FR20C1058I1 - GENE DELIVERY TO THE CENTRAL NERVOUS SYSTEM BY PERIPHERAL DELIVERY OF AAV VECTOR - Google Patents
GENE DELIVERY TO THE CENTRAL NERVOUS SYSTEM BY PERIPHERAL DELIVERY OF AAV VECTORInfo
- Publication number
- FR20C1058I1 FR20C1058I1 FR20C1058C FR20C1058C FR20C1058I1 FR 20C1058 I1 FR20C1058 I1 FR 20C1058I1 FR 20C1058 C FR20C1058 C FR 20C1058C FR 20C1058 C FR20C1058 C FR 20C1058C FR 20C1058 I1 FR20C1058 I1 FR 20C1058I1
- Authority
- FR
- France
- Prior art keywords
- delivery
- nervous system
- central nervous
- aav vector
- peripheral
- 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.)
- Active
Links
- 210000003169 central nervous system Anatomy 0.000 title 1
- 238000001476 gene delivery Methods 0.000 title 1
- 230000002093 peripheral effect Effects 0.000 title 1
Classifications
-
- 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
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/0075—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 delivery route, e.g. oral, subcutaneous
-
- 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
-
- 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)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Biotechnology (AREA)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Molecular Biology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Epidemiology (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Biochemistry (AREA)
- Virology (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
EP07301263A EP2019143A1 (en) | 2007-07-23 | 2007-07-23 | CNS gene delivery using peripheral administration of AAV vectors |
EP08786331.2A EP2185712B1 (en) | 2007-07-23 | 2008-07-22 | Cns gene delivery using peripheral administration of aav vectors |
PCT/EP2008/059595 WO2009013290A1 (en) | 2007-07-23 | 2008-07-22 | Cns gene delivery using peripheral administration of aav vectors |
Publications (1)
Publication Number | Publication Date |
---|---|
FR20C1058I1 true FR20C1058I1 (en) | 2020-12-25 |
Family
ID=38573418
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
FR20C1058C Active FR20C1058I1 (en) | 2007-07-23 | 2020-11-17 | GENE DELIVERY TO THE CENTRAL NERVOUS SYSTEM BY PERIPHERAL DELIVERY OF AAV VECTOR |
Country Status (7)
Country | Link |
---|---|
US (3) | US10208318B2 (en) |
EP (4) | EP2019143A1 (en) |
CA (2) | CA2999549C (en) |
ES (1) | ES2586434T3 (en) |
FR (1) | FR20C1058I1 (en) |
PT (1) | PT2185712T (en) |
WO (1) | WO2009013290A1 (en) |
Families Citing this family (60)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
EP2019143A1 (en) | 2007-07-23 | 2009-01-28 | Genethon | CNS gene delivery using peripheral administration of AAV vectors |
EP2058401A1 (en) * | 2007-10-05 | 2009-05-13 | Genethon | Widespread gene delivery to motor neurons using peripheral injection of AAV vectors |
EP2271763A2 (en) * | 2008-04-03 | 2011-01-12 | Yeda Research And Development Company Ltd. | Methods of expressing a polypeptide in the brain and nucleic acid constructs capable of same |
US9415121B2 (en) | 2008-12-19 | 2016-08-16 | Nationwide Children's Hospital | Delivery of MECP2 polynucleotide using recombinant AAV9 |
US11219696B2 (en) | 2008-12-19 | 2022-01-11 | Nationwide Children's Hospital | Delivery of polynucleotides using recombinant AAV9 |
JP5879256B2 (en) * | 2009-05-02 | 2016-03-08 | ジェンザイム・コーポレーション | Gene therapy for neurodegenerative disorders |
US8734809B2 (en) | 2009-05-28 | 2014-05-27 | University Of Massachusetts | AAV's and uses thereof |
CA3066596A1 (en) | 2010-04-23 | 2011-10-27 | University Of Massachusetts | Cns targeting aav vectors and methods of use thereof |
US20130039888A1 (en) | 2011-06-08 | 2013-02-14 | Nationwide Children's Hospital Inc. | Products and methods for delivery of polynucleotides by adeno-associated virus for lysosomal storage disorders |
WO2013184209A1 (en) | 2012-06-04 | 2013-12-12 | Ludwig Institute For Cancer Research Ltd. | Mif for use in methods of treating subjects with a neurodegenerative disorder |
