BRPI0611379A2 - terapia genética para distúrbios neurometabólicos - Google Patents

terapia genética para distúrbios neurometabólicos Download PDF

Info

Publication number
BRPI0611379A2
BRPI0611379A2 BRPI0611379-6A BRPI0611379A BRPI0611379A2 BR PI0611379 A2 BRPI0611379 A2 BR PI0611379A2 BR PI0611379 A BRPI0611379 A BR PI0611379A BR PI0611379 A2 BRPI0611379 A2 BR PI0611379A2
Authority
BR
Brazil
Prior art keywords
aav
site
aav2
asm
disease
Prior art date
Application number
BRPI0611379-6A
Other languages
English (en)
Portuguese (pt)
Inventor
Marco A Passini
James Dodge
Gregory R Stewart
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
Application filed by Genzyme Corp filed Critical Genzyme Corp
Publication of BRPI0611379A2 publication Critical patent/BRPI0611379A2/pt

Links

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
    • C12N15/63Introduction 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075Medicinal 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 delivery route, e.g. oral, subcutaneous
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00Drugs for skeletal disorders
    • 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
    • A61P25/28Drugs 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • A61P3/06Antihyperlipidemics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • C12N15/864Parvoviral vectors, e.g. parvovirus, densovirus
    • C12N15/8645Adeno-associated virus
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • C12N2750/14041Use of virus, viral particle or viral elements as a 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
    • C12N2750/14141Use 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
    • C12N2799/00Uses of viruses
    • C12N2799/02Uses of viruses as vector
    • C12N2799/021Uses of viruses as vector for the expression of a heterologous nucleic acid
    • C12N2799/025Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a parvovirus

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Zoology (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Physical Education & Sports Medicine (AREA)
BRPI0611379-6A 2005-05-02 2006-05-02 terapia genética para distúrbios neurometabólicos BRPI0611379A2 (pt)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US67705705P 2005-05-02 2005-05-02
US60/677,057 2005-05-02
US68580805P 2005-05-31 2005-05-31
US60/685,808 2005-05-31
PCT/US2006/017242 WO2006119458A1 (en) 2005-05-02 2006-05-02 Gene therapy for neurometabolic disorders

Publications (1)

Publication Number Publication Date
BRPI0611379A2 true BRPI0611379A2 (pt) 2010-08-31

Family

ID=37308324

Family Applications (1)

Application Number Title Priority Date Filing Date
BRPI0611379-6A BRPI0611379A2 (pt) 2005-05-02 2006-05-02 terapia genética para distúrbios neurometabólicos

Country Status (13)

Country Link
US (2) US10632213B2 (https=)
EP (4) EP1879624B1 (https=)
JP (3) JP5829372B2 (https=)
CN (4) CN107362371A (https=)
AT (1) ATE525092T1 (https=)
AU (1) AU2006243776A1 (https=)
BR (1) BRPI0611379A2 (https=)
CA (2) CA2998603C (https=)
ES (1) ES2887076T3 (https=)
IL (2) IL187078A (https=)
PL (4) PL3058959T3 (https=)
PT (4) PT3520823T (https=)
WO (1) WO2006119458A1 (https=)

