 (en) Gene therapies for lysosomal disorders
KR102879598B1 (ko) 무세포 합성으로부터 수득된 폐쇄 말단 DNA 벡터 및 ceDNA 벡터를 수득하는 방법
AU2020260485B2 (en) Gene therapies for lysosomal disorders
AU2020205228B2 (en) Gene therapies for lysosomal disorders
KR20220006527A (ko) 리소좀 장애에 대한 유전자 요법
JP2024028931A (ja) 閉端dna(cedna)ベクターを使用した導入遺伝子の制御された発現
CN116157527A (zh) 用于溶酶体病症的基因疗法
HK40055729A (en) Gene therapies for neurodegenerative disease
HK40039908A (en) Gene therapies for neurodegenerative disease
RU2820586C2 (ru) ДНК-ВЕКТОРЫ С ЗАМКНУТЫМИ КОНЦАМИ, ПОЛУЧАЕМЫЕ ПУТЕМ БЕСКЛЕТОЧНОГО СИНТЕЗА, И СПОСОБ ПОЛУЧЕНИЯ зкДНК-ВЕКТОРОВ
RU2816871C2 (ru) КОНТРОЛИРУЕМАЯ ЭКСПРЕССИЯ ТРАНСГЕНОВ С ИСПОЛЬЗОВАНИЕМ ДНК-ВЕКТОРОВ С ЗАМКНУТЫМИ КОНЦАМИ (зкДНК)
HK40047428A (en) Gene therapies for lysosomal disorders
HK40048832B (zh) 用於溶酶体障碍的基因疗法
HK40048832A (en) Gene therapies for lysosomal disorders
HK40046514A (en) Gene therapies for lysosomal disorders
KR20210150487A (ko) 리소좀 장애를 위한 유전자 요법
HK40067825A (zh) 用於溶酶体障碍的基因疗法

Legal Events

Date Code Title Description
PA0105 International application

Patent event date: 20200522

Patent event code: PA01051R01D

Comment text: International Patent Application

PG1501 Laying open of application
A201 Request for examination
PA0201 Request for examination

Patent event code: PA02012R01D

Patent event date: 20211021

Comment text: Request for Examination of Application

E902 Notification of reason for refusal
PE0902 Notice of grounds for rejection

Comment text: Notification of reason for refusal

Patent event date: 20230911

Patent event code: PE09021S01D

E701 Decision to grant or registration of patent right
PE0701 Decision of registration

Patent event code: PE07011S01D

Comment text: Decision to Grant Registration

Patent event date: 20240516