US20150182637A1 (en) * | 2012-06-21 | 2015-07-02 | Association Institut De Myologie | Widespread gene delivery of gene therapy vectors |
PT2879719T (en) | 2012-08-01 | 2018-10-08 | Ohio State Innovation Foundation | Intrathecal delivery of recombinant adeno-associated virus 9 |
JP2015529685A (en) | 2012-09-17 | 2015-10-08 | ザ・リサーチ・インスティテュート・アット・ネイションワイド・チルドレンズ・ホスピタルThe Research Institute Atnationwide Children’S Hospital | Compositions and methods for the treatment of amyotrophic lateral sclerosis |
CN111621507A (en) * | 2012-09-28 | 2020-09-04 | 北卡罗来纳-查佩尔山大学 | AAV vectors targeting oligodendrocytes |
EP2749569A1 (en) | 2012-12-28 | 2014-07-02 | Annibale Alessandro Puca | Variant of BPIFB4 protein |
WO2014178863A1 (en) * | 2013-05-01 | 2014-11-06 | Genzyme Corporation | Compositions and methods for treating spinal muscular atrophy |
PT3702466T (en) | 2013-08-27 | 2023-01-24 | Res Inst Nationwide Childrens Hospital | Products and methods for treatment of amyotrophic lateral sclerosis |
AU2014346987A1 (en) | 2013-11-05 | 2016-06-23 | The Research Institute At Nationwide Children's Hospital | Compositions and methods for inhibiting NF-kB and SOD-1 to treat amyotrophic lateral sclerosis |
US20160333071A1 (en) * | 2014-01-14 | 2016-11-17 | The Trustees Of Columbia University In The City Of New York | Biological Pacemakers Incorporating HCN2 and SkM1 Genes |
WO2015158365A1 (en) * | 2014-04-14 | 2015-10-22 | Association Institut De Myologie | Treatment of myotonic dystrophy |
RU2738421C2 (en) | 2014-10-21 | 2020-12-14 | Юниверсити Оф Массачусетс | Versions of recombinant aav and use thereof |
MX2017006216A (en) | 2014-11-14 | 2018-08-29 | Voyager Therapeutics Inc | Compositions and methods of treating amyotrophic lateral sclerosis (als). |
RU2727015C2 (en) | 2014-11-21 | 2020-07-17 | Дзе Юниверсити Оф Норт Каролина Эт Чепел Хилл | Aav vectors aimed at the central nervous system |
EP3233131A1 (en) | 2014-12-16 | 2017-10-25 | Board of Regents of the University of Nebraska | Gene therapy for juvenile batten disease |
EP3368054A4 (en) | 2015-10-28 | 2019-07-03 | Voyager Therapeutics, Inc. | Regulatable expression using adeno-associated virus (aav) |
WO2017085317A1 (en) * | 2015-11-19 | 2017-05-26 | Universitat Autonoma De Barcelona | Secreted splicing variant of mammal klotho as a medicament for cognition and behaviour impairments |
LT3411484T (en) * | 2016-02-05 | 2023-11-27 | Emory University | Injection of single-stranded or self-complementary adeno-associated virus 9 into the cerebrospinal fluid |
IL302748A (en) | 2016-05-18 | 2023-07-01 | Voyager Therapeutics Inc | Modulatory polynucleotides |
CN109831916B (en) | 2016-05-18 | 2023-07-21 | 沃雅戈治疗公司 | Compositions and methods for treating huntington's disease |
US11504435B2 (en) | 2016-06-13 | 2022-11-22 | The University Of North Carolina At Chapel Hill | Optimized CLN1 genes and expression cassettes and their use |
KR20190075964A (en) | 2016-10-13 | 2019-07-01 | 유니버시티 오브 매사추세츠 | AAV capsid design |
CA3058189A1 (en) | 2017-04-03 | 2018-10-11 | Encoded Therapeutics, Inc. | Tissue selective transgene expression |
CN110913866A (en) | 2017-05-05 | 2020-03-24 | 沃雅戈治疗公司 | Compositions and methods for treating Amyotrophic Lateral Sclerosis (ALS) |
US11752181B2 (en) | 2017-05-05 | 2023-09-12 | Voyager Therapeutics, Inc. | Compositions and methods of treating Huntington's disease |
WO2019018342A1 (en) | 2017-07-17 | 2019-01-24 | Voyager Therapeutics, Inc. | Trajectory array guide system |
EP3662060A2 (en) | 2017-08-03 | 2020-06-10 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of aav |
US20200237799A1 (en) | 2017-10-16 | 2020-07-30 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (als) |
JP7502991B2 (en) | 2017-10-16 | 2024-06-19 | ボイジャー セラピューティクス インコーポレイテッド | Treatment of amyotrophic lateral sclerosis (ALS) |
CA3099306A1 (en) | 2018-05-15 | 2019-11-21 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of parkinson's disease |
EP3793616A1 (en) | 2018-05-15 | 2021-03-24 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of aav |
