SG11202011296VA - Compositions and methods for the treatment of parkinson's disease - Google Patents

Compositions and methods for the treatment of parkinson's disease

Info

Publication number
SG11202011296VA
SG11202011296VA SG11202011296VA SG11202011296VA SG11202011296VA SG 11202011296V A SG11202011296V A SG 11202011296VA SG 11202011296V A SG11202011296V A SG 11202011296VA SG 11202011296V A SG11202011296V A SG 11202011296VA SG 11202011296V A SG11202011296V A SG 11202011296VA
Authority
SG
Singapore
Prior art keywords
parkinson
disease
compositions
treatment
methods
Prior art date
Application number
SG11202011296VA
Other languages
English (en)
Inventor
Bernard Ravina
Adrian Philip Kells
Original Assignee
Voyager Therapeutics Inc
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 Voyager Therapeutics Inc filed Critical Voyager Therapeutics Inc
Publication of SG11202011296VA publication Critical patent/SG11202011296VA/en

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
    • 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
    • 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
    • A61K38/51—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
    • A61P25/16—Anti-Parkinson drugs
    • 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
    • C12N15/62—DNA sequences coding for fusion proteins
    • 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
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biochemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Virology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Psychology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
SG11202011296VA 2018-05-15 2019-05-15 Compositions and methods for the treatment of parkinson's disease SG11202011296VA (en)

Applications Claiming Priority (12)

Application Number Priority Date Filing Date Title
US201862671944P 2018-05-15 2018-05-15
US201862681891P 2018-06-07 2018-06-07
US201862684384P 2018-06-13 2018-06-13
US201862691748P 2018-06-29 2018-06-29
US201862698419P 2018-07-16 2018-07-16
US201862703137P 2018-07-25 2018-07-25
US201862741021P 2018-10-04 2018-10-04
US201862748119P 2018-10-19 2018-10-19
US201862756897P 2018-11-07 2018-11-07
US201962789909P 2019-01-08 2019-01-08
US201962831400P 2019-04-09 2019-04-09
PCT/US2019/032384 WO2019222328A1 (en) 2018-05-15 2019-05-15 Compositions and methods for the treatment of parkinson's disease

Publications (1)

Publication Number Publication Date
SG11202011296VA true SG11202011296VA (en) 2020-12-30

Family

ID=67060450

Family Applications (1)

Application Number Title Priority Date Filing Date
SG11202011296VA SG11202011296VA (en) 2018-05-15 2019-05-15 Compositions and methods for the treatment of parkinson's disease

Country Status (13)

Country Link
US (1) US20210198691A1 (es)
EP (1) EP3794126A1 (es)
JP (1) JP2021523914A (es)
KR (1) KR20210019996A (es)
CN (1) CN112424359A (es)
AU (1) AU2019268330A1 (es)
BR (1) BR112020023082A2 (es)
CA (1) CA3099306A1 (es)
MA (1) MA52631A (es)
MX (1) MX2020012077A (es)
SG (1) SG11202011296VA (es)
TW (1) TW202012628A (es)
WO (1) WO2019222328A1 (es)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3215191A4 (en) 2014-11-05 2018-08-01 Voyager Therapeutics, Inc. Aadc polynucleotides for the treatment of parkinson's disease
TWI744300B (zh) * 2016-03-23 2021-11-01 里爾中央醫學中心 改良性熱處理的血小板顆粒裂解液在製備用於治療神經系統疾病的組合物的用途
EP3510161A4 (en) 2016-08-23 2020-04-22 Akouos, Inc. COMPOSITIONS AND METHODS FOR TREATING PERSONAL HEARING LOSS IN A PERSON
CA3059891A1 (en) 2017-04-14 2018-10-18 National Taiwan University Gene therapy for aadc deficiency
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
WO2021007382A1 (en) * 2019-07-09 2021-01-14 Voyager Therapeutics, Inc. Compositions and methods for the treatment of parkinson's disease
MX2022010050A (es) 2020-02-21 2022-09-05 Akouos Inc Composiciones y metodos para tratar deficiencia auditiva no asociada con la edad en un sujeto humano.
CN115607684A (zh) * 2021-07-15 2023-01-17 华中科技大学 一种内耳药物纳米载体及其应用
CN116554278A (zh) * 2022-01-30 2023-08-08 上海玮美基因科技有限责任公司 变异型腺相关病毒及其在疾病治疗中的应用

