FR22C1056I1 - WIDESPREAD GENE DELIVERY TO MOTOR NEURONS USING PERIPHERAL AAV VECTOR INJECTION - Google Patents

WIDESPREAD GENE DELIVERY TO MOTOR NEURONS USING PERIPHERAL AAV VECTOR INJECTION

Info

Publication number
FR22C1056I1
FR22C1056I1 FR22C1056C FR22C1056C FR22C1056I1 FR 22C1056 I1 FR22C1056 I1 FR 22C1056I1 FR 22C1056 C FR22C1056 C FR 22C1056C FR 22C1056 C FR22C1056 C FR 22C1056C FR 22C1056 I1 FR22C1056 I1 FR 22C1056I1
Authority
FR
France
Prior art keywords
peripheral
gene delivery
aav vector
motor neurons
vector injection
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
Application number
FR22C1056C
Other languages
French (fr)
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.)
Centre National de la Recherche Scientifique CNRS
Genethon
Original Assignee
Centre National de la Recherche Scientifique CNRS
Genethon
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=39186802&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=FR22C1056(I1) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Centre National de la Recherche Scientifique CNRS, Genethon filed Critical Centre National de la Recherche Scientifique CNRS
Publication of FR22C1056I1 publication Critical patent/FR22C1056I1/en
Active legal-status Critical Current

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
    • A61K48/005Medicinal 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
    • A61P17/00Drugs for dermatological disorders
    • A61P17/02Drugs for dermatological disorders for treating wounds, ulcers, burns, scars, keloids, or the like
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00Drugs for disorders of the muscular or neuromuscular 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/04Centrally acting analgesics, e.g. opioids
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/20Hypnotics; Sedatives
    • 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
    • A61P29/00Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • A61P37/02Immunomodulators
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • A61P37/02Immunomodulators
    • A61P37/06Immunosuppressants, e.g. drugs for graft rejection
    • 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
    • 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
    • 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
    • A61K48/0083Medicinal 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 administration regime
    • 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
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/008Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Immunology (AREA)
  • Neurology (AREA)
  • Biotechnology (AREA)
  • Neurosurgery (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Pain & Pain Management (AREA)
  • Transplantation (AREA)
  • Hospice & Palliative Care (AREA)
  • Rheumatology (AREA)
  • Anesthesiology (AREA)
  • Dermatology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Epidemiology (AREA)
FR22C1056C 2007-10-05 2022-11-17 WIDESPREAD GENE DELIVERY TO MOTOR NEURONS USING PERIPHERAL AAV VECTOR INJECTION Active FR22C1056I1 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP07301435A EP2058401A1 (en) 2007-10-05 2007-10-05 Widespread gene delivery to motor neurons using peripheral injection of AAV vectors
EP19158152.9A EP3505635B1 (en) 2007-10-05 2008-10-03 Widespread gene delivery to motor neurons using peripheral injection of aav vectors

Publications (1)

Publication Number Publication Date
FR22C1056I1 true FR22C1056I1 (en) 2023-01-06

Family

ID=39186802

Family Applications (2)

Application Number Title Priority Date Filing Date
FR20C1057C Active FR20C1057I2 (en) 2007-10-05 2020-11-17 BROADCAST DELIVERY OF GENES TO MOTOR NEURONS BY PERIPHERAL INJECTION OF AAV VECTORS
FR22C1056C Active FR22C1056I1 (en) 2007-10-05 2022-11-17 WIDESPREAD GENE DELIVERY TO MOTOR NEURONS USING PERIPHERAL AAV VECTOR INJECTION

Family Applications Before (1)

Application Number Title Priority Date Filing Date
FR20C1057C Active FR20C1057I2 (en) 2007-10-05 2020-11-17 BROADCAST DELIVERY OF GENES TO MOTOR NEURONS BY PERIPHERAL INJECTION OF AAV VECTORS

Country Status (18)

Country Link
US (3) US9926574B2 (en)
EP (5) EP2058401A1 (en)
JP (2) JP5620819B2 (en)
CN (2) CN105087650A (en)
CA (2) CA2999607A1 (en)
CY (1) CY1125608T1 (en)
DK (2) DK2212424T3 (en)
ES (3) ES2924892T3 (en)
FI (1) FI2212424T4 (en)
FR (2) FR20C1057I2 (en)
HR (2) HRP20220992T3 (en)
HU (2) HUE059864T2 (en)
LT (2) LT3505635T (en)
NO (1) NO2022048I1 (en)
PL (2) PL3505635T3 (en)
PT (3) PT2212424E (en)
SI (2) SI3505635T1 (en)
WO (1) WO2009043936A1 (en)