WO2019222444A2 (en) | 2018-05-16 | 2019-11-21 | Voyager Therapeutics, Inc. | Directed evolution |
JP2021529513A (en) | 2018-07-02 | 2021-11-04 | 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. | Treatment of amyotrophic lateral sclerosis and spinal cord-related disorders |
WO2020010035A1 (en) | 2018-07-02 | 2020-01-09 | Voyager Therapeutics, Inc. | Cannula system |
US20210355454A1 (en) | 2018-07-24 | 2021-11-18 | Voyager Therapeutics, Inc. | Systems and methods for producing gene therapy formulations |
TW202035689A (en) | 2018-10-04 | 2020-10-01 | 美商航海家醫療公司 | Methods for measuring the titer and potency of viral vector particles |
WO2020077165A1 (en) | 2018-10-12 | 2020-04-16 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of aav |
AU2020208467A1 (en) | 2019-01-18 | 2021-08-05 | Voyager Therapeutics, Inc. | Methods and systems for producing AAV particles |
WO2021046155A1 (en) | 2019-09-03 | 2021-03-11 | Voyager Therapeutics, Inc. | Vectorized editing of nucleic acids to correct overt mutations |
EP4135841A1 (en) | 2020-04-15 | 2023-02-22 | Voyager Therapeutics, Inc. | Tau binding compounds |
WO2021230987A1 (en) | 2020-05-13 | 2021-11-18 | Voyager Therapeutics, Inc. | Redirection of tropism of aav capsids |
US20240141378A1 (en) | 2021-03-03 | 2024-05-02 | Voyager Therapeutics, Inc. | Controlled expression of viral proteins |
WO2023044483A2 (en) | 2021-09-20 | 2023-03-23 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of her2 positive cancer |
TW202334436A (en) | 2021-11-02 | 2023-09-01 | 美商航海家醫療公司 | Aav capsid variants and uses thereof |
WO2023091948A1 (en) | 2021-11-17 | 2023-05-25 | Voyager Therapeutics, Inc. | Aav capsid variants and uses thereof |
TW202346599A (en) | 2022-02-08 | 2023-12-01 | 美商航海家醫療公司 | Aav capsid variants and uses thereof |
WO2023235791A1 (en) | 2022-06-02 | 2023-12-07 | Voyager Therapeutics, Inc. | Aav capsid variants and uses thereof |
WO2024006741A1 (en) | 2022-06-28 | 2024-01-04 | Voyager Therapeutics, Inc. | Aav capsid variants and uses thereof |
WO2024011112A1 (en) | 2022-07-06 | 2024-01-11 | Voyager Therapeutics, Inc. | Aav capsid variants and uses thereof |
WO2024030976A2 (en) | 2022-08-03 | 2024-02-08 | Voyager Therapeutics, Inc. | Compositions and methods for crossing the blood brain barrier |
WO2024059739A1 (en) | 2022-09-15 | 2024-03-21 | Voyager Therapeutics, Inc. | Tau binding compounds |
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IL122349A0 (en) | 1995-06-07 | 1998-04-05 | Univ Yale | Adeno-associated viral vectors |
FR2756297B1 (en) | 1996-11-22 | 1999-01-08 | Centre Nat Rech Scient | PROCESS FOR PRODUCING RECOMBINANT VIRUSES |
CA2297490A1 (en) | 1997-07-31 | 1999-02-11 | Chiron Corporation | Method enabling readministration of aav vector via immunosuppression of host |
US6491907B1 (en) | 1998-11-10 | 2002-12-10 | The University Of North Carolina At Chapel Hill | Recombinant parvovirus vectors and method of making |
US6498244B1 (en) | 1999-05-28 | 2002-12-24 | Cell Genesys, Inc. | Adeno-associated virus capsid immunologic determinants |
AU1071701A (en) | 1999-09-29 | 2001-04-30 | Trustees Of The University Of Pennsylvania, The | Methods for rapid peg-modification of viral vectors, compositions for enhanced gene transduction, compositions with enhanced physical stability, and uses therefor |
WO2001036603A2 (en) * | 1999-11-17 | 2001-05-25 | Avigen, Inc. | Recombinant adeno-associated virus virions for the treatment of lysosomal disorders |
WO2001091803A2 (en) | 2000-06-01 | 2001-12-06 | University Of North Carolina At Chapel Hill | Methods and compounds for controlled release of recombinant parvovirus vectors |
CA2410828C (en) | 2000-06-01 | 2012-01-24 | University Of North Carolina At Chapel Hill | Duplexed parvovirus vectors |
EP1402043A1 (en) * | 2001-07-03 | 2004-03-31 | Institut National De La Sante Et De La Recherche Medicale (Inserm) | Methods of administering vectors to synaptically connected neurons |