Family Cites Families (148)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US3467096A (en) 1966-04-12 1969-09-16 Ferrell S Horn Multiple hypodermic syringe arrangement
BE786542A (fr) 1971-07-22 1973-01-22 Dow Corning Dispositif d'aspiration permettant d'obtenir des echantillons de cellules
US4411657A (en) 1980-05-19 1983-10-25 Anibal Galindo Hypodermic needle
US4474569A (en) 1982-06-28 1984-10-02 Denver Surgical Developments, Inc. Antenatal shunt
US5078140A (en) 1986-05-08 1992-01-07 Kwoh Yik S Imaging device - aided robotic stereotaxis system
FR2640638B1 (fr) 1988-12-20 1991-02-15 Commissariat Energie Atomique Bioreacteur et dispositif pour la culture de cellules animales
US5012818A (en) 1989-05-04 1991-05-07 Joishy Suresh K Two in one bone marrow surgical needle
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
US5199441A (en) 1991-08-20 1993-04-06 Hogle Hugh H Fine needle aspiration biopsy apparatus and method
US5387484A (en) 1992-07-07 1995-02-07 International Business Machines Corporation Two-sided mask for patterning of materials with electromagnetic radiation
AU5665694A (en) 1992-11-04 1994-05-24 Denver Biomaterials Inc. Apparatus for removal of pleural effusion fluid
FR2703253B1 (fr) 1993-03-30 1995-06-23 Centre Nat Rech Scient Applicateur d'impulsions electriques pour traitement de tissus biologiques.
US6204059B1 (en) 1994-06-30 2001-03-20 University Of Pittsburgh AAV capsid vehicles for molecular transfer
US5625048A (en) 1994-11-10 1997-04-29 The Regents Of The University Of California Modified green fluorescent proteins
US5588960A (en) 1994-12-01 1996-12-31 Vidamed, Inc. Transurethral needle delivery device with cystoscope and method for treatment of urinary incontinence
JPH10511264A (ja) 1994-12-06 1998-11-04 ターゲティッド ジェネティックス コーポレイション 高力価組換えａａｖベクターの生成のためのパッケージング細胞株
DE69629326D1 (de) 1995-02-15 2003-09-11 Joseph Eldor Spinalnadel mit mehreren Löchern
US5741657A (en) 1995-03-20 1998-04-21 The Regents Of The University Of California Fluorogenic substrates for β-lactamase and methods of use
US5756283A (en) 1995-06-05 1998-05-26 The Trustees Of The University Of Pennsylvania Method for improved production of recombinant adeno-associated viruses for gene therapy
US6281010B1 (en) 1995-06-05 2001-08-28 The Trustees Of The University Of Pennsylvania Adenovirus gene therapy vehicle and cell line
US6506379B1 (en) 1995-06-07 2003-01-14 Ariad Gene Therapeutics, Inc. Intramuscular delivery of recombinant AAV
US5741683A (en) 1995-06-07 1998-04-21 The Research Foundation Of State University Of New York In vitro packaging of adeno-associated virus DNA
US5688676A (en) 1995-06-07 1997-11-18 Research Foundation Of State University Of New York In vitro packaging of adeno-associated virus DNA
US6265389B1 (en) 1995-08-31 2001-07-24 Alkermes Controlled Therapeutics, Inc. Microencapsulation and sustained release of oligonucleotides
US5846528A (en) 1996-01-18 1998-12-08 Avigen, Inc. Treating anemia using recombinant adeno-associated virus virions comprising an EPO DNA sequence
US5858351A (en) 1996-01-18 1999-01-12 Avigen, Inc. Methods for delivering DNA to muscle cells using recombinant adeno-associated virus vectors
AU722624B2 (en) 1996-09-06 2000-08-10 Trustees Of The University Of Pennsylvania, The An inducible method for production of recombinant adeno-associated viruses utilizing T7 polymerase
US20030215422A1 (en) 1996-09-11 2003-11-20 John A. Chiorini Aav4 vector and uses thereof
US5865744A (en) 1996-09-16 1999-02-02 Lemelson; Jerome H. Method and system for delivering therapeutic agents
AU732703B2 (en) 1996-11-20 2001-04-26 Crucell Holland B.V. An improved method for the production and purification of adenoviral vectors
