PL3137497T5 - Wektory AAV do terapii genowej siatkówki i OUN - Google Patents

Wektory AAV do terapii genowej siatkówki i OUN

Info

Publication number
PL3137497T5
PL3137497T5 PL15723386.7T PL15723386T PL3137497T5 PL 3137497 T5 PL3137497 T5 PL 3137497T5 PL 15723386 T PL15723386 T PL 15723386T PL 3137497 T5 PL3137497 T5 PL 3137497T5
Authority
PL
Poland
Prior art keywords
retinal
gene therapy
aav vectors
cns gene
cns
Prior art date
Application number
PL15723386.7T
Other languages
English (en)
Polish (pl)
Other versions
PL3137497T4 (pl
PL3137497T3 (pl
Inventor
Abraham Scaria
Jennifer Sullivan
Lisa M. Stanek
Lamya Shihabuddin
Original Assignee
Genzyme Corporation
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=53189202&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=PL3137497(T5) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Genzyme Corporation filed Critical Genzyme Corporation
Publication of PL3137497T3 publication Critical patent/PL3137497T3/pl
Publication of PL3137497T4 publication Critical patent/PL3137497T4/pl
Publication of PL3137497T5 publication Critical patent/PL3137497T5/pl

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • 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
    • 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
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A61P27/06—Antiglaucoma agents or miotics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C07K14/01—DNA viruses
    • C07K14/015—Parvoviridae, e.g. feline panleukopenia virus, human parvovirus
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/71—Receptors; Cell surface antigens; Cell surface determinants for growth factors; for growth regulators
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141—MicroRNAs, miRNAs
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific
    • 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
    • C12N2750/14022—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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
    • C12N2750/14023—Virus like particles [VLP]
    • 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
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • 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
    • C12N2750/14145—Special targeting system for viral vectors

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Virology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Microbiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Immunology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Epidemiology (AREA)
  • Cell Biology (AREA)
  • Toxicology (AREA)
  • Psychology (AREA)
  • Pain & Pain Management (AREA)
  • Psychiatry (AREA)
PL15723386.7T 2014-05-02 2015-05-02 Wektory AAV do terapii genowej siatkówki i OUN PL3137497T5 (pl)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201461988131P 2014-05-02 2014-05-02
US201562114575P 2015-02-10 2015-02-10
EP15723386.7A EP3137497B2 (en) 2014-05-02 2015-05-02 Aav vectors for retinal and cns gene therapy
PCT/US2015/028966 WO2015168666A2 (en) 2014-05-02 2015-05-02 Aav vectors for retinal and cns gene therapy

Publications (3)

Publication Number Publication Date
PL3137497T3 PL3137497T3 (pl) 2021-10-25
PL3137497T4 PL3137497T4 (pl) 2023-04-11
PL3137497T5 true PL3137497T5 (pl) 2025-06-09

Family

ID=53189202

Family Applications (1)

Application Number Title Priority Date Filing Date
PL15723386.7T PL3137497T5 (pl) 2014-05-02 2015-05-02 Wektory AAV do terapii genowej siatkówki i OUN

Country Status (32)

Country Link
US (3) US10982228B2 (https=)
EP (3) EP3137497B2 (https=)
JP (4) JP6741591B2 (https=)
KR (3) KR102863726B1 (https=)
CN (2) CN106661591B (https=)
AU (2) AU2015252797C1 (https=)
CA (1) CA2946593A1 (https=)
CL (2) CL2016002713A1 (https=)
CR (1) CR20160555A (https=)
DK (1) DK3137497T4 (https=)
DO (1) DOP2016000280A (https=)
EA (1) EA034355B1 (https=)
ES (1) ES2879636T5 (https=)
FI (1) FI3137497T4 (https=)
GT (1) GT201600227A (https=)
HR (1) HRP20211024T4 (https=)
HU (1) HUE054768T2 (https=)
IL (2) IL292951B2 (https=)
LT (1) LT3137497T (https=)
MX (2) MX2016014220A (https=)
MY (1) MY188987A (https=)
PE (1) PE20170260A1 (https=)
PH (1) PH12016502046A1 (https=)
PL (1) PL3137497T5 (https=)
PT (1) PT3137497T (https=)
RS (1) RS62078B2 (https=)
SG (3) SG11201607991WA (https=)
SI (1) SI3137497T2 (https=)
TW (1) TWI686476B (https=)
UY (1) UY36106A (https=)
WO (1) WO2015168666A2 (https=)
ZA (1) ZA201606763B (https=)

