JP2009526066A - 遅速性脳室内送達 - Google Patents

遅速性脳室内送達 Download PDF

Info

Publication number
JP2009526066A
JP2009526066A JP2008554340A JP2008554340A JP2009526066A JP 2009526066 A JP2009526066 A JP 2009526066A JP 2008554340 A JP2008554340 A JP 2008554340A JP 2008554340 A JP2008554340 A JP 2008554340A JP 2009526066 A JP2009526066 A JP 2009526066A
Authority
JP
Japan
Prior art keywords
drug
patient
brain
rate
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.)
Pending
Application number
JP2008554340A
Other languages
English (en)
Japanese (ja)
Other versions
JP2009526066A5 (https=
Inventor
ジェイムズ・ドッジ
マルコ・パッシーニ
ラムヤ・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
Application filed by Genzyme Corp filed Critical Genzyme Corp
Publication of JP2009526066A publication Critical patent/JP2009526066A/ja
Publication of JP2009526066A5 publication Critical patent/JP2009526066A5/ja
Pending legal-status Critical Current

Links

Images

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • 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
    • A61K9/0024—Solid, semi-solid or solidifying implants, which are implanted or injected in body tissue
    • 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
    • A61K9/0085—Brain, e.g. brain implants; Spinal cord
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P23/00—Anaesthetics
    • 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
    • A61P25/08—Antiepileptics; Anticonvulsants
    • 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
    • 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
    • A61P25/28—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
    • A61P3/00—Drugs for disorders of the metabolism
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • 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
    • 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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biomedical Technology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Diabetes (AREA)
  • Genetics & Genomics (AREA)
  • Epidemiology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Psychology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Dermatology (AREA)
  • Molecular Biology (AREA)
  • Emergency Medicine (AREA)
  • Endocrinology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Anesthesiology (AREA)
JP2008554340A 2006-02-09 2007-02-08 遅速性脳室内送達 Pending JP2009526066A (ja)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US77145106P 2006-02-09 2006-02-09
PCT/US2007/003382 WO2007095056A2 (en) 2006-02-09 2007-02-08 Slow intraventricular delivery

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2013171131A Division JP6129685B2 (ja) 2006-02-09 2013-08-21 遅速性脳室内送達

Publications (2)

Publication Number Publication Date
JP2009526066A true JP2009526066A (ja) 2009-07-16
JP2009526066A5 JP2009526066A5 (https=) 2010-04-08

Family

ID=38372004

Family Applications (4)

Application Number Title Priority Date Filing Date
JP2008554340A Pending JP2009526066A (ja) 2006-02-09 2007-02-08 遅速性脳室内送達
JP2013171131A Active JP6129685B2 (ja) 2006-02-09 2013-08-21 遅速性脳室内送達
JP2015188744A Active JP6408970B2 (ja) 2006-02-09 2015-09-25 遅速性脳室内送達
JP2017152871A Active JP6617125B2 (ja) 2006-02-09 2017-08-08 遅速性脳室内送達

Family Applications After (3)

Application Number Title Priority Date Filing Date
JP2013171131A Active JP6129685B2 (ja) 2006-02-09 2013-08-21 遅速性脳室内送達
JP2015188744A Active JP6408970B2 (ja) 2006-02-09 2015-09-25 遅速性脳室内送達
JP2017152871A Active JP6617125B2 (ja) 2006-02-09 2017-08-08 遅速性脳室内送達

Country Status (19)

Country Link
US (3) US20090123451A1 (https=)
EP (2) EP1988823B1 (https=)
JP (4) JP2009526066A (https=)
CN (1) CN101404927A (https=)
AR (1) AR059372A1 (https=)
BR (1) BRPI0707900A2 (https=)
CA (2) CA3168881C (https=)
CY (1) CY1121822T1 (https=)
DK (1) DK1988823T3 (https=)
ES (2) ES3054134T3 (https=)
HU (1) HUE040490T2 (https=)
IL (2) IL193242A (https=)
LT (1) LT1988823T (https=)
MX (4) MX358492B (https=)
PL (1) PL1988823T3 (https=)
PT (1) PT1988823T (https=)
RU (1) RU2452368C2 (https=)
SI (1) SI1988823T1 (https=)
WO (1) WO2007095056A2 (https=)

