JP2016503405A - タンパク質症を処置するための組成物および方法 - Google Patents

タンパク質症を処置するための組成物および方法 Download PDF

Info

Publication number
JP2016503405A
JP2016503405A JP2015540845A JP2015540845A JP2016503405A JP 2016503405 A JP2016503405 A JP 2016503405A JP 2015540845 A JP2015540845 A JP 2015540845A JP 2015540845 A JP2015540845 A JP 2015540845A JP 2016503405 A JP2016503405 A JP 2016503405A
Authority
JP
Japan
Prior art keywords
gba1
aav
disease
protein
agent
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.)
Abandoned
Application number
JP2015540845A
Other languages
English (en)
Japanese (ja)
Other versions
JP2016503405A5 (enExample
Inventor
セルジオ・パブロ・サルディ
ラミヤ・シハブッディン
セン・チェン
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.)
Genzyme Corp
Original Assignee
Genzyme Corp
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
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=49578599&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=JP2016503405(A) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Genzyme Corp filed Critical Genzyme Corp
Publication of JP2016503405A publication Critical patent/JP2016503405A/ja
Publication of JP2016503405A5 publication Critical patent/JP2016503405A5/ja
Abandoned legal-status Critical Current

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/40—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against enzymes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • 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
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • 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
    • 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
    • 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
    • A61P25/18—Antipsychotics, i.e. neuroleptics; Drugs for mania or schizophrenia
    • 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
    • A61P25/28—Drugs 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
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • 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)
    • C12Y302/01045—Glucosylceramidase (3.2.1.45), i.e. beta-glucocerebrosidase
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Epidemiology (AREA)
  • Biotechnology (AREA)
  • Microbiology (AREA)
  • Virology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Psychiatry (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Mycology (AREA)
  • Dermatology (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
JP2015540845A 2012-11-05 2013-11-04 タンパク質症を処置するための組成物および方法 Abandoned JP2016503405A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201261722434P 2012-11-05 2012-11-05
US61/722,434 2012-11-05
PCT/US2013/068242 WO2014071282A1 (en) 2012-11-05 2013-11-04 Compositions and methods for treating proteinopathies

Publications (2)

Publication Number Publication Date
JP2016503405A true JP2016503405A (ja) 2016-02-04
JP2016503405A5 JP2016503405A5 (enExample) 2016-12-22

Family

ID=49578599

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2015540845A Abandoned JP2016503405A (ja) 2012-11-05 2013-11-04 タンパク質症を処置するための組成物および方法

Country Status (22)

Country Link
US (1) US20150284472A1 (enExample)
EP (1) EP2914281A1 (enExample)
JP (1) JP2016503405A (enExample)
KR (1) KR20150079751A (enExample)
CN (1) CN104902923A (enExample)
AR (1) AR093355A1 (enExample)
AU (1) AU2013337354A1 (enExample)
BR (1) BR112015009746A2 (enExample)
CA (1) CA2889990A1 (enExample)
CL (1) CL2015001157A1 (enExample)
CR (1) CR20150216A (enExample)
EA (1) EA201590880A1 (enExample)
HK (1) HK1214521A1 (enExample)
IL (1) IL238416A0 (enExample)
MA (1) MA38144A1 (enExample)
MX (1) MX2015005722A (enExample)
PH (1) PH12015500879A1 (enExample)
SG (1) SG11201502989XA (enExample)
TN (1) TN2015000171A1 (enExample)
TW (1) TW201427695A (enExample)
WO (1) WO2014071282A1 (enExample)
ZA (1) ZA201502618B (enExample)

Cited By (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2019524775A (ja) * 2016-08-03 2019-09-05 ユニヴァーシティ オブ サウス フロリダ 神経障害治療用のリーリン組成物
JP2019527821A (ja) * 2016-07-01 2019-10-03 セントジーン アーゲー ドラッガブルターゲットとしてのリゾＧｂ１の使用
JP2020537543A (ja) * 2017-10-03 2020-12-24 プリベイル セラピューティクス, インコーポレーテッドPrevail Therapeutics, Inc. ライソゾーム病の遺伝子治療
JP2020537544A (ja) * 2017-10-03 2020-12-24 プリベイル セラピューティクス, インコーポレーテッドPrevail Therapeutics, Inc. ライソゾーム病の遺伝子治療
JP2021500049A (ja) * 2017-10-23 2021-01-07 プリベイル セラピューティクス, インコーポレーテッドPrevail Therapeutics, Inc. 神経変性疾患の遺伝子治療
JP2021501135A (ja) * 2017-10-26 2021-01-14 シャイア ヒューマン ジェネティック セラピーズ インコーポレイテッド グルコセレブロシダーゼ及びイソファゴミンを含む製剤
JP2021059586A (ja) * 2016-04-29 2021-04-15 オーファザイム エー／エス グルコセレブロシダーゼ関連障害を治療するためのアリモクロモル
JP2022526021A (ja) * 2019-04-10 2022-05-20 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
JP2023545462A (ja) * 2020-10-14 2023-10-30 デナリ セラピューティクス インコーポレイテッド 前頭側頭型認知症を治療及びモニタリングするための方法