Families Citing this family (94)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20120322861A1 (en) 2007-02-23 2012-12-20 Barry John Byrne Compositions and Methods for Treating Diseases
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
CA2711370C (en) 2008-01-15 2017-06-13 The Board Of Trustees Of The Leland Stanford Junior University Markers of acute myeloid leukemia stem cells
US9217155B2 (en) 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
US11219696B2 (en) 2008-12-19 2022-01-11 Nationwide Children's Hospital Delivery of polynucleotides using recombinant AAV9
US9415121B2 (en) 2008-12-19 2016-08-16 Nationwide Children's Hospital Delivery of MECP2 polynucleotide using recombinant AAV9
RU2603740C2 (en) 2009-05-02 2016-11-27 Джензим Корпорейшн Gene therapy for neurodegenerative disorders
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
CA3050894C (en) 2010-04-23 2022-10-18 University Of Massachusetts Multicistronic expression constructs
CA3066596A1 (en) * 2010-04-23 2011-10-27 University Of Massachusetts Cns targeting aav vectors and methods of use thereof
EP3444346B1 (en) 2010-04-23 2022-07-27 University of Massachusetts Aav-based treatment of cholesterol-related disorders
EP2634253B1 (en) 2010-10-27 2016-05-11 Jichi Medical University Adeno-associated virus virions for transferring genes into neural cells
EP3318634A1 (en) 2011-04-21 2018-05-09 University of Massachusetts Raav-based compositions and methods for treating diseases involving dominant-negative or gain of function mutations
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
EP3818991A3 (en) * 2012-06-19 2021-07-14 University of Florida Research Foundation, Inc. Compositions and methods for treating diseases
CN104704123A (en) * 2012-06-21 2015-06-10 肌肉学研究协会 Widespread gene delivery of gene therapy vectors
DK2879719T3 (en) 2012-08-01 2018-09-03 Nationwide Childrens Hospital INTRATEKAL ADMINISTRATION OF RECOMBINANT ADENOASSOCATED VIRUSES
US9539307B2 (en) 2012-09-17 2017-01-10 The Research Institute At Nationwide Children's Hospital Compositions and methods for treating amyotrophic lateral sclerosis
KR20220119187A (en) * 2013-05-15 2022-08-26 리젠츠 오브 더 유니버시티 오브 미네소타 Adeno-associated virus mediated gene transfer to the central nervous system
FI3702466T3 (en) 2013-08-27 2023-03-23 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
EP3116900B1 (en) * 2014-03-09 2020-07-08 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of ornithine transcarbamylase (otc) deficiency
EP2933335A1 (en) * 2014-04-18 2015-10-21 Genethon A method of treating peripheral neuropathies and motor neuron diseases
US10689653B2 (en) 2014-06-03 2020-06-23 University Of Massachusetts Compositions and methods for modulating dysferlin expression
JP6872479B2 (en) 2014-07-31 2021-05-19 アソシアシオン・アンスティテュ・ドゥ・ミオロジーAssociation Institut De Myologie Treatment of amyotrophic lateral sclerosis
US10370432B2 (en) 2014-10-03 2019-08-06 University Of Massachusetts Heterologous targeting peptide grafted AAVS
EP3795580A1 (en) 2014-10-03 2021-03-24 University of Massachusetts High efficiency library-identified aav vectors
CN107073051B (en) 2014-10-21 2021-08-24 马萨诸塞大学 Recombinant AAV variants and uses thereof
US10597660B2 (en) 2014-11-14 2020-03-24 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
JP2017536116A (en) 2014-11-21 2017-12-07 ザ ユニバーシティ オブ ノース カロライナ アット チャペル ヒル AAV vector targeting the central nervous system
CA2967468A1 (en) 2014-12-16 2016-06-23 Board Of Regents Of The University Of Nebraska Gene therapy for juvenile batten disease
US10584321B2 (en) 2015-02-13 2020-03-10 University Of Massachusetts Compositions and methods for transient delivery of nucleases