Families Citing this family (39)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP3302529B2 (ja) 1995-04-28 2002-07-15 株式会社クボタ ポンプ吸水槽の整流装置
PL1879623T3 (pl) * 2005-05-02 2013-03-29 Genzyme Corp Terapia genowa zaburzeń rdzenia kręgowego
EP1879624B1 (en) 2005-05-02 2011-09-21 Genzyme Corporation Gene therapy for neurometabolic disorders
WO2007146046A2 (en) 2006-06-07 2007-12-21 Genzyme Corporation Gene therapy for amyotrophic lateral sclerosis and other spinal cord disorders
PL3252161T3 (pl) 2007-06-06 2022-03-07 Genzyme Corporation Terapia genowa dla lizosomalnych chorób spichrzeniowych
EP2687609B1 (en) 2008-11-10 2017-01-04 The United States of America, as represented by The Secretary, Department of Health and Human Services Method for treating solid tumor
JP5879256B2 (ja) 2009-05-02 2016-03-08 ジェンザイム・コーポレーション 神経変性障害のための遺伝子治療
US9150926B2 (en) 2010-12-06 2015-10-06 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Diagnosis and treatment of adrenocortical tumors using human microRNA-483
WO2012145646A1 (en) * 2011-04-20 2012-10-26 Miguel Sena-Esteves Methods for the treatment of tay-sachs disease, sandhoff disease, and gmi-gangliosidosis
WO2015191508A1 (en) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
SG11201703148TA (en) 2014-11-05 2017-05-30 Voyager Therapeutics Inc Aadc polynucleotides for the treatment of parkinson's disease
CN119876138A (zh) 2014-11-14 2025-04-25 沃雅戈治疗公司 调节性多核苷酸
CA3193811A1 (en) 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
US11319555B2 (en) 2014-11-20 2022-05-03 Duke University Compositions, systems and methods for cell therapy
HK1245326A1 (zh) 2014-12-12 2018-08-24 Voyager Therapeutics, Inc. 用於生产scaav的组合物和方法
TN2017000353A1 (en) * 2015-02-10 2019-01-16 Genzyme Corp Enhanced delivery of viral particles to the striatum and cortex
WO2016150403A1 (en) * 2015-03-26 2016-09-29 Fujian Tiantai Medical Technology Co. Ltd Method of diagnosis or treatment of neurological disorders with p75ecd and/or p75
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
US11027024B2 (en) 2015-05-29 2021-06-08 University Of Iowa Research Foundation Methods of delivery of transgenes for treating brain diseases
EP3448874A4 (en) 2016-04-29 2020-04-22 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
EP3448987A4 (en) 2016-04-29 2020-05-27 Voyager Therapeutics, Inc. COMPOSITIONS FOR TREATING A DISEASE
CA3024448C (en) 2016-05-18 2025-09-09 Voyager Therapeutics, Inc. MODULATING POLYNUCLEOTIDES
CA3024449A1 (en) 2016-05-18 2017-11-23 Voyager Therapeutics, Inc. Compositions and methods of treating huntington's disease
WO2018044933A1 (en) 2016-08-30 2018-03-08 The Regents Of The University Of California Methods for biomedical targeting and delivery and devices and systems for practicing the same
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
JP2020518259A (ja) 2017-05-05 2020-06-25 ボイジャー セラピューティクス インコーポレイテッドVoyager Therapeutics,Inc. ハンチントン病治療組成物および方法
EP3618839A4 (en) 2017-05-05 2021-06-09 Voyager Therapeutics, Inc. COMPOSITIONS AND METHODS OF TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
FI3635009T3 (fi) 2017-06-07 2026-04-07 Regeneron Pharma Koostumuksia ja menetelmiä entsyymien internalisoimiseksi
JOP20190269A1 (ar) 2017-06-15 2019-11-20 Voyager Therapeutics Inc بولي نوكليوتيدات aadc لعلاج مرض باركنسون
JP7229989B2 (ja) 2017-07-17 2023-02-28 ボイジャー セラピューティクス インコーポレイテッド 軌道アレイガイドシステム
JP7221275B2 (ja) 2017-08-03 2023-02-13 ボイジャー セラピューティクス インコーポレイテッド Aavを送達するための組成物および方法
US20200263199A1 (en) 2017-09-29 2020-08-20 Voyager Therapeutics, Inc. Rescue of central and peripheral neurological phenotype of friedreich's ataxia by intravenous delivery
TW202413649A (zh) 2017-10-16 2024-04-01 美商航海家醫療公司 肌萎縮性脊髓側索硬化症(als)之治療
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS (ALS)
JP7779653B2 (ja) 2018-02-07 2025-12-03 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 治療用タンパク質送達のための方法および組成物
SG11202011296VA (en) 2018-05-15 2020-12-30 Voyager Therapeutics Inc Compositions and methods for the treatment of parkinson's disease
EP3793591A1 (en) 2018-05-17 2021-03-24 Regeneron Pharmaceuticals, Inc. Anti-cd63 antibodies, conjugates, and uses thereof
US12281321B2 (en) 2018-09-28 2025-04-22 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
KR20240126916A (ko) * 2023-02-14 2024-08-22 경북대학교 산학협력단 Asm 단백질 또는 이의 단편을 포함하는 퇴행성 신경질환 또는 우울증 예방 또는 치료용 조성물