WO1998027204A2 (en) 1996-12-18 1998-06-25 Targeted Genetics Corporation Aav split-packaging genes and cell lines comprising such genes for use in the production of recombinant aav vectors
US5759179A (en) 1996-12-31 1998-06-02 Johnson & Johnson Medical, Inc. Needle and valve assembly for use with a catheter
EP0969876A2 (en) 1997-04-17 2000-01-12 Paola Leone Delivery system for gene therapy to the brain
US6156303A (en) 1997-06-11 2000-12-05 University Of Washington Adeno-associated virus (AAV) isolates and AAV vectors derived therefrom
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
AU9319198A (en) 1997-09-19 1999-04-05 Trustees Of The University Of Pennsylvania, The Methods and vector constructs useful for production of recombinant aav
CA2304168A1 (en) 1997-09-19 1999-04-01 The Trustees Of The University Of Pennsylvania Methods and cell line useful for production of recombinant adeno-associated viruses
AU3097399A (en) 1998-03-20 1999-10-11 Trustees Of The University Of Pennsylvania, The Compositions and methods for helper-free production of recombinant adeno-associated viruses
US6953690B1 (en) 1998-03-20 2005-10-11 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
EP1621626B1 (en) * 1998-05-27 2009-07-08 Genzyme Corporation AAV Vectors for the manufacture of medicaments for convection enhanced delivery
WO1999061066A2 (en) * 1998-05-27 1999-12-02 Avigen, Inc. Convection-enhanced delivery of aav vectors
GB9817662D0 (en) 1998-08-13 1998-10-07 Crocker Peter J Substance delivery
EP1124976A1 (en) 1998-10-27 2001-08-22 Crucell Holland B.V. Improved aav vector production
WO2000028004A1 (en) 1998-11-10 2000-05-18 The University Of North Carolina At Chapel Hill Virus vectors and methods of making and administering the same
DE19905501B4 (de) 1999-02-10 2005-05-19 MediGene AG, Gesellschaft für molekularbiologische Kardiologie und Onkologie Verfahren zur Herstellung eines rekombinanten Adeno-assoziierten Virus, geeignete Mittel hierzu sowie Verwendung zur Herstellung eines Arzneimittels
JP4693244B2 (ja) 1999-03-18 2011-06-01 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 組換えアデノ随伴ウイルスのヘルパー無しの生産のための組成物および方法
US6258595B1 (en) 1999-03-18 2001-07-10 The Trustees Of The University Of Pennsylvania Compositions and methods for helper-free production of recombinant adeno-associated viruses
EP1183380A1 (en) 1999-06-02 2002-03-06 The Trustees Of The University Of Pennsylvania Compositions and methods useful for production of recombinant viruses which require helper viruses
US6365394B1 (en) 1999-09-29 2002-04-02 The Trustees Of The University Of Pennsylvania Cell lines and constructs useful in production of E1-deleted adenoviruses in absence of replication competent adenovirus
CA2384814A1 (en) 1999-09-29 2001-04-05 The Trustees Of The University Of Pennsylvania Methods for rapid peg-modification of viral vectors, compositions for enhanced gene transduction, compositions with enhanced physical stability, and uses therefor
US6613026B1 (en) 1999-12-08 2003-09-02 Scimed Life Systems, Inc. Lateral needle-less injection apparatus and method
US6726659B1 (en) 1999-12-09 2004-04-27 John E. Stocking Catheter assembly having a fenestrated dilator
US6382212B1 (en) 2000-01-18 2002-05-07 Medtronic, Inc. Fenestrated surgical drape with in situ features
US6565572B2 (en) 2000-04-10 2003-05-20 Sdgi Holdings, Inc. Fenestrated surgical screw and method
AU6808001A (en) 2000-05-23 2001-12-03 Neurologix Inc Glutamic acid decarboxylase (gad) based delivery systems
US7588757B2 (en) 2001-03-14 2009-09-15 Genzyme Corporation Methods of treating Parkinson's disease using recombinant adeno-associated virus virions
US6520949B2 (en) 2001-04-02 2003-02-18 Martin St. Germain Method and apparatus for administering fluid to animals subcutaneously
EP1390490B1 (en) 2001-05-24 2009-04-15 Genzyme Corporation Muscle-specific expression vectors