WO2016164642A1 (en) * 2015-04-08 2016-10-13 The United States Of America, As Represented By The Secretary Of Health And Human Services Viral gene therapy as treatment for cholesterol storage disease or disorder
EP3285780A4 (en) 2015-04-24 2018-12-19 University of Massachusetts Modified aav constructions and uses thereof
CA2999279A1 (en) * 2015-10-01 2017-04-06 Goleini Inc. Targeted expression of chloride channels and methods of use thereof
PL3365438T3 (en) * 2015-10-21 2021-10-18 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Codon-optimized reduced-size atp7a cdna and uses for treatment of copper transport disorders
WO2017070525A1 (en) 2015-10-22 2017-04-27 University Of Massachusetts Methods and compositions for treating metabolic imbalance in neurodegenerative disease
US11426469B2 (en) 2015-10-22 2022-08-30 University Of Massachusetts Prostate-targeting adeno-associated virus serotype vectors
WO2017075335A1 (en) 2015-10-28 2017-05-04 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
US11826433B2 (en) 2016-02-02 2023-11-28 University Of Massachusetts Method to enhance the efficiency of systemic AAV gene delivery to the central nervous system
CA3012344A1 (en) 2016-02-12 2017-08-17 University Of Massachusetts Anti-angiogenic mirna therapeutics for inhibiting corneal neovascularization
US11207426B2 (en) 2016-04-05 2021-12-28 University Of Massachusetts Compositions and methods for selective inhibition of grainyhead-like protein expression
US11413356B2 (en) 2016-04-15 2022-08-16 University Of Massachusetts Methods and compositions for treating metabolic imbalance
BR112018071156A2 (en) 2016-04-15 2019-03-12 The Trustees Of The University Of Pennsylvania gene therapy to treat mucopolysaccharidosis type ii
WO2017218450A1 (en) 2016-06-13 2017-12-21 The University Of North Carolina At Chapel Hill Optimized cln1 genes and expression cassettes and their use
WO2017218852A1 (en) 2016-06-15 2017-12-21 University Of Massachusetts Recombinant adeno-associated viruses for delivering gene editing molecules to embryonic cells
CN110650673B (en) 2016-08-30 2024-04-09 加利福尼亚大学董事会 Methods for biomedical targeting and delivery and devices and systems for practicing the methods
US10457940B2 (en) 2016-09-22 2019-10-29 University Of Massachusetts AAV treatment of Huntington's disease
JP2019530463A (en) 2016-10-13 2019-10-24 ユニバーシティ オブ マサチューセッツ AAV capsid design
CN108524952A (en) * 2017-03-06 2018-09-14 复旦大学 Adeno-associated virus AAV9 and its purposes in the preparation for preparing treatment dagger-axe Xie Shi diseases
US11603542B2 (en) 2017-05-05 2023-03-14 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
EP3654860A1 (en) 2017-07-17 2020-05-27 Voyager Therapeutics, Inc. Trajectory array guide system
US11512327B2 (en) 2017-08-03 2022-11-29 Voyager Therapeutics, Inc. Compositions and methods for delivery of AAV
EP3684938A1 (en) 2017-09-22 2020-07-29 The Trustees of the University of Pennsylvania Gene therapy for treating mucopolysaccharidosis type ii
AU2018352236A1 (en) 2017-10-16 2020-04-23 The Curators Of The University Of Missouri Treatment of amyotrophic lateral sclerosis (ALS)
US20200237799A1 (en) 2017-10-16 2020-07-30 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
AU2018365677A1 (en) * 2017-11-08 2020-05-14 Novartis Ag Means and method for preparing viral vectors and uses of same
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
US11306329B2 (en) 2018-02-19 2022-04-19 City Of Hope Adeno-associated virus compositions for restoring F8 gene function and methods of use thereof