Families Citing this family (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
FI3578195T3 (fi) 2008-06-26 2023-11-08 Zevra Denmark As Hsp70:n käyttö entsyymitoiminnan säätelijänä
RU2013125923A (ru) 2010-11-30 2015-01-10 Орфазиме Апс СПОСОБЫ УВЕЛИЧЕНИЯ ВНУТРИКЛЕТОЧНОЙ АКТИВНОСТИ Hsp70
JP6678676B2 (ja) 2014-09-15 2020-04-08 オーファザイム エー／エス アリモクロモル製剤
EP3292206B8 (en) * 2015-05-07 2022-02-09 Takeda Pharmaceutical Company Limited Glucocerebrosidase gene therapy for parkinson's disease
US10874749B2 (en) * 2015-07-21 2020-12-29 Thomas Jefferson University Gene therapies for neurodegenerative disorders targeting ganglioside biosynthetic pathways
EP3405898A1 (en) * 2016-01-21 2018-11-28 Protein Dynamic Solutions LLC Method and system for spectral data analysis
WO2017178029A1 (en) 2016-04-13 2017-10-19 Orphazyme Aps Heat shock proteins and cholesterol homeostasis
CN111465691A (zh) 2017-10-03 2020-07-28 普利维尔治疗公司 用于溶酶体障碍的基因疗法
US20210041461A1 (en) * 2018-03-14 2021-02-11 Yissum Research Developmen Company Of The Hebrew University Of Jerusalem Ltd Detection of phospho-serine 129 alpha-synuclein in blood cells as a biomarker for synucleinopathies
HRP20251506T1 (hr) 2019-04-10 2026-01-02 Prevail Therapeutics, Inc. Genska terapija za lizosomske poremećaje
EP3986412A4 (en) 2019-06-21 2023-11-15 The Broad Institute, Inc. AGENTS TO REVERSE TOXIC PROTEINOPATHIES
US20230277687A1 (en) 2020-08-06 2023-09-07 Fundacion Para La Investigacion Medica Aplicada Viral particles for use in treating tauopathies such as alzheimer's diseases by gene therapy
IL303026A (en) 2020-11-19 2023-07-01 Zevra Denmark As Processes for preparing arimoclomol citrate and intermediates thereof
CN112569354B (zh) * 2020-12-29 2022-06-10 四川大学华西医院 tau蛋白及其基因作为药物靶点在制备治疗糖尿病药物中的应用
EP4448763A1 (en) * 2021-12-17 2024-10-23 F. Hoffmann-La Roche AG Oligonucleotide gba agonists
EP4448762A1 (en) * 2021-12-17 2024-10-23 F. Hoffmann-La Roche AG Oligonucleotides capable of increasing glucocerebrosidase expression