Families Citing this family (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AR059089A1 (es) * 2006-01-20 2008-03-12 Genzyme Corp Administracion intraventricular de una enzima para enfermedades de almacenamiento lisosomal
HUE040490T2 (hu) 2006-02-09 2019-03-28 Genzyme Corp Lassú intraventrikuláris szállítás
US20120322861A1 (en) 2007-02-23 2012-12-20 Barry John Byrne Compositions and Methods for Treating Diseases
ES2635726T3 (es) 2007-06-06 2017-10-04 Genzyme Corporation Terapia génica para enfermedades de almacenamiento lisosómico
EP2167071B1 (en) * 2007-06-13 2020-03-18 Wayne State University Board Of Governors A baclofen solution for low-volume therapeutic delivery
JP5903045B2 (ja) 2009-07-31 2016-04-13 パックスバックス， インコーポレイテッド アデノウイルスに基づくベクター
SI3998078T1 (sl) 2009-08-28 2026-02-27 Genzyme Corporation Encimsko nadomestno zdravljenje z naraščajočim odmerkom za zdravljenje pomanjkanja kisle sfingomielinaze
PT3103469T (pt) 2010-06-25 2021-03-04 Shire Human Genetic Therapies Administração de agentes terapêuticos no snc
KR20150020250A (ko) 2012-06-19 2015-02-25 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 당뇨병을 치료하기 위한 조성물 및 방법
CN115120746A (zh) 2013-05-15 2022-09-30 明尼苏达大学董事会 腺相关病毒介导的基因向中枢神经系统转移
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
KR102883778B1 (ko) 2017-09-22 2025-11-12 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Ii형 점액다당류증의 치료를 위한 유전자 요법
US12018087B2 (en) 2018-08-02 2024-06-25 Dyne Therapeutics, Inc. Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject
SG11202100928QA (en) 2018-08-02 2021-02-25 Dyne Therapeutics Inc Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2005002515A2 (en) * 2003-06-20 2005-01-13 Biomarin Pharmaceutical Inc. Delivery of therapeutic compounds to the brain and other tissues
WO2005021064A2 (en) * 2003-08-29 2005-03-10 Biomarin Pharmaceutical Inc. Delivery of therapeutic compounds to the brain and other tissues
JP2009525963A (ja) * 2006-01-20 2009-07-16 ジェンザイム・コーポレーション リソソーム蓄積症のための脳室内酵素の輸送

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
US5670488A (en) 1992-12-03 1997-09-23 Genzyme Corporation Adenovirus vector for gene therapy
US5919676A (en) 1993-06-24 1999-07-06 Advec, Inc. Adenoviral vector system comprising Cre-loxP recombination
WO1995005864A1 (en) * 1993-08-27 1995-03-02 Government Of The United States Of America, Represented By The Secretary Of The Department Of Health And Human Services Convection-enhanced drug delivery
CA2189067A1 (en) 1994-04-28 1995-11-09 Gary J. Nabel Gene delivery vector using plasmid dna packaged into an adenovirus and a packaging cell line
AU5551996A (en) 1995-04-17 1996-11-07 Board Of Regents, The University Of Texas System An adenovirus helper-virus system
IL160406A0 (en) 1995-06-15 2004-07-25 Crucell Holland Bv A cell harbouring nucleic acid encoding adenoritus e1a and e1b gene products
US6013516A (en) 1995-10-06 2000-01-11 The Salk Institute For Biological Studies Vector and method of use for nucleic acid delivery to non-dividing cells
WO1997025446A1 (en) 1996-01-05 1997-07-17 Genetic Therapy, Inc. Recombinase-mediated generation of adenoviral vectors
US5735814A (en) * 1996-04-30 1998-04-07 Medtronic, Inc. Techniques of treating neurodegenerative disorders by brain infusion
MX340738B (es) 1996-09-13 2016-07-22 Shire Human Genetic Therapies Terapia para deficiencia de a-galactosidasa a.
US7033598B2 (en) * 1996-11-19 2006-04-25 Intrabrain International N.V. Methods and apparatus for enhanced and controlled delivery of a biologically active agent into the central nervous system of a mammal
US6042579A (en) * 1997-04-30 2000-03-28 Medtronic, Inc. Techniques for treating neurodegenerative disorders by infusion of nerve growth factors into the brain
US5994136A (en) 1997-12-12 1999-11-30 Cell Genesys, Inc. Method and means for producing high titer, safe, recombinant lentivirus vectors
WO1999057296A1 (en) 1998-05-01 1999-11-11 Genzyme Corporation Partially deleted adenoviral vectors
US6702945B2 (en) 2000-12-28 2004-03-09 Exxonmobil Research And Engineering Company Ionic membranes for organic sulfur separation from liquid hydrocarbon solutions
US6689756B2 (en) * 2001-03-02 2004-02-10 Integra Lifesciences Corporation Treatment of neurological disease
US20030163181A1 (en) * 2001-12-07 2003-08-28 Neuron Therapeutics, Inc. Protection of neurological tissue by direct CNS perfusion cooling
EP1575562B1 (en) * 2002-11-26 2016-10-05 Seacoast Neurosciene, Inc. Buoyant polymer particles delivering therapeutic agents
RU2253486C2 (ru) * 2003-03-03 2005-06-10 Ростовский научно-исследовательский онкологический институт МЗ РФ Способ химиотерапии опухолей центральной нервной системы
US20050102708A1 (en) * 2003-03-12 2005-05-12 Laurent Lecanu Animal model simulating neurologic disease
US20040258666A1 (en) 2003-05-01 2004-12-23 Passini Marco A. Gene therapy for neurometabolic disorders
US20050026823A1 (en) * 2003-06-20 2005-02-03 Biomarin Pharmaceutical Inc. Use of the chaperone receptor-associated protein (RAP) for the delivery of therapeutic compounds to the brain and other tissues
EP1730183A2 (en) 2004-01-27 2006-12-13 Compugen USA, Inc. Novel nucleotide and amino acid sequences, and assays and methods of use thereof for diagnosis of endometriosis
US20050208090A1 (en) * 2004-03-18 2005-09-22 Medtronic, Inc. Methods and systems for treatment of neurological diseases of the central nervous system
WO2005095955A1 (en) * 2004-03-31 2005-10-13 Children, Youth And Women's Health Service Screening for lysosomal storage disease status
AR059088A1 (es) * 2006-01-20 2008-03-12 Genzyme Corp Administracion intraventricular de una proteina para esclerosis lateral amiotrofica
HUE040490T2 (hu) * 2006-02-09 2019-03-28 Genzyme Corp Lassú intraventrikuláris szállítás
ES2635726T3 (es) 2007-06-06 2017-10-04 Genzyme Corporation Terapia génica para enfermedades de almacenamiento lisosómico