EP3774854A1 (en) 2018-04-03 2021-02-17 Stridebio, Inc. Antibody-evading virus vectors
TW202015742A (en) 2018-05-15 2020-05-01 美商航海家醫療公司 Compositions and methods for delivery of aav
JP2021523914A (en) 2018-05-15 2021-09-09 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. Compositions and Methods for Treating Parkinson's Disease
EP3793615A2 (en) 2018-05-16 2021-03-24 Voyager Therapeutics, Inc. Directed evolution of aav to improve tropism for cns
WO2020010035A1 (en) 2018-07-02 2020-01-09 Voyager Therapeutics, Inc. Cannula system
EP3818161A1 (en) 2018-07-02 2021-05-12 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis and disorders associated with the spinal cord
WO2020023612A1 (en) 2018-07-24 2020-01-30 Voyager Therapeutics, Inc. Systems and methods for producing gene therapy formulations
US20210348242A1 (en) 2018-10-04 2021-11-11 Voyager Therapeutics, Inc. Methods for measuring the titer and potency of viral vector particles
US20210371470A1 (en) 2018-10-12 2021-12-02 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
TW202039858A (en) 2019-01-18 2020-11-01 美商航海家醫療公司 Methods and systems for producing aav particles
WO2020163475A1 (en) * 2019-02-05 2020-08-13 The Trustees Of Princeton University Recombinant nucleic acids containing alphaherpesvirus promoter sequences
CN109706176A (en) * 2019-03-12 2019-05-03 青岛大学附属医院 Transfection dorsal root ganglion satellite spongiocyte adenovirus vector construct method can be targeted
TW202102525A (en) 2019-03-21 2021-01-16 美商史崔德生物公司 Recombinant adeno-associated virus vectors
MX2021011701A (en) * 2019-03-28 2021-12-10 Massachusetts Gen Hospital Engineered adeno-associated (aav) vectors for transgene expression.
AU2020310201A1 (en) * 2019-07-10 2022-01-27 Locanabio, Inc. RNA-targeting knockdown and replacement compositions and methods for use
AU2020336332A1 (en) * 2019-08-29 2022-03-03 The Board Of Trustees Of The Leland Stanford Junior University Adeno-associated viral vectors for crossing the human blood brain barrier
US20220333133A1 (en) 2019-09-03 2022-10-20 Voyager Therapeutics, Inc. Vectorized editing of nucleic acids to correct overt mutations
MX2022004352A (en) 2019-10-17 2022-07-19 Stridebio Inc Adeno-associated viral vectors for treatment of niemann-pick disease type c.
TW202140791A (en) 2020-01-13 2021-11-01 美商霍蒙拉奇醫藥公司 Methods of treating phenylketonuria
CN116249712A (en) 2020-04-15 2023-06-09 沃雅戈治疗公司 TAU binding compounds
CN116096734A (en) 2020-05-13 2023-05-09 沃雅戈治疗公司 Redirection of tropism of AAV capsids
CN112121179A (en) * 2020-09-15 2020-12-25 山东兴瑞生物科技有限公司 Composition and application thereof in treating spinal muscular atrophy
US20240141378A1 (en) 2021-03-03 2024-05-02 Voyager Therapeutics, Inc. Controlled expression of viral proteins
CN115708866A (en) * 2021-08-23 2023-02-24 中国科学院深圳先进技术研究院 Application of bone origin factor RANKL in preparation of medicine or medicine composition for regulating and controlling central nervous system homeostasis
WO2023044483A2 (en) 2021-09-20 2023-03-23 Voyager Therapeutics, Inc. Compositions and methods for the treatment of her2 positive cancer
WO2023081648A1 (en) 2021-11-02 2023-05-11 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
TW202334181A (en) 2021-11-17 2023-09-01 美商航海家醫療公司 Aav capsid variants and uses thereof
WO2023154693A1 (en) 2022-02-08 2023-08-17 Voyager Therapeutics, Inc. 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
CN116064671A (en) * 2022-08-10 2023-05-05 深圳先进技术研究院 Application of gene delivery system in delivering genes to microglia
WO2024059739A1 (en) 2022-09-15 2024-03-21 Voyager Therapeutics, Inc. Tau binding compounds