Family Cites Families (33)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4816567A (en) 1983-04-08 1989-03-28 Genentech, Inc. Recombinant immunoglobin preparations
US4683195A (en) 1986-01-30 1987-07-28 Cetus Corporation Process for amplifying, detecting, and/or-cloning nucleic acid sequences
US4676980A (en) 1985-09-23 1987-06-30 The United States Of America As Represented By The Secretary Of The Department Of Health And Human Services Target specific cross-linked heteroantibodies
US5225539A (en) 1986-03-27 1993-07-06 Medical Research Council Recombinant altered antibodies and methods of making altered antibodies
US5892019A (en) 1987-07-15 1999-04-06 The United States Of America, As Represented By The Department Of Health And Human Services Production of a single-gene-encoded immunoglobulin
GB8823869D0 (en) 1988-10-12 1988-11-16 Medical Res Council Production of antibodies
GB9015198D0 (en) 1990-07-10 1990-08-29 Brien Caroline J O Binding substance
US6172197B1 (en) 1991-07-10 2001-01-09 Medical Research Council Methods for producing members of specific binding pairs
US5625126A (en) 1990-08-29 1997-04-29 Genpharm International, Inc. Transgenic non-human animals for producing heterologous antibodies
US5633425A (en) 1990-08-29 1997-05-27 Genpharm International, Inc. Transgenic non-human animals capable of producing heterologous antibodies
US5661016A (en) 1990-08-29 1997-08-26 Genpharm International Inc. Transgenic non-human animals capable of producing heterologous antibodies of various isotypes
US5545806A (en) 1990-08-29 1996-08-13 Genpharm International, Inc. Ransgenic non-human animals for producing heterologous antibodies
DE69127627T2 (de) 1990-08-29 1998-02-19 Genpharm Int Produktion und Nützung nicht-menschliche transgentiere zur Produktion heterologe Antikörper
ATE164395T1 (de) 1990-12-03 1998-04-15 Genentech Inc Verfahren zur anreicherung von proteinvarianten mit geänderten bindungseigenschaften
ATE408012T1 (de) 1991-12-02 2008-09-15 Medical Res Council Herstellung von autoantikörpern auf phagenoberflächen ausgehend von antikörpersegmentbibliotheken
AU687829B2 (en) 1993-06-24 1998-03-05 Advec, Inc. Adenovirus vectors for gene therapy
JP3875990B2 (ja) 1993-10-25 2007-01-31 カンジ，インコーポレイテッド 組換えアデノウイルスベクターおよび使用方法
US7252989B1 (en) 1994-04-04 2007-08-07 Board Of Regents, The University Of Texas System Adenovirus supervector system
US6696248B1 (en) 1995-08-18 2004-02-24 Morphosys Ag Protein/(poly)peptide libraries
DK1143006T3 (da) 1995-08-18 2008-07-14 Morphosys Ip Gmbh Vektorer/DNA-sekvenser fra humane kombinatoriske antistofbiblioteker
US6924123B2 (en) 1996-10-29 2005-08-02 Oxford Biomedica (Uk) Limited Lentiviral LTR-deleted vector
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6995006B2 (en) 1997-09-05 2006-02-07 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
ATE482275T1 (de) 1999-07-02 2010-10-15 Morphosys Ag Erzeugung spezifischer bindungspartner die an von genomischen dns-fragmenten oder ests kodierten (poly)peptiden binden
ES2308989T3 (es) 1999-08-09 2008-12-16 Targeted Genetics Corporation Aumento de la expresion de una secuencia nucleotidica heterologa a partir de vectores viricos recombinantes que contienen una secuencia que forman pares de bases intracatenarios.
ES2505700T3 (es) 2000-06-01 2014-10-10 University Of North Carolina At Chapel Hill Vectores de parvovirus duplicados
WO2001097829A2 (en) 2000-06-19 2001-12-27 Genzyme Corporation Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases
US7446098B2 (en) * 2003-02-18 2008-11-04 Mount Sinai School Of Medicine Of New York University Combination therapy for treating protein deficiencies
US7765583B2 (en) 2005-02-28 2010-07-27 France Telecom System and method for managing virtual user domains
BRPI0713442A2 (pt) * 2006-06-23 2012-03-06 Amicus Therapeutics, Inc. Método para o tratamento de desrodens neurológicas, pelo aumento da atividade de b-glicocerebrosidase
PL2154969T3 (pl) * 2007-05-16 2016-04-29 Brigham & Womens Hospital Inc Leczenie synukleinopatii
WO2012177997A1 (en) 2011-06-22 2012-12-27 The General Hospital Corporation Treatment of proteinopathies
JP2015512913A (ja) 2012-03-28 2015-04-30 ザ ユナイテッド ステイツ オブ アメリカ， アズ リプレゼンテッド バイ ザ セクレタリー， デパートメント オブ ヘルス アンド ヒューマン サービシーズ グルコセレブロシダーゼ活性剤として有用なサリチル酸誘導体

