KR20200075865A - 신경변성 질환에 대한 유전자 요법 - Google Patents

신경변성 질환에 대한 유전자 요법 Download PDF

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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
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nucleic acid
isolated nucleic
vector
itr
c9orf72
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Korean (ko)
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아사 아벨리오비치
로라 헤크만
허브 린
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프리베일 테라퓨틱스, 인크.
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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

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KR20200075865A true KR20200075865A (ko) 2020-06-26

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US (1) US20210261981A1 (enExample)
EP (1) EP3701030A4 (enExample)
JP (2) JP7413256B2 (enExample)
KR (1) KR20200075865A (enExample)
CN (2) CN111819281B (enExample)
AU (1) AU2018354195A1 (enExample)
BR (1) BR112020008033A2 (enExample)
CA (2) CA3083582A1 (enExample)
IL (2) IL274129A (enExample)
MX (1) MX2020004207A (enExample)
WO (1) WO2019084068A1 (enExample)

Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2022164259A1 (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
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 (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP7361037B2 (ja) 2017-10-03 2023-10-13 プリベイル セラピューティクス，インコーポレーテッド ライソゾーム病の遺伝子治療
US11802294B2 (en) * 2017-10-03 2023-10-31 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
KR20200078513A (ko) 2017-10-03 2020-07-01 프리베일 테라퓨틱스, 인크. 리소좀 장애를 위한 유전자 요법
US20210000929A1 (en) * 2018-03-09 2021-01-07 Avrobio, Inc. Compositions and methods for treating parkinson's disease
CN114174324A (zh) 2019-04-10 2022-03-11 普利维尔治疗公司 用于溶酶体病症的基因疗法
BR112021019880A2 (pt) 2019-04-10 2022-02-15 Prevail Therapeutics Inc Terapias gênicas para distúrbios lisossomais
WO2021081236A1 (en) * 2019-10-22 2021-04-29 Applied Genetic Technologies Corporation Triple function adeno-associated virus (aav) vectors for the treatment of c9orf72 associated diseases
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
CN116710567A (zh) 2020-10-15 2023-09-05 普利维尔治疗公司 重组腺相关病毒组合物及其产生方法
WO2022081985A1 (en) 2020-10-15 2022-04-21 Prevail Therapeutics, Inc. Assay for measuring potency of gene therapy drug product
JPWO2022097727A1 (enExample) 2020-11-06 2022-05-12
WO2023133574A1 (en) * 2022-01-10 2023-07-13 The Trustees Of The University Of Pennsylvania Compositions and methods useful for treatment of c9orf72-mediated disorders
WO2024257847A1 (ja) 2023-06-16 2024-12-19 住友ファーマ株式会社 Ｒｐｓ２５遺伝子の発現及び／又はその機能を調節するアンチセンスオリゴヌクレオチド

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
AU2006210973A1 (en) * 2005-01-31 2006-08-10 University Of Iowa Research Foundation Nucleic acid silencing of Huntington's Disease gene
WO2010055290A1 (en) * 2008-11-11 2010-05-20 Oxford Biomedica (Uk) Limited Method
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
EP2678433B1 (en) * 2011-02-22 2017-05-03 California Institute of Technology Delivery of proteins using adeno-associated virus (aav) vectors
HRP20171254T1 (hr) * 2012-04-23 2018-01-12 Biomarin Technologies B.V. Oligonukleotidi koji moduliraju rnk s poboljšanim karakteristikama za liječenje neuromišićnih poremećaja
MA38144A1 (fr) * 2012-11-05 2018-08-31 Genzyme Corp Compositions et procédés pour le traitement de protéinopathies
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
RU2748426C2 (ru) * 2013-10-11 2021-05-25 Ионис Фармасьютикалз, Инк. Композиции для модуляции экспрессии c90rf72
EP4410805A3 (en) * 2014-03-18 2024-11-27 University of Massachusetts Raav-based compositions and methods for treating amyotrophic lateral sclerosis
PL3119888T3 (pl) * 2014-03-19 2021-12-20 Ionis Pharmaceuticals, Inc. Kompozycje do modulacji ekspresji ataksyny 2
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
US10597660B2 (en) * 2014-11-14 2020-03-24 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
WO2016172155A1 (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
CA3016314A1 (en) * 2016-03-02 2017-09-08 Julianne REIDERS Therapy for frontotemporal dementia
EP3518667B1 (en) * 2016-09-30 2024-10-30 Regeneron Pharmaceuticals, Inc. Rodents having a hexanucleotide repeat expansion in a c9orf72 locus
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
WO2022164259A1 (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
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

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