Families Citing this family (184)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9217155B2 (en) 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
HRP20250897T1 (hr) 2009-05-02 2025-09-26 Genzyme Corporation Genska terapija za neurodegenerativne poremećaje
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
EP2561073B1 (en) 2010-04-23 2016-08-24 University of Massachusetts Cns targeting aav vectors and methods of use thereof
US9546369B2 (en) 2010-04-23 2017-01-17 University Of Massachusetts Multicistronic expression constructs
EP3444346B1 (en) 2010-04-23 2022-07-27 University of Massachusetts Aav-based treatment of cholesterol-related disorders
US8663624B2 (en) 2010-10-06 2014-03-04 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
EP3318635A1 (en) 2011-04-21 2018-05-09 University of Massachusetts Raav-based compositions and methods for treating alpha-1 anti-trypsin deficiencies
CN105755044A (zh) 2011-04-22 2016-07-13 加利福尼亚大学董事会 具有变异衣壳的腺相关病毒病毒体及其使用方法
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
EP3003391B1 (en) 2013-05-31 2021-09-22 The Regents of The University of California Adeno-associated virus variants and methods of use thereof
ES2739288T3 (es) 2013-09-13 2020-01-30 California Inst Of Techn Recuperación selectiva
US10072251B2 (en) 2014-02-19 2018-09-11 University Of Massachusetts Recombinant AAVS having useful transcytosis properties
EP3800191B1 (en) 2014-03-17 2025-08-20 Adverum Biotechnologies, Inc. Compositions and methods for enhanced gene expression in cone cells
CA2942515C (en) 2014-03-18 2025-12-09 University Of Massachusetts Raav-based compositions and methods for treating amyotrophic lateral sclerosis
WO2015164786A1 (en) 2014-04-25 2015-10-29 University Of Massachusetts Recombinant aav vectors useful for reducing immunity against transgene products
LT3137497T (lt) 2014-05-02 2021-07-26 Genzyme Corporation Aav vektoriai, skirti tinklainės ir cns genų terapijai
CA2947468C (en) * 2014-05-14 2022-10-25 Laboratorios Del Dr. Esteve S.A. Adenoassociated virus vectors for the treatment of lysosomal storage disorders
WO2015187825A2 (en) 2014-06-03 2015-12-10 University Of Massachusetts Compositions and methods for modulating dysferlin expression
US10370432B2 (en) 2014-10-03 2019-08-06 University Of Massachusetts Heterologous targeting peptide grafted AAVS
EP3200830B1 (en) 2014-10-03 2020-09-09 University of Massachusetts High efficiency library-identified aav vectors
CN107073051B (zh) 2014-10-21 2021-08-24 马萨诸塞大学 重组aav变体及其用途
CR20170406A (es) 2015-02-10 2017-11-14 Genzyme Corp Arni variante
CN107530447A (zh) 2015-02-10 2018-01-02 建新公司 病毒颗粒至纹状体和皮质的增强递送
EP3256170B1 (en) 2015-02-13 2020-09-23 University of Massachusetts Compositions and methods for transient delivery of nucleases
US11021519B2 (en) 2015-03-02 2021-06-01 Adverum Biotechnologies, Inc. Compositions and methods for intravitreal delivery of polynucleotides to retinal cones
EP3268481B1 (en) * 2015-03-11 2020-05-27 The United States of America, as represented by The Secretary, Department of Health and Human Services Rpgr vectors for treating x-linked retinitis pigmentosa
JP6836999B2 (ja) 2015-03-24 2021-03-03 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニアＴｈｅ Ｒｅｇｅｎｔｓ Ｏｆ Ｔｈｅ Ｕｎｉｖｅｒｓｉｔｙ Ｏｆ Ｃａｌｉｆｏｒｎｉａ アデノ随伴ウイルス変異体及びその使用方法
RS61907B1 (sr) 2015-04-06 2021-06-30 Subdomain Llc Polipeptidi koji sadrže de novo vezujući domen i njihova primena
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
TWI707951B (zh) 2015-04-08 2020-10-21 美商健臻公司 過大腺相關載體之製造