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2005002515A2 (en) * 2003-06-20 2005-01-13 Biomarin Pharmaceutical Inc. Delivery of therapeutic compounds to the brain and other tissues
WO2005021064A2 (en) * 2003-08-29 2005-03-10 Biomarin Pharmaceutical Inc. Delivery of therapeutic compounds to the brain and other tissues
JP2009525963A (ja) * 2006-01-20 2009-07-16 ジェンザイム・コーポレーション リソソーム蓄積症のための脳室内酵素の輸送

Also Published As

Publication number Publication date
JP2016028711A (ja) 2016-03-03
US20190328682A1 (en) 2019-10-31
CA2641359A1 (en) 2007-08-23
DK1988823T3 (en) 2018-12-03
JP2014012694A (ja) 2014-01-23
PT1988823T (pt) 2018-11-22
US11253485B2 (en) 2022-02-22
LT1988823T (lt) 2018-12-10
CA2641359C (en) 2022-10-04
EP1988823A4 (en) 2009-02-11
MX392030B (es) 2025-03-21
CA3168881C (en) 2025-09-09
JP6408970B2 (ja) 2018-10-17
MX361816B (es) 2018-12-17
WO2007095056A2 (en) 2007-08-23
MX358492B (es) 2018-08-22
EP3459441A1 (en) 2019-03-27
IL248492A0 (en) 2016-12-29
CN101404927A (zh) 2009-04-08
ES3054134T3 (en) 2026-01-30
US20090123451A1 (en) 2009-05-14
ES2697502T3 (es) 2019-01-24
CY1121822T1 (el) 2020-07-31
RU2008136196A (ru) 2010-03-20
PL1988823T3 (pl) 2019-01-31
HUE040490T2 (hu) 2019-03-28
WO2007095056A3 (en) 2008-01-10
JP6129685B2 (ja) 2017-05-17
JP6617125B2 (ja) 2019-12-11
EP1988823A2 (en) 2008-11-12
EP1988823B1 (en) 2018-08-15
EP3459441B1 (en) 2025-09-03
BRPI0707900A2 (pt) 2011-05-10
IL193242A (en) 2017-11-30
AR059372A1 (es) 2008-03-26
SI1988823T1 (sl) 2018-12-31
JP2018024664A (ja) 2018-02-15
US20220370348A1 (en) 2022-11-24
RU2452368C2 (ru) 2012-06-10
CA3168881A1 (en) 2007-08-23
IL193242A0 (en) 2009-08-03
MX2022005303A (es) 2022-05-16

Similar Documents

Publication Publication Date Title
JP6617125B2 (ja) 遅速性脳室内送達
JP6312771B2 (ja) 神経代謝性疾患についての遺伝子治療
TWI730952B (zh) 病毒顆粒至紋狀體和皮質的增強遞送
JP6417322B2 (ja) 遺伝子治療ベクターの広範な遺伝子送達
US20100216709A1 (en) Systemic insulin-like growth factor-1 therapy reduces diabetic peripheral neuropathy and improves renal function in diabetic nephropathy
CN101068575A (zh) 向肝脏定向递送遗传物质的方法
JP2023002721A (ja) 神経変性疾患を処置するための遺伝子移入用組成物、遺伝子導入法、及び遺伝子導入の使用
AU2021396150A1 (en) Treatment of danon disease
Zhao et al. In vivo applications and toxicities of AAV-based gene therapies in rare diseases
JP2024505739A (ja) 組換えヒトイズロン酸－２－スルファターゼ（ｉｄｓ）によるムコ多糖症ｉｉの治療
WO2025075963A1 (en) Methods and formulations for treating mucopolysaccharidosis ii-associated hearing loss with recombinant human iduronate-2-sulfatase
Lancaster 982. Delivery of Encapsulated VSMC Expressing GLP-1 To Treat Diabetic T2DM Rats

Legal Events

Date Code Title Description
A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20100208

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20100216

RD04 Notification of resignation of power of attorney

Free format text: JAPANESE INTERMEDIATE CODE: A7424

Effective date: 20120309

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20120904

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20121203

A602 Written permission of extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A602

Effective date: 20121210

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20121227

A602 Written permission of extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A602

Effective date: 20130109

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20130201

A602 Written permission of extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A602

Effective date: 20130208

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20130423

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20130821