PL220644B1 (pl) 2001-11-13 2015-11-30 Univ Pennsylvania Wirus stowarzyszony z adenowirusem (AAV), kompozycja, wyizolowane białko kapsydowe, wyizolowane lub syntetyczne cząsteczki kwasu nukleinowego, sposób wytwarzania zrekombinowanego wirusa, komórka gospodarza
EP3339430A1 (en) 2001-12-17 2018-06-27 The Trustees of The University of Pennsylvania Adeno-associated virus (aav) serotype 9 sequences, vectors containing same, and uses thereof
US7605249B2 (en) 2002-11-26 2009-10-20 Medtronic, Inc. Treatment of neurodegenerative disease through intracranial delivery of siRNA
US8946151B2 (en) 2003-02-24 2015-02-03 Northern Bristol N.H.S. Trust Frenchay Hospital Method of treating Parkinson's disease in humans by convection-enhanced infusion of glial cell-line derived neurotrophic factor to the putamen
US8927269B2 (en) 2003-05-19 2015-01-06 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Avian adenoassociated virus and uses thereof
ATE490307T1 (de) 2003-05-21 2010-12-15 Genzyme Corp Verfahren zur herstellung von präparationen rekombinanter aav-virionen, die weitgehend frei von leeren capsiden sind
EP2357189B1 (en) 2003-06-19 2017-03-08 Genzyme Corporation AAV virions with decreased immunoreactivity and uses therefor
US7291498B2 (en) 2003-06-20 2007-11-06 The Trustees Of The University Of Pennsylvania Methods of generating chimeric adenoviruses and uses for such chimeric adenoviruses
US7491508B2 (en) 2003-06-20 2009-02-17 The Trustees Of The University Of Pennsylvania Methods of generating chimeric adenoviruses and uses for such chimeric adenoviruses
US9441244B2 (en) 2003-06-30 2016-09-13 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
US9233131B2 (en) 2003-06-30 2016-01-12 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
US8333734B2 (en) 2003-07-03 2012-12-18 Walter A. Zohmann Fenestrated peripheral nerve block needle and method for using the same
US7575572B2 (en) 2003-07-15 2009-08-18 Spinal Generations, Llc Method and device for delivering medicine to bone
US7906111B2 (en) 2003-09-30 2011-03-15 The Trustees Of The University Of Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses therefor
US7135010B2 (en) 2003-09-30 2006-11-14 Damage Control Surgical Technologies, Inc. Method and apparatus for rapid deployment chest drainage
US7699852B2 (en) 2003-11-19 2010-04-20 Zimmer Spine, Inc. Fenestrated bone tap and method
US8137960B2 (en) 2003-12-04 2012-03-20 The United States Of America As Represented By The Department Of Health And Human Services Bovine adeno-associated viral (BAAV) vector and uses thereof
US20060166363A1 (en) 2004-01-27 2006-07-27 Sergei Zolotukhin Modified baculovirus expression system for production of pseudotyped rAAV vector
US7427396B2 (en) 2004-06-03 2008-09-23 Genzyme Corporation AAV vectors for gene delivery to the lung
EP4234687A2 (en) 2005-04-07 2023-08-30 The Trustees of the University of Pennsylvania Method of increasing the function of an aav vector
WO2006119432A2 (en) 2005-04-29 2006-11-09 The Government Of The U.S.A., As Rep. By The Sec., Dept. Of Health & Human Services Isolation, cloning and characterization of new adeno-associated virus (aav) serotypes
EP1857552A1 (en) 2006-05-20 2007-11-21 Cargill Incorporated Thermostable xylose isomerase enzyme
EP3272872B1 (en) 2005-10-20 2020-06-17 uniQure IP B.V. Improved aav vectors produced in insect cells
EP2305109A1 (en) 2005-11-29 2011-04-06 Surgi-Vision, Inc. MRI-guided localization and/or lead placement systems, related methods, devices and computer program products
EP2007795B1 (en) 2006-03-30 2016-11-16 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid proteins
KR101187628B1 (ko) 2007-05-17 2012-10-05 메드제네시스 테라퓨틱스 인코포레이티드 제거가능한 보강 부재를 가진 전달-강화 전달 카테터 및 이를 사용하는 방법
ES2602610T3 (es) 2007-05-31 2017-02-21 Medigene Ag Proteína estructural mutada de un parvovirus