US11046955B2 (en) 2015-04-24 2021-06-29 University Of Massachusetts Modified AAV constructs and uses thereof
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
ES2865487T3 (es) 2015-09-28 2021-10-15 Univ North Carolina Chapel Hill Métodos y composiciones para vectores virales que evaden los anticuerpos
EP3364997B1 (en) 2015-10-22 2024-01-17 University of Massachusetts Aspartoacylase gene therapy in the treatment of canavan disease
WO2017070516A1 (en) 2015-10-22 2017-04-27 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)
KR102423442B1 (ko) * 2015-12-11 2022-07-20 캘리포니아 인스티튜트 오브 테크놀로지 아데노-관련 바이러스를 지시하기 위한 타겟팅 펩타이드
MX2018007330A (es) * 2015-12-15 2019-03-14 Genzyme Corp Vectores virales adeno-asociados para tratar mucolipidosis de tipo ii.
CA3011939A1 (en) * 2016-02-02 2017-08-10 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
WO2017147477A1 (en) * 2016-02-26 2017-08-31 University Of Florida Research Foundation, Inc. Aav heparin mutants that display significantly improved eye and brain transduction
US11207426B2 (en) 2016-04-05 2021-12-28 University Of Massachusetts Compositions and methods for selective inhibition of grainyhead-like protein expression
CN107287238B (zh) * 2016-04-11 2020-10-16 厦门继景生物技术有限责任公司 一种基因载体及其用于治疗雷柏氏先天性黑矇2型疾病的基因治疗药物
US11413356B2 (en) 2016-04-15 2022-08-16 University Of Massachusetts Methods and compositions for treating metabolic imbalance
GB201608046D0 (en) 2016-05-09 2016-06-22 Cambridge Entpr Ltd And Syndey Children S Hospitals Network Randwick And Westmead Incorporating The Treatment of complement-mediated disorders
KR102427379B1 (ko) * 2016-05-18 2022-08-02 보이저 테라퓨틱스, 인크. 헌팅톤 질환을 치료하기 위한 조성물 및 방법
US11882815B2 (en) 2016-06-15 2024-01-30 University Of Massachusetts Recombinant adeno-associated viruses for delivering gene editing molecules to embryonic cells
WO2018009814A1 (en) * 2016-07-08 2018-01-11 The Trustees Of The University Of Pennsylvania Methods and compositions for treatment of disorders and diseases involving rdh12
EP3485005A1 (en) * 2016-07-12 2019-05-22 The University Of Manchester Gene therapy
AU2017302013B2 (en) 2016-07-29 2022-05-26 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
US20190262373A1 (en) * 2016-08-16 2019-08-29 The University Of North Carolina At Chapel Hill Methods and compositions for targeted gene transfer
IL315358A (en) * 2016-08-18 2024-11-01 Univ California CRISPR-CAS genome engineering using a modular AAV delivery system
EP3500279A4 (en) * 2016-08-19 2020-04-22 Calimmune, Inc. METHODS AND COMPOSITIONS FOR THE TREATMENT OF CANINE CONDITIONS USING A RECOMBINANT SELF-COMPLEMENTARY ADENO-ASSOCIATED VIRUS.
JP7165410B2 (ja) 2016-08-19 2022-11-04 ユニバーシティ オブ フロリダ リサーチ ファウンデーション，インコーポレイティド 組換え自己相補的アデノ随伴ウイルスを使用して疾患を治療するための方法及び組成物
US10457940B2 (en) 2016-09-22 2019-10-29 University Of Massachusetts AAV treatment of Huntington's disease
CA3038292A1 (en) 2016-09-28 2018-04-05 Cohbar, Inc. Therapeutic mots-c related peptides
EP3518985A4 (en) * 2016-09-29 2020-08-05 University of Florida Research Foundation, Incorporated AAVRH.10 VARIANTS WITH HOST ANTIBODY EXHAUST CAPACITIES AND MODIFIED TISSUE TARGETING PROPERTIES
AU2017341849B2 (en) 2016-10-13 2024-03-21 University Of Massachusetts AAV capsid designs
CA3040179A1 (en) 2016-10-19 2018-04-26 Adverum Biotechnologies, Inc. Modified aav capsids and uses thereof
KR20190088561A (ko) 2016-12-07 2019-07-26 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 인터류킨-1 수용체 길항제(IL-1RA) cDNA
WO2018145009A1 (en) * 2017-02-06 2018-08-09 University Of Tennessee Research Foundation Dna-zyme based methods & compositions for treating huntington's disease