Cited By (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2021059586A (ja) * 2016-04-29 2021-04-15 オーファザイム エー／エス グルコセレブロシダーゼ関連障害を治療するためのアリモクロモル
JP2019527821A (ja) * 2016-07-01 2019-10-03 セントジーン アーゲー ドラッガブルターゲットとしてのリゾＧｂ１の使用
JP2019524775A (ja) * 2016-08-03 2019-09-05 ユニヴァーシティ オブ サウス フロリダ 神経障害治療用のリーリン組成物
US12516091B2 (en) 2016-08-03 2026-01-06 University Of South Florida Reelin compositions for treatment of neurological disorders
JP7253800B2 (ja) 2016-08-03 2023-04-07 ユニヴァーシティ オブ サウス フロリダ 神経障害治療用のリーリン組成物
JP2023086740A (ja) * 2017-10-03 2023-06-22 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
JP2020537543A (ja) * 2017-10-03 2020-12-24 プリベイル セラピューティクス, インコーポレーテッドPrevail Therapeutics, Inc. ライソゾーム病の遺伝子治療
JP2020537544A (ja) * 2017-10-03 2020-12-24 プリベイル セラピューティクス, インコーポレーテッドPrevail Therapeutics, Inc. ライソゾーム病の遺伝子治療
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
JP7254815B2 (ja) 2017-10-03 2023-04-10 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
JP2021500049A (ja) * 2017-10-23 2021-01-07 プリベイル セラピューティクス, インコーポレーテッドPrevail Therapeutics, Inc. 神経変性疾患の遺伝子治療
JP7413256B2 (ja) 2017-10-23 2024-01-15 プリベイル セラピューティクス，インコーポレーテッド 神経変性疾患の遺伝子治療
JP2021501135A (ja) * 2017-10-26 2021-01-14 シャイア ヒューマン ジェネティック セラピーズ インコーポレイテッド グルコセレブロシダーゼ及びイソファゴミンを含む製剤
JP2022526021A (ja) * 2019-04-10 2022-05-20 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
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
JP2023545462A (ja) * 2020-10-14 2023-10-30 デナリ セラピューティクス インコーポレイテッド 前頭側頭型認知症を治療及びモニタリングするための方法

Also Published As

Publication number Publication date
KR20150079751A (ko) 2015-07-08
CN104902923A (zh) 2015-09-09
AU2013337354A1 (en) 2015-05-21
CA2889990A1 (en) 2014-05-08
CR20150216A (es) 2015-05-29
TW201427695A (zh) 2014-07-16
HK1214521A1 (zh) 2016-07-29
US20150284472A1 (en) 2015-10-08
EA201590880A1 (ru) 2015-09-30
ZA201502618B (en) 2016-01-27
MA38144A1 (fr) 2018-08-31
EP2914281A1 (en) 2015-09-09
CL2015001157A1 (es) 2015-10-16
IL238416A0 (en) 2015-06-30
MX2015005722A (es) 2016-01-12
SG11201502989XA (en) 2015-05-28
TN2015000171A1 (fr) 2016-10-03
WO2014071282A1 (en) 2014-05-08
PH12015500879A1 (en) 2015-06-29
AR093355A1 (es) 2015-06-03
BR112015009746A2 (pt) 2017-08-15

Similar Documents

Publication Publication Date Title
JP2016503405A (ja) タンパク質症を処置するための組成物および方法
JP7190352B2 (ja) 認知保護を提供しつつ疾患の発症及び進行を遅らせるための遺伝子治療を用いた神経変性疾患の治療方法
Neverman et al. Experimental therapies in the neuronal ceroid lipofuscinoses
EP3254702B1 (en) Aav/xbp1s-ha virus, gene therapy method and use thereof in the optimisation and improvement of learning, memory and cognitive capacities
US20230227802A1 (en) Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
WO2022098699A1 (en) Compositions and methods for the treatment of tauopathy
US20230044351A1 (en) Apoe gene therapy
US20160256571A1 (en) Invention
CA3183251A1 (en) Compositions and methods for the treatment of synucleinopathies
CN114728169A (zh) tau蛋白病的治疗
US20230285596A1 (en) Compositions and methods for the treatment of niemann-pick type c1 disease
JP2025535780A (ja) アルツハイマー病においてｒｅｅｌｉｎを使用する組成物および方法
OA17295A (en) Compositions and methods for treating proteinopathies.
KR102922353B1 (ko) 응집 억제를 위한 Aβ 변이체의 전달
WO2025233460A1 (en) Treatment and prevention of alpha-synuclein-mediated disease
Walker Gene Replacement Therapy in Mouse Models of Mitochondrial Encephalopathy
WO2025175135A1 (en) Modifying neurons to treat or prevent parkinson's disease
TW202449149A (zh) 用於治療與葡萄糖神經醯胺酶β１缺陷相關之神經病症的組合物及方法
WO2025016362A1 (zh) 基于脑内磷稳态促进剂的疗法
JP2022531177A (ja) 神経変性障害を治療するための方法
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
KR20230124010A (ko) 응집 억제를 위한 Aβ 변이체의 전달
KR20260020502A (ko) 글루코실세라미다제 베타 1 결핍과 관련된 장애의 치료를 위한 조성물 및 방법
JP2023548632A (ja) アルツハイマー病の処置のための組成物および方法

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20161031

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20161031

A762 Written abandonment of application

Free format text: JAPANESE INTERMEDIATE CODE: A762

Effective date: 20170531