Family Cites Families (38)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IL122349A0 (en) 1995-06-07 1998-04-05 Univ Yale Adeno-associated viral vectors
US20040076613A1 (en) 2000-11-03 2004-04-22 Nicholas Mazarakis Vector system
FR2756297B1 (en) 1996-11-22 1999-01-08 Centre Nat Rech Scient PROCESS FOR PRODUCING RECOMBINANT VIRUSES
JP2001512142A (en) 1997-07-31 2001-08-21 カイロン コーポレイション Method for enabling re-administration of AAV vectors via immunosuppression of the 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
EP1218035A2 (en) 1999-09-29 2002-07-03 The Trustees Of The University Of Pennsylvania Rapid peg-modification of viral vectors
AU1775901A (en) 1999-11-17 2001-05-30 Avigen, Inc. Recombinant adeno-associated virus virions for the treatment of lysosomal disorders
ES2327609T3 (en) 2000-06-01 2009-11-02 University Of North Carolina At Chapel Hill PROCEDURES AND COMPOUNDS TO CONTROL THE RELEASE OF RECONBINANT PARVOVIRUS VECTORS.
AU2001269723B9 (en) 2000-06-01 2006-11-16 University Of North Carolina At Chapel Hill Duplexed parvovirus vectors
US20050032219A1 (en) 2001-07-03 2005-02-10 Patrick Aubourg Methods of administering vectors to synaptically connected neurons
CA2406745C (en) * 2001-11-13 2006-01-10 The Trustees Of The University Of Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
AU2002359284A1 (en) 2001-12-17 2003-06-30 The Trustees Of The University Of Pennsylvania Adeno-associated virus (aav) serotype 9 sequences, vectors containing same, and uses therefor
AU2002359786A1 (en) 2001-12-19 2003-07-09 Hiroaki Mizukami Adeno-associated virus-mediated delivery of gdnf to skeletal muscles
US6998118B2 (en) * 2001-12-21 2006-02-14 The Salk Institute For Biological Studies Targeted retrograde gene delivery for neuronal protection
WO2003055983A2 (en) * 2001-12-21 2003-07-10 The Salk Institute For Biological Studies Targeted retrograde gene delivery to motor neurons
US20060018882A1 (en) * 2004-06-21 2006-01-26 Kaemmerer William F Medical devices and methods for delivering compositions to cells
JP2006521825A (en) * 2003-03-31 2006-09-28 ユニバーシテイ・オブ・アイオワ・リサーチ・フアウンデーシヨン Compounds and methods for enhancing rAAV introduction
WO2004106517A1 (en) * 2003-06-03 2004-12-09 Benitec Australia Limited Double-stranded nucleic acid
HUE054046T2 (en) 2003-06-19 2021-08-30 Genzyme Corp Aav virions with decreased immunoreactivity and uses therefor
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
AU2004278684B2 (en) * 2003-09-30 2011-05-12 The Trustees Of The University Of Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses therefor
WO2005037211A2 (en) * 2003-10-14 2005-04-28 Neurologix Research, Inc. Methods and compositions for the treatment of neurological disease
US8734810B2 (en) 2003-10-29 2014-05-27 Allergan, Inc. Botulinum toxin treatments of neurological and neuropsychiatric disorders
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
WO2005072364A2 (en) 2004-01-27 2005-08-11 University Of Florida A modified baculovirus expression system for production of pseudotyped raav vector
US8926958B2 (en) * 2004-04-06 2015-01-06 Cedars-Sinai Medical Center Prevention and treatment of vascular disease with recombinant adeno-associated virus vectors encoding apolipoprotein A-I and apolipoprotein A-I milano
US20060110364A1 (en) * 2004-08-20 2006-05-25 Ludwig Institute For Cancer Research Vector-mediated delivery of polynucleotides encoding soluble VEGF receptors
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
CA2606490C (en) 2005-05-02 2018-02-06 Genzyme Corporation Gene therapy for spinal cord disorders
WO2007089632A2 (en) * 2006-01-27 2007-08-09 The University Of North Carolina At Chapel Hill Heparin and heparan sulfate binding chimeric vectors
US20070280906A1 (en) 2006-06-03 2007-12-06 Ognjen Petras Method to generate mirrored adenoassociated viral vectors
US20100221225A1 (en) * 2007-02-23 2010-09-02 University Of Florida Research Foundation, Inc 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
CN101815536A (en) 2007-10-01 2010-08-25 爱尔康研究有限公司 Self complementary AAV-mediated delivery of interfering RNA molecules to treat or prevent ocular disorders
EP2058401A1 (en) * 2007-10-05 2009-05-13 Genethon Widespread gene delivery to motor neurons using peripheral injection of AAV vectors
US11219696B2 (en) * 2008-12-19 2022-01-11 Nationwide Children's Hospital Delivery of polynucleotides using recombinant AAV9