US11117930B2 (en) 2017-02-23 2021-09-14 Adrx, Inc. Peptide inhibitors of transcription factor aggregation
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
US10898585B2 (en) 2017-04-14 2021-01-26 Ptc Therapeutics .Inc. Gene therapy for AADC deficiency
WO2018200419A1 (en) * 2017-04-23 2018-11-01 The Trustees Of The University Of Pennsylvania Viral vectors comprising engineered aav capsids and compositions containing the same
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
US11859179B2 (en) 2017-05-09 2024-01-02 University Of Massachusetts Methods of treating amyotrophic lateral sclerosis (ALS)
CA3061968A1 (en) 2017-05-10 2018-11-15 Massachusetts Eye And Ear Infirmary Methods and compositions for modifying assembly-activating protein (aap)-dependence of viruses
EP3634978A1 (en) 2017-06-07 2020-04-15 Adrx, Inc. Tau aggregation inhibitors
CA3053154A1 (en) 2017-06-30 2019-01-03 The Regents Of The University Of California Adeno-associated virus virions with variant capsids and methods of use thereof
SG11202000840YA (en) 2017-07-31 2020-02-27 Reflection Biotechnologies Ltd Cellular models of and therapies for ocular diseases
EP3808849A1 (en) 2017-08-03 2021-04-21 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
JP2020534788A (ja) 2017-08-28 2020-12-03 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア アデノ随伴ウイルスカプシド変異体及びその使用方法
US20200377887A1 (en) * 2017-09-22 2020-12-03 Voyager Therapeutics, Inc. Compositions and methods of treating huntington's disease
CN111448321A (zh) 2017-09-22 2020-07-24 马萨诸塞大学 Sod1双表达载体及其用途
CN111356763B (zh) 2017-09-22 2024-03-12 建新公司 变体RNAi
US12043848B2 (en) * 2017-10-16 2024-07-23 Vigeneron Gmbh AAV vectors
CN111867635A (zh) * 2017-10-20 2020-10-30 法国国家卫生及研究医学协会 包括视网膜下递送治疗有效量的重组aav9衍生载体的在对象的视锥细胞光感受器中表达目的多核苷酸的方法
US11464803B2 (en) 2017-11-14 2022-10-11 Arcellx, Inc. D-domain containing polypeptides and uses thereof
KR20250070609A (ko) 2017-11-14 2025-05-20 아르셀엑스, 인크. 다기능성 면역 세포 요법
MX2020005033A (es) * 2017-11-15 2020-10-28 Friedrich Miescher Institute For Biomedical Res Promotor especifico de las celulas del epitelio pigmentario retinal en primates.
KR102704296B1 (ko) * 2017-11-15 2024-09-05 더 리젠츠 오브 더 유니버시티 오브 미시간 Rdh12 암호화 영역을 포함하는 바이러스 벡터 및 망막 이영양증을 치료하는 방법
CA3082450A1 (en) 2017-11-21 2019-05-31 Crispr Therapeutics Ag Materials and methods for treatment of autosomal dominant retinitis pigmentosa
CN108085316B (zh) * 2017-12-25 2021-02-09 中国人民解放军第四军医大学 一种通过抑制微小核糖核酸来促进小鼠视网膜前体细胞体外扩增的方法
CN112153990A (zh) * 2018-03-23 2020-12-29 纽约市哥伦比亚大学理事会 用于常染色体显性疾病的基因编辑
MX2020010465A (es) 2018-04-03 2021-01-08 Vectores de virus para direccionamiento a tejidos oftalmicos.
AU2019247748A1 (en) 2018-04-03 2020-10-08 Ginkgo Bioworks, Inc. Antibody-evading virus vectors
EP3774852A1 (en) 2018-04-03 2021-02-17 Stridebio, Inc. Antibody-evading virus vectors
MX2020011386A (es) 2018-04-27 2021-01-29 Spacecraft Seven Llc Terapia genica para la degeneracion del snc.
KR20210019996A (ko) 2018-05-15 2021-02-23 보이저 테라퓨틱스, 인크. 파킨슨병의 치료를 위한 조성물 및 방법
EP3813845A4 (en) * 2018-05-15 2022-10-26 President And Fellows Of Harvard College Viral vectors exhibiting improved gene delivery properties
WO2019222329A1 (en) 2018-05-15 2019-11-21 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
US20210214749A1 (en) 2018-05-16 2021-07-15 Voyager Therapeutics, Inc. Directed evolution
CN112204145B (zh) * 2018-06-01 2024-03-26 佛罗里达大学研究基金会有限公司 用于治疗显性视网膜色素变性的组合物和方法