US8175677B2 (en) 2007-06-07 2012-05-08 MRI Interventions, Inc. MRI-guided medical interventional systems and methods
EP2019143A1 (en) 2007-07-23 2009-01-28 Genethon CNS gene delivery using peripheral administration of AAV vectors
CA2700523A1 (en) 2007-09-24 2009-04-02 Surgivision, Inc. Mri-guided medical interventional systems and methods
US8548569B2 (en) 2007-09-24 2013-10-01 MRI Interventions, Inc. Head fixation assemblies for medical procedures
US8099150B2 (en) 2007-09-24 2012-01-17 MRI Interventions, Inc. MRI-compatible head fixation frame with cooperating head coil apparatus
EP2058401A1 (en) 2007-10-05 2009-05-13 Genethon Widespread gene delivery to motor neurons using peripheral injection of AAV vectors
US8340743B2 (en) 2007-11-21 2012-12-25 MRI Interventions, Inc. Methods, systems and computer program products for positioning a guidance apparatus relative to a patient
EP2910637A1 (en) 2008-05-20 2015-08-26 University of Florida Research Foundation, Inc. Vectors for delivery of light-sensitive proteins and methods of use
WO2010093784A2 (en) 2009-02-11 2010-08-19 The University Of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
CN102625670B (zh) 2009-06-16 2015-07-15 核磁共振成像介入技术有限公司 Mri导向装置以及能够近实时地跟踪和生成该装置的动态可视化的mri导向的介入系统
JP2012531893A (ja) 2009-06-29 2012-12-13 ボード・オブ・リージエンツ，ザ・ユニバーシテイ・オブ・テキサス・システム アルギナーゼ製剤および方法
US9163261B2 (en) 2010-02-22 2015-10-20 Koteswara Rao KOLLIPARA Adeno-associated virus 2/8—micro RNA-101 therapy for liver cancer
EP2550021A2 (en) 2010-03-22 2013-01-30 Association Institut de Myologie Methods of increasing efficiency of vector penetration of target tissue
EP2558154B1 (en) 2010-04-16 2020-06-17 ClearPoint Neuro, Inc. Mri surgical systems including mri-compatible surgical cannulae for transferring a substance to and/or from a patient
EP2826860B1 (en) 2010-04-23 2018-08-22 University of Massachusetts CNS targeting AAV vectors and methods of use thereof
EP2394667A1 (en) 2010-06-10 2011-12-14 Laboratorios Del Dr. Esteve, S.A. Vectors and sequences for the treatment of diseases
WO2012057363A1 (ja) 2010-10-27 2012-05-03 学校法人自治医科大学 神経系細胞への遺伝子導入のためのアデノ随伴ウイルスビリオン
JP6091435B2 (ja) 2011-02-22 2017-03-08 カリフォルニア インスティチュート オブ テクノロジー アデノ随伴ウイルス（ａａｖ）ベクターを用いたタンパク質の送達
US20140024909A1 (en) 2011-02-24 2014-01-23 MRI Interventions, Inc. Mri-guided catheters
US20120295960A1 (en) * 2011-05-20 2012-11-22 Oxford Biomedica (Uk) Ltd. Treatment regimen for parkinson's disease
US20130035660A1 (en) 2011-08-01 2013-02-07 Alcyone Lifesciences, Inc. Multidirectional microfluidic drug delivery devices with conformable balloons
EP3147295B2 (en) 2011-08-24 2023-11-22 The Board of Trustees of the Leland Stanford Junior University New avv capsid proteins for nucleic acid transfer
US20140359799A1 (en) 2011-12-23 2014-12-04 Case Western Reserve University Targeted gene modification using hybrid recombinant adeno-associated virus
US9192393B2 (en) 2012-05-31 2015-11-24 MRI Interventions, Inc. MRI compatible surgical drills and related methods
US10206693B2 (en) 2012-07-19 2019-02-19 MRI Interventions, Inc. MRI-guided medical interventional systems and methods
US9498290B2 (en) 2012-07-19 2016-11-22 MRI Interventions, Inc. Surgical navigation devices and methods
US9031636B2 (en) 2012-07-19 2015-05-12 MRI Interventions, Inc. MRI-compatible head fixation apparatus
US9192446B2 (en) 2012-09-05 2015-11-24 MRI Interventions, Inc. Trajectory guide frame for MRI-guided surgeries
PL2900686T3 (pl) 2012-09-28 2021-01-25 The University Of North Carolina At Chapel Hill Wektory aav ukierunkowane na oligodendrocyty
US9567376B2 (en) 2013-02-08 2017-02-14 The Trustees Of The University Of Pennsylvania Enhanced AAV-mediated gene transfer for retinal therapies
CN105143443A (zh) 2013-02-22 2015-12-09 建新公司 微载体灌注培养方法及其用途