Also Published As

Publication number Publication date
NO2022048I1 (en) 2022-11-16
EP2527457B1 (en) 2019-02-20
HRP20220992T3 (en) 2022-11-11
EP3505635B1 (en) 2022-05-18
JP6309373B2 (en) 2018-04-11
PT2527457T (en) 2019-05-31
PL3505635T3 (en) 2022-09-12
US9926574B2 (en) 2018-03-27
JP2014221789A (en) 2014-11-27
PT2212424E (en) 2014-07-25
CA2999607A1 (en) 2009-04-09
US20180066283A1 (en) 2018-03-08
CN105087650A (en) 2015-11-25
ES2924892T3 (en) 2022-10-11
PL2212424T3 (en) 2014-11-28
FI2212424T4 (en) 2023-12-15
DK3505635T3 (en) 2022-08-22
ES2482094T3 (en) 2014-08-01
PT3505635T (en) 2022-08-12
EP2058401A1 (en) 2009-05-13
FR20C1057I1 (en) 2020-12-25
HRP20140643T1 (en) 2014-11-07
LT3505635T (en) 2022-09-12
CA2701561A1 (en) 2009-04-09
EP2212424B2 (en) 2023-11-01
LTPA2022525I1 (en) 2022-12-12
FR20C1057I2 (en) 2024-02-16
SI3505635T1 (en) 2022-10-28
HUS2200047I1 (en) 2022-12-28
DK2212424T3 (en) 2014-07-14
US20200010852A1 (en) 2020-01-09
EP2212424A1 (en) 2010-08-04
US11767538B2 (en) 2023-09-26
EP3505635A1 (en) 2019-07-03
ES2727034T3 (en) 2019-10-11
EP2212424B1 (en) 2014-06-11
JP5620819B2 (en) 2014-11-05
CN101883859A (en) 2010-11-10
EP2527457A1 (en) 2012-11-28
HUE059864T2 (en) 2023-01-28
CA2701561C (en) 2018-05-15
WO2009043936A1 (en) 2009-04-09
SI2212424T1 (en) 2014-12-31
JP2010540598A (en) 2010-12-24
CY1125608T1 (en) 2023-06-09
EP3854878A1 (en) 2021-07-28
US20100240739A1 (en) 2010-09-23

Similar Documents

Publication Publication Date Title
FR22C1056I1 (en) WIDESPREAD GENE DELIVERY TO MOTOR NEURONS USING PERIPHERAL AAV VECTOR INJECTION
FR20C1058I1 (en) GENE DELIVERY TO THE CENTRAL NERVOUS SYSTEM BY PERIPHERAL DELIVERY OF AAV VECTOR
BRPI0911743A2 (en) "peptide"
BRPI0906082A2 (en) "moisturizing hand sanitizer"
BRPI0916123A2 (en) "concentrated shampoo composition"
ITMI20062388A1 (en) DEVICE FOR WHEEL ALIGNMENT
ATE496924T1 (en) PRODRUGS OF 5-AMINO-3-(3'-DEOXY-BETA-D-RIBOFURANOSYL)-THIAZOLÄ4,5-DÜPYRIMIDINE-2,7-DIONE
BRPI0914503A2 (en) "concentrated shampoo composition"
BRPI0719007A2 (en) OXYR GENES OF CORINEFORM BACTERIA GENE
ITRM20080103A1 (en) INDUCIBLE GENE EXPRESSION SYSTEM IN CHLAMYDOMONAS
FR2905600B1 (en) TREATMENT OF VERTIGS BY ACETYL-L-LEUCINE.
BRPI0916080A2 (en) "liquid or solid-soft personal cleansing composition"
ITBO20060234A1 (en) DEVICE FOR ORIENTATION OF EXTENDED ITEMS.
UA13109S (en) WOMEN'S FUR JACKET "AZA"
UA18444S (en) WOMEN'S FUR JACKET "BLUE" MODEL-MV-99
UA20180S (en) WOMEN'S FUR JACKET "FIELDS". MODEL-MV-127
UA17312S (en) WOMEN'S FUR JACKET "DIBROVA". MODEL-MV
UA17029S (en) WOMEN'S FUR JACKET "AMALIA". MODEL-MV
UA17533S (en) WOMEN'S FUR JACKET "SULTANKA". MODEL-MV
UA17031S (en) WOMEN'S FUR JACKET "MARICHKA". MODEL-MV
UA17532S (en) WOMEN'S FUR JACKET "MARSIANA". MODEL-MV
UA17032S (en) WOMEN'S FUR JACKET "GRACIELLA". MODEL-MV
UA17033S (en) WOMEN'S SMERICHKA FUR JACKET. MODEL-MV
UA17030S (en) WOMEN'S FUR JACKET "CLASSIC". MODEL-MV
UA15015S (en) WOMEN'S FUR JACKET "POLONINA" MODEL-MV