WO2019236995A1 (en) 2018-06-08 2019-12-12 University Of Massachusetts Antisense oligonucleotides to restore dysferlin protein expression in dysferlinopathy patient cells
KR102931619B1 (ko) 2018-08-03 2026-02-26 젠자임 코포레이션 알파-시뉴클레인에 대한 변이형 RNAi
CN109022487A (zh) * 2018-08-15 2018-12-18 上海市第人民医院 一种表达p65的基因载体及其用于治疗视网膜神经节细胞变性的基因治疗药物
CN113966399A (zh) 2018-09-26 2022-01-21 加州理工学院 用于靶向基因疗法的腺相关病毒组合物
EP3856762A1 (en) 2018-09-28 2021-08-04 Voyager Therapeutics, Inc. Frataxin expression constructs having engineered promoters and methods of use thereof
WO2020077091A1 (en) * 2018-10-10 2020-04-16 Wisconsin Alumni Research Foundation Kir 7.1 gene therapy vectors and methods of using the same
AU2019357039B2 (en) * 2018-10-12 2025-10-09 Genzyme Corporation Generation of improved human pah for treatment of severe PKU by liver-directed gene replacement therapy
US20210371470A1 (en) 2018-10-12 2021-12-02 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
UY38407A (es) 2018-10-15 2020-05-29 Novartis Ag Anticuerpos estabilizadores de trem2
US20230061069A1 (en) * 2018-11-13 2023-03-02 Jayandharan Giridhara Rao A process for producing a plurality of sumoylation target-site modified aav vector and product thereof
CA3120923A1 (en) * 2018-11-26 2020-06-04 Universitat Autonoma De Barcelona Fibroblast growth factor 21 (fgf21) gene therapy
WO2020117715A1 (en) 2018-12-03 2020-06-11 Board Of Regents, The University Of Texas System Oligo-benzamide analogs and their use in cancer treatment
US20220089670A1 (en) * 2018-12-28 2022-03-24 University Of Rochester Gene Therapy for BEST1 Dominant Mutations
DK3906066T5 (da) * 2019-01-04 2024-08-05 Ultragenyx Pharmaceutical Inc Geneterapikonstruktioner til behandling af wilsons sygdom
SG11202107645RA (en) 2019-01-18 2021-08-30 Voyager Therapeutics Inc Methods and systems for producing aav particles
EP3914229A4 (en) * 2019-01-23 2022-11-02 University of Florida Research Foundation, Incorporated Highly efficient transduction and lateral spread in the retina by a novel aav virus enhanced by rational design
SG11202107761SA (en) 2019-01-28 2021-08-30 Cohbar Inc Therapeutic peptides
KR20210148101A (ko) 2019-02-04 2021-12-07 프리라인 테라퓨틱스 리미티드 폴리뉴클레오티드
JP7597380B2 (ja) * 2019-03-04 2024-12-10 アドヴェラム バイオテクノロジーズ， インコーポレイテッド 対側眼へのａａｖ遺伝子治療の逐次的硝子体内投与
AU2020233396B2 (en) 2019-03-04 2025-09-11 Duke University Compositions and methods for the diagnosis and treatment of retinopathies
JP7663504B2 (ja) * 2019-03-08 2025-04-16 ユニベルシテ パリ－サクレー 遺伝子置換による希少な眼疾患のための改善された治療方法
AR118465A1 (es) 2019-03-21 2021-10-06 Stridebio Inc Vectores de virus adenoasociados recombinantes
JP7664848B2 (ja) * 2019-04-10 2025-04-18 プリベイル セラピューティクス，インコーポレーテッド リソソーム障害のための遺伝子療法
US20240124889A1 (en) 2019-05-07 2024-04-18 Voyager Therapeutics, Inc. Compositions and methods for the vectored augmentation of protein destruction, expression and/or regulation
US12491263B2 (en) 2019-05-13 2025-12-09 University of Pittsburgh—of the Commonwealth System of Higher Education Polymer-based implant for retinal therapy and methods of making and using the same
US20220233655A1 (en) * 2019-05-17 2022-07-28 Research Institute At Nationwide Children's Hospital Improved delivery of gene therapy vectors to retinal cells using a glycoside hydrolase enzyme
WO2020251954A1 (en) * 2019-06-10 2020-12-17 Homology Medicines, Inc. Adeno-associated virus compositions for arsa gene transfer and methods of use thereof
CA3144986A1 (en) * 2019-06-27 2020-12-30 University Of Florida Research Foundation, Incorporated Enhancing aav-mediated transduction of ocular tissues with hyaluronic acid