Family Cites Families (29)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SE8303626D0 (sv) 1983-06-23 1983-06-23 Kabigen Ab A recombinant plasmid a transformant microorganism, a polydoxyrebonucleotide segment, a process for producing a biologically active protein, and the protein thus produced
US5672344A (en) 1987-12-30 1997-09-30 The Regents Of The University Of Michigan Viral-mediated gene transfer system
US5773278A (en) 1991-05-03 1998-06-30 Mount Sinai Medical Center Acid sphingomyelinase gene
ES2249761T3 (es) 1993-06-24 2006-04-01 Advec Inc. Vectores de adenovirus para terapia genica.
EP1637608B1 (en) 1993-10-25 2009-07-22 CANJI, Inc. Recombinant adenoviral vector and methods of use
PT733103E (pt) 1993-11-09 2004-07-30 Targeted Genetics Corp Criacao de elevados titulos de vectores de aav recombinantes
US7252989B1 (en) 1994-04-04 2007-08-07 Board Of Regents, The University Of Texas System Adenovirus supervector system
CA2187626C (en) 1994-04-13 2009-11-03 Michael G. Kaplitt Aav-mediated delivery of dna to cells of the nervous system
AU1525797A (en) 1996-04-22 1997-11-12 Medtronic, Inc. Two-stage angled venous cannula
US6544785B1 (en) 1998-09-14 2003-04-08 Mount Sinai School Of Medicine Of New York University Helper-free rescue of recombinant negative strand RNA viruses
AU1775901A (en) * 1999-11-17 2001-05-30 Avigen, Inc. Recombinant adeno-associated virus virions for the treatment of lysosomal disorders
US20030165481A1 (en) * 2000-02-24 2003-09-04 Hersh Louis B. Amyloid peptide inactivating enzyme to treat Alzheimer's disease
AU2001290984A1 (en) 2000-09-18 2002-04-02 Genzyme Corporation Expression vectors containing hybrid ubiquitin promoters
US20030050273A1 (en) * 2001-08-29 2003-03-13 Keiya Ozawa Compositions and methods for treating neurodegenerative diseases
US7232670B2 (en) * 2001-09-28 2007-06-19 St. Jude Children's Research Hospital Targeting proteins to cells expressing mannose receptors via expression in insect cells
DK2359869T3 (en) 2001-12-17 2019-04-15 Univ Pennsylvania Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof
NZ533833A (en) 2001-12-21 2008-01-31 Salk Inst For Biological Studi Targeted retrograde gene delivery to motor neurons
US6998118B2 (en) 2001-12-21 2006-02-14 The Salk Institute For Biological Studies Targeted retrograde gene delivery for neuronal protection
AU2003220115A1 (en) * 2002-05-20 2003-12-12 Board Of Regents, The University Of Texas System Methods and compositions for delivering enzymes and nucleic acid molecules to brain, bone, and other tissues
ES2562490T3 (es) * 2003-05-01 2016-03-04 Genzyme Corporation Terapia génica para trastornos neurometabólicos
US20040258666A1 (en) * 2003-05-01 2004-12-23 Passini Marco A. Gene therapy for neurometabolic disorders
US7740168B2 (en) 2003-08-18 2010-06-22 Visa U.S.A. Inc. Method and system for generating a dynamic verification value
AU2003272868A1 (en) 2003-10-15 2005-04-27 Agtc Gene Technology Company Ltd. Method for large-scale production, isolation, purification and the uses of multi-type recombinant adeno-associated virus vectors
EP1716870A1 (en) 2005-04-29 2006-11-02 Bracco Imaging S.p.A. MRI contrast agents endowed with concentration independent responsiveness
EP1879624B1 (en) 2005-05-02 2011-09-21 Genzyme Corporation Gene therapy for neurometabolic disorders
PL1879623T3 (pl) 2005-05-02 2013-03-29 Genzyme Corp Terapia genowa zaburzeń rdzenia kręgowego
JP5706602B2 (ja) 2006-02-08 2015-04-22 ジェンザイム・コーポレーション ニーマンピック病a型に対する遺伝子治療
RU2638802C2 (ru) 2011-05-16 2017-12-15 Джензим Корпорейшн Применение антагонистов cxcr4
TWI821227B (zh) 2017-12-22 2023-11-11 美商健臻公司 胺甲喋呤誘發的免疫耐受性之生物標記