ES2526341T3 (en) * | 2001-12-17 | 2015-01-09 | The Trustees Of The University Of Pennsylvania | Sequences of serotype 9 of adeno-associated virus (AAV), vectors containing them, and uses thereof |
WO2003053476A1 (en) * | 2001-12-19 | 2003-07-03 | Lijun Wang | Adeno-associated virus-mediated delivery of gdnf to skeletal muscles |
WO2003055983A2 (en) | 2001-12-21 | 2003-07-10 | The Salk Institute For Biological Studies | 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 |
ES2862206T3 (en) | 2003-06-19 | 2021-10-07 | Genzyme Corp | AAV Virions with Decreased Immunoreactivity and Their Uses |
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 |
EP2298926A1 (en) * | 2003-09-30 | 2011-03-23 | The Trustees of The University of Pennsylvania | Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof |
US8734810B2 (en) * | 2003-10-29 | 2014-05-27 | Allergan, Inc. | Botulinum toxin treatments of neurological and neuropsychiatric disorders |
WO2005056807A2 (en) | 2003-12-04 | 2005-06-23 | The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services, National Institutes Of Health | Bovine adeno-associated viral (baav) vector and uses thereof |
WO2005072364A2 (en) | 2004-01-27 | 2005-08-11 | University Of Florida | A modified baculovirus expression system for production of pseudotyped raav vector |
US20060110364A1 (en) | 2004-08-20 | 2006-05-25 | Ludwig Institute For Cancer Research | Vector-mediated delivery of polynucleotides encoding soluble VEGF receptors |
WO2006029196A1 (en) * | 2004-09-08 | 2006-03-16 | The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services, National Institutes Of H Ealth | Transcytosis of adeno-associated viruses |
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 |
EP2132309A4 (en) * | 2007-02-23 | 2011-01-05 | Univ Florida | Compositions and methods for treating glycogen storage diseases |
US10696983B2 (en) * | 2007-05-30 | 2020-06-30 | The Trustees Of The University Of Pennsylvania | Method for transducing cells with primary cilia |
EP2019143A1 (en) | 2007-07-23 | 2009-01-28 | Genethon | CNS gene delivery using peripheral administration of AAV vectors |
RU2010117178A (en) | 2007-10-01 | 2011-11-10 | Алькон Рисерч, Лтд. (Us) | SELF-COMPLETE AAV DELIVERED DELIVERY OF MOLECULES INTERFERING RNA FOR TREATMENT OR PREVENTION OF EYE DISEASES |
EP2058401A1 (en) | 2007-10-05 | 2009-05-13 | Genethon | Widespread gene delivery to motor neurons using peripheral injection of AAV vectors |
-
2007
- 2007-07-23 EP EP07301263A patent/EP2019143A1/en not_active Withdrawn
-
2008
- 2008-07-22 PT PT87863312T patent/PT2185712T/en unknown
- 2008-07-22 EP EP18212543.5A patent/EP3498851A1/en active Pending
- 2008-07-22 CA CA2999549A patent/CA2999549C/en active Active
- 2008-07-22 EP EP08786331.2A patent/EP2185712B1/en active Active
- 2008-07-22 EP EP12172848A patent/EP2514827A1/en not_active Ceased
- 2008-07-22 WO PCT/EP2008/059595 patent/WO2009013290A1/en active Application Filing
- 2008-07-22 ES ES08786331.2T patent/ES2586434T3/en active Active
- 2008-07-22 US US12/452,789 patent/US10208318B2/en active Active
- 2008-07-22 CA CA2694241A patent/CA2694241C/en active Active
-
2018
- 2018-12-10 US US16/214,214 patent/US20190153474A1/en not_active Abandoned
-
2020
- 2020-11-17 FR FR20C1058C patent/FR20C1058I1/en active Active
-
2023
- 2023-08-01 US US18/363,453 patent/US20230374547A1/en active Pending
Also Published As
Publication number | Publication date |
---|---|
CA2999549C (en) | 2021-10-19 |
EP3498851A1 (en) | 2019-06-19 |
CA2999549A1 (en) | 2009-01-29 |
CA2694241C (en) | 2018-05-15 |
ES2586434T3 (en) | 2016-10-14 |
WO2009013290A1 (en) | 2009-01-29 |
EP2185712A1 (en) | 2010-05-19 |
EP2185712B1 (en) | 2016-05-18 |
EP2514827A1 (en) | 2012-10-24 |
PT2185712T (en) | 2016-08-12 |
CA2694241A1 (en) | 2009-01-29 |
US10208318B2 (en) | 2019-02-19 |
EP2019143A1 (en) | 2009-01-28 |
US20230374547A1 (en) | 2023-11-23 |
US20100130594A1 (en) | 2010-05-27 |
US20190153474A1 (en) | 2019-05-23 |
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