US11446084B2 (en) 2019-07-12 2022-09-20 Neuralink Corp. Laser drilling of pia mater
MX2022000551A (es) * 2019-07-15 2022-05-18 Meiragtx Uk Ii Ltd Proteínas de la cápside de aav modificadas para el tratamiento de una enfermedad artrítica.
CN110295151A (zh) * 2019-07-17 2019-10-01 博鑫仪器(天津)有限公司 一类腺依赖病毒病毒重组质粒、重组病毒及构建方法
WO2021041324A2 (en) * 2019-08-23 2021-03-04 Duke University Compositions and methods for the treatment of pathological pain and itch
WO2021046169A1 (en) * 2019-09-03 2021-03-11 University Of Cincinnati Methods and compositions for the treatment of als
WO2021046155A1 (en) 2019-09-03 2021-03-11 Voyager Therapeutics, Inc. Vectorized editing of nucleic acids to correct overt mutations
EP4041755A4 (en) * 2019-10-10 2023-10-25 Solid Biosciences Inc. Modified aav capsids and uses thereof
US12611436B2 (en) 2019-10-17 2026-04-28 Sarepta Therapeutics, Inc. AAV transfer cassette
JP2022551739A (ja) 2019-10-17 2022-12-13 ストライドバイオ，インコーポレイテッド ニーマン・ピック病ｃ型の治療のためのアデノ随伴ウイルスベクター
WO2021081217A1 (en) * 2019-10-23 2021-04-29 The Trustees Of The University Of Pennsylvania Compositions for drg-specific reduction of transgene expression
WO2021092300A1 (en) * 2019-11-08 2021-05-14 President And Fellows Of Harvard College Viral capsid polypeptides
CN113025618B (zh) * 2019-12-24 2024-02-06 朗信启昇(苏州)生物制药有限公司 一种x连锁遗传性视网膜劈裂症的基因治疗方案与应用
EP4097121A1 (en) * 2020-01-29 2022-12-07 Genzyme Corporation Modified adeno-associated viral capsid proteins for ocular gene therapy and methods of use thereof
JP2023513208A (ja) * 2020-02-07 2023-03-30 ザ トラスティーズ オブ コロンビア ユニバーシティー イン ザ シティー オブ ニューヨーク 神経変性の発症を遅延させるかまたは神経変性を処置するためのメタボロームのリプログラミング
EP4107169A4 (en) * 2020-02-18 2024-07-17 The University of North Carolina at Chapel Hill CAPSID-AAV PROMOTER INTERACTIONS AND CELL SELECTIVE GENE EXPRESSION
EP4110913A4 (en) * 2020-02-28 2024-07-24 University of Massachusetts OLIGONUCLEOTIDES FOR PRNP MODULATION
EP4117644A4 (en) 2020-03-11 2024-08-14 Massachusetts Eye and Ear Infirmary GENE THERAPY FOR NMNAT1-ASSOCIATED RETINAL DEGENERATION
WO2021202494A1 (en) * 2020-03-31 2021-10-07 University Of Massachusetts Aav capsids variants and uses thereof
JP2023523573A (ja) * 2020-04-14 2023-06-06 ジェネトン 酸性セラミダーゼ欠乏症を処置するためのベクター
JP7713956B2 (ja) 2020-04-15 2025-07-28 ボイジャー セラピューティクス インコーポレイテッド タウ結合化合物
TWI900553B (zh) 2020-05-13 2025-10-11 美商航海家醫療公司 Aav蛋白殼之趨性重定向
WO2021247995A2 (en) 2020-06-04 2021-12-09 Voyager Therapeutics, Inc. Compositions and methods of treating neuropathic pain
WO2021252989A2 (en) * 2020-06-13 2021-12-16 Oculogenex Inc. Aav-mediated gene transfer for retinopathy
CN113952472A (zh) * 2020-07-21 2022-01-21 英斯培瑞有限公司 用于治疗眼部疾病的组合物和方法
WO2022017630A1 (en) 2020-07-23 2022-01-27 Ucl Business Ltd GENE THERAPY VECTOR FOR eEF1A2 AND USES THEREOF
BR112023001456A2 (pt) 2020-07-27 2023-04-11 Voyager Therapeutics Inc Composições e métodos para o tratamento de distúrbios neurológicos relacionados à deficiência de beta glicosilceramidase
WO2022026410A2 (en) 2020-07-27 2022-02-03 Voyager Therapeutics, Inc Compositions and methods for the treatment of niemann-pick type c1 disease
KR20230042468A (ko) 2020-08-05 2023-03-28 스페이스크래프트 세븐, 엘엘씨 Csrp3 (시스테인 및 글리신 풍부 단백질 3) 유전자 요법
KR20230043869A (ko) 2020-08-07 2023-03-31 스페이스크래프트 세븐, 엘엘씨 Aav 벡터를 사용한 플라코필린-2(pkp2) 유전자 요법
AU2021328475A1 (en) * 2020-08-19 2023-03-16 Sarepta Therapeutics, Inc. Adeno-associated virus vectors for treatment of Rett syndrome
CA3191543A1 (en) * 2020-09-04 2022-03-10 Lei Cao Novel engineered capsid serotype of recombinant adeno-associated viral vector with enhanced transduction efficiency and widespread distribution in the brain