Also Published As

Publication number Publication date
IL187078A0 (en) 2008-02-09
CN101212988A (zh) 2008-07-02
US20140356327A9 (en) 2014-12-04
WO2006119458A1 (en) 2006-11-09
PL2420256T3 (pl) 2016-12-30
JP5829372B2 (ja) 2015-12-09
IL239167A0 (en) 2015-07-30
EP2420256A1 (en) 2012-02-22
CA2998603A1 (en) 2006-11-09
US11957765B2 (en) 2024-04-16
CN107362371A (zh) 2017-11-21
PT3520823T (pt) 2021-09-16
IL187078A (en) 2015-06-30
CN104306986A (zh) 2015-01-28
JP2016041706A (ja) 2016-03-31
EP3058959A1 (en) 2016-08-24
EP3520823B1 (en) 2021-06-30
US10632213B2 (en) 2020-04-28
PL3520823T3 (pl) 2021-11-22
ATE525092T1 (de) 2011-10-15
EP3058959B1 (en) 2019-01-30
PT3058959T (pt) 2019-05-24
PL3058959T3 (pl) 2019-07-31
EP3520823A1 (en) 2019-08-07
AU2006243776A1 (en) 2006-11-09
PT1879624E (pt) 2012-02-02
ES2887076T3 (es) 2021-12-21
JP2008540445A (ja) 2008-11-20
PT2420256T (pt) 2016-09-13
US20200306387A1 (en) 2020-10-01
EP1879624A1 (en) 2008-01-23
JP6338560B2 (ja) 2018-06-06
EP2420256B1 (en) 2016-06-22
CA2607173A1 (en) 2006-11-09
PL1879624T3 (pl) 2012-02-29
CA2607173C (en) 2018-04-24
CA2998603C (en) 2019-07-23
EP1879624A4 (en) 2009-03-25
CN107007842A (zh) 2017-08-04
US20080292593A1 (en) 2008-11-27
JP2014037418A (ja) 2014-02-27
WO2006119458A9 (en) 2007-02-15
EP1879624B1 (en) 2011-09-21

Similar Documents

Publication Publication Date Title
US11957765B2 (en) Gene therapy for neurometabolic disorders
JP6312771B2 (ja) 神経代謝性疾患についての遺伝子治療
WO2005120581A2 (en) Gene therapy for neurometabolic disorders
JP2017132774A (ja) 脊髄疾患の遺伝子療法
BRPI1010868B1 (pt) Composição compreendendo um virion de aav recombinante, e uso do referido vírion

Legal Events

Date Code Title Description
B06F Objections, documents and/or translations needed after an examination request according [chapter 6.6 patent gazette]
B06H Technical and formal requirements: requirement cancelled [chapter 6.8 patent gazette]

Free format text: REF. A RPI NO 2153 DE 10/04/2012. ANULACAO DA EXIGENCIA 6.6 POR TER SIDO INDEVIDA. REQUERENTE NAO APRESENTOU RESPOSTA A EXIGENCIA CONFORME DISPOE A RESOLUCAO 207 DE 24/04/2009, ENTRETANTO, A RESPOSTA CONSTA NO FORMULARIO DE DEPOSITO.

B07D Technical examination (opinion) related to article 229 of industrial property law [chapter 7.4 patent gazette]
B07E Notification of approval relating to section 229 industrial property law [chapter 7.5 patent gazette]
B06U Preliminary requirement: requests with searches performed by other patent offices: procedure suspended [chapter 6.21 patent gazette]
B07A Application suspended after technical examination (opinion) [chapter 7.1 patent gazette]
B09B Patent application refused [chapter 9.2 patent gazette]
B09B Patent application refused [chapter 9.2 patent gazette]

Free format text: MANTIDO O INDEFERIMENTO UMA VEZ QUE NAO FOI APRESENTADO RECURSO DENTRO DO PRAZO LEGAL