US9572928B2 (en) 2013-03-13 2017-02-21 MRI Interventions, Inc. Substance delivery devices, systems and methods
US9498575B2 (en) 2013-03-14 2016-11-22 MRI Interventions, Inc. Substance delivery devices, systems and methods
US10077291B2 (en) 2013-03-15 2018-09-18 The University Of North Carolina At Chapel Hill Methods and compositions for dual glycan binding AAV vectors
US9618590B2 (en) 2013-03-15 2017-04-11 MRI Interventions, Inc. Tip assembly for MRI-compatible medical devices and MRI-compatible medical devices incorporating same
TWI500551B (zh) 2013-04-26 2015-09-21 Ful Chee Ent Co Ltd Bicycle quick release structure
BR112015027336A2 (pt) 2013-05-01 2017-09-26 Genzyme Corp composições e processos para tratamento de atrofia muscular espinhal
EP3003391B1 (en) 2013-05-31 2021-09-22 The Regents of The University of California Adeno-associated virus variants and methods of use thereof
WO2015038958A1 (en) 2013-09-13 2015-03-19 California Institute Of Technology Selective recovery
AU2014324717B2 (en) 2013-09-26 2020-10-08 University Of Florida Research Foundation, Inc. Synthetic combinatorial AAV capsid library for targeted gene therapy
WO2015057807A1 (en) 2013-10-16 2015-04-23 MRI Interventions, Inc. Surgical navigation systems, related devices and methods
US10087224B2 (en) 2013-11-01 2018-10-02 Cornell University Gene therapy for Alzheimer's and other neurodegenerative diseases and conditions
US10595744B2 (en) 2014-02-14 2020-03-24 MRI Interventions, Inc. Surgical tool-positioning devices and related methods
GB201403684D0 (en) 2014-03-03 2014-04-16 King S College London Vector
US10982228B2 (en) 2014-05-02 2021-04-20 Genzyme Corporation AAV vectors for retinal and CNS gene therapy
WO2015191508A1 (en) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
KR20230067694A (ko) 2014-09-24 2023-05-16 시티 오브 호프 고효율 게놈 편집을 위한 아데노-관련 바이러스 벡터 변이체 및 이의 방법
CN107073051B (zh) 2014-10-21 2021-08-24 马萨诸塞大学 重组aav变体及其用途
EP3215191A4 (en) * 2014-11-05 2018-08-01 Voyager Therapeutics, Inc. Aadc polynucleotides for the treatment of parkinson's disease
EP3256588A2 (en) 2015-02-10 2017-12-20 Genzyme Corporation VARIANT RNAi
US20180135074A1 (en) * 2015-02-19 2018-05-17 University Of Florida Research Foundation, Incorporated Recombinant aav vectors for gene therapy of human hematopoietic disorders
US10472650B2 (en) 2015-02-20 2019-11-12 University Of Iowa Research Foundation Methods and compositions for treating genetic eye diseases
WO2017015102A1 (en) 2015-07-17 2017-01-26 The Trustees Of The University Of Pennsylvania Compositions and methods for achieving high levels of transduction in human liver cells
WO2017023724A1 (en) * 2015-07-31 2017-02-09 Voyager Therapeutics, Inc. Compositions and methods for the treatment of aadc deficiency
IL292830B2 (en) 2015-09-28 2023-06-01 Univ Florida Methods and compositions for stealth virus antibody vectors
WO2017066764A2 (en) 2015-10-16 2017-04-20 William Marsh Rice University Modification of n-terminal region of capsid proteins for enhanced properties of adeno-associated viruses
WO2017083722A1 (en) 2015-11-11 2017-05-18 Greenberg Kenneth P Crispr compositions and methods of using the same for gene therapy
US10240145B2 (en) 2015-11-25 2019-03-26 The Board Of Trustees Of The Leland Stanford Junior University CRISPR/Cas-mediated genome editing to treat EGFR-mutant lung cancer
JP7066619B2 (ja) 2015-12-11 2022-05-13 カリフォルニア インスティチュート オブ テクノロジー アデノ随伴ウイルス（ａａｖ）を誘導するための標的指向性ペプチド
JOP20190269A1 (ar) * 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
EP3784780A1 (en) * 2018-04-27 2021-03-03 Voyager Therapeutics, Inc. Methods for measuring the potency of aadc viral vectors
TW202142692A (zh) * 2020-01-29 2021-11-16 美商航海家醫療公司 用於生產aav顆粒之方法及系統

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