WO2022087238A1 (en) * 2020-10-21 2022-04-28 Wisconsin Alumni Research Foundation Anti-apoptotic vector and method of using the same
US20220160825A1 (en) * 2020-11-25 2022-05-26 The Penn State Research Foundation Brain repair after traumatic brain injury through neurod1-mediated astrocyte-to-neuron conversion
JP2024507860A (ja) * 2021-02-22 2024-02-21 ザ・リージエンツ・オブ・ザ・ユニバーシテイ・オブ・コロラド、ア・ボデイー・コーポレイト 眼病態に対するウイルスベクターベースの遺伝子療法
CN115044614B (zh) * 2021-03-09 2023-10-20 上海目镜生物医药科技有限公司 一种用于基因靶向与表达的aav-8型血清型的改造型载体及其构建方法及应用
CA3216809A1 (en) * 2021-04-26 2022-11-03 Brendan BEAHM Compositions and methods for treating ngyl1 deficiency
US20240316219A1 (en) * 2021-05-03 2024-09-26 North Carolina State University Compositions and methods related to the treatment of ocular diseases in equines
EP4399314A4 (en) * 2021-09-06 2025-09-10 Huidagene Therapeutics Singapore Pte Ltd TREATMENT OF EYE DISEASES AND DISORDERS ASSOCIATED WITH RPE65
US20250049955A1 (en) 2021-11-17 2025-02-13 Voyager Therapeutics, Inc. Compositons and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
TW202325850A (zh) 2021-11-29 2023-07-01 大陸商上海瑞宏迪醫藥有限公司 Aadc、gdnf多核苷酸及其用於治療帕金森病
AU2023220237A1 (en) 2022-02-21 2024-08-15 Shanghai Regenelead Therapies Co., Ltd Vegf-binding molecule and pharmaceutical use thereof
US20250213727A1 (en) * 2022-03-25 2025-07-03 Regenxbio Inc. Dominant-negative tumor necrosis factor alpha adeno-associated virus gene therapy
EP4511482A4 (en) * 2022-04-19 2026-03-18 Shanghai Vitalgen Biopharma Co Ltd Recombinant AAV vectors for the treatment of neurodegenerative disorders
WO2023212521A2 (en) * 2022-04-25 2023-11-02 Myrtelle Inc. Treatments of disorders of myelin
EP4514949A1 (en) 2022-04-25 2025-03-05 Hadasit Medical Research Services&Development Ltd. Methods and compositions for treating vision loss
US20250353883A1 (en) 2022-05-06 2025-11-20 Novartis Ag Novel recombinant aav vp2 fusion polypeptides
WO2023240220A1 (en) * 2022-06-09 2023-12-14 The University Of North Carolina At Chapel Hill Aav-sgsh vectors for treatment of mucopolysaccharidosis iiia
WO2023240236A1 (en) 2022-06-10 2023-12-14 Voyager Therapeutics, Inc. Compositions and methods for the treatment of spinal muscular atrophy related disorders
EP4303226A1 (en) 2022-07-08 2024-01-10 Ospedale San Raffaele S.r.l. Transgene cassettes and epigenetic silencers for the treatment of disorders
EP4551253A1 (en) 2022-07-08 2025-05-14 Ospedale San Raffaele S.r.l. Transgene cassettes
JP2025524636A (ja) * 2022-07-12 2025-07-30 リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル アデノ随伴ウイルス遺伝子療法産物及び方法
AU2023427408A1 (en) 2023-02-02 2025-09-04 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
EP4698206A1 (fr) 2023-04-21 2026-02-25 PulseSight Therapeutics Compositions à base de décorine pour la réparation et la régéneration de l'épithélium pigmentaire de la rétine
GB202306952D0 (en) * 2023-05-11 2023-06-28 Neurochase Innovations Ltd Method
TW202508610A (zh) 2023-08-23 2025-03-01 大陸商上海瑞宏迪醫藥有限公司 醫藥組成物及其用途
US20250197862A1 (en) 2023-12-15 2025-06-19 Genzyme Corporation ARTIFICIAL microRNAs TARGETING TAU
TW202547561A (zh) 2024-01-25 2025-12-16 美商健臻公司 基於ｆｕｓ向大腦遞送病毒顆粒之方法
WO2025160429A1 (en) 2024-01-26 2025-07-31 Genzyme Corporation Artificial micrornas targeting snca
WO2025160434A1 (en) 2024-01-26 2025-07-31 Genzyme Corporation Artificial micrornas targeting huntington's disease
CN120829932A (zh) * 2024-04-18 2025-10-24 临港国家实验室 治疗眼部疾病的药物组合物及方法

Family Cites Families (36)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4692147A (en) 1980-04-02 1987-09-08 Medtronic, Inc. Drug administration device
US5735815A (en) 1993-07-26 1998-04-07 Sentinel Medical, Inc. Method of using fluid jet surgical cutting tool
EP0931158A1 (en) 1996-09-06 1999-07-28 The Trustees Of The University Of Pennsylvania An inducible method for production of recombinant adeno-associated viruses utilizing t7 polymerase
WO1998048009A2 (en) 1997-04-21 1998-10-29 University Of Florida Materials and methods for ribozyme treatment of retinal diseases
US6042579A (en) 1997-04-30 2000-03-28 Medtronic, Inc. Techniques for treating neurodegenerative disorders by infusion of nerve growth factors into the brain
US6989264B2 (en) 1997-09-05 2006-01-24 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
WO1999061066A2 (en) 1998-05-27 1999-12-02 Avigen, Inc. Convection-enhanced delivery of aav vectors
CA2379166C (en) 1999-08-09 2013-03-26 Targeted Genetics Corporation Enhancement of expression of a single-stranded, heterologous nucleotide sequence from recombinant viral vectors by designing the sequence such that it forms instrastrand base pairs
ATE318923T1 (de) 2000-06-01 2006-03-15 Univ North Carolina Doppelsträngige parvovirus-vektoren
US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
GB2389791B (en) 2002-04-30 2006-12-13 Steven Gill Implantable drug delivery pump
US20060093589A1 (en) * 2004-02-19 2006-05-04 Warrington Kenneth H Vp2-modified raav vector compositions and uses therefor
SI1496944T1 (sl) * 2002-05-01 2009-02-28 Univ Florida Izboljšani sistemi ekspresije RAAV za genetsko modifikacijo specifičnih kapsidnih proteinov
CA2500523A1 (en) * 2003-05-01 2004-11-18 University Of Florida Research Foundation, Inc. Vp2-modified raav vector compositions and uses therefor
US7261544B2 (en) 2003-05-21 2007-08-28 Genzyme Corporation Methods for producing preparations of recombinant AAV virions substantially free of empty capsids
EP1486567A1 (en) 2003-06-11 2004-12-15 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Improved adeno-associated virus (AAV) vector for gene therapy
SI1633772T1 (sl) * 2003-06-19 2016-06-30 Genzyme Corporation AAV virioni z zmanjšano imunoreaktivnostjo in uporaba za ta namen
MX2007003850A (es) 2004-10-05 2007-11-21 Genzyme Corp Canula escalonada.
ES2525067T3 (es) * 2005-04-07 2014-12-17 The Trustees Of The University Of Pennsylvania Método de incremento de la función de un vector de AAV
EP1928557B1 (en) 2005-08-23 2018-06-06 The Regents of The University of California Reflux resistant cannula and system for chronic delivery of therapeutic agents using convection-enhanced delivery
US7867484B2 (en) * 2006-01-27 2011-01-11 University Of North Carolina At Chapel Hill Heparin and heparan sulfate binding chimeric vectors
DK2019683T4 (da) 2006-04-25 2022-08-29 Univ California Indgivelse af vækstfaktorer til behandling af CNS-lidelser
US20070259031A1 (en) 2006-04-26 2007-11-08 The Regents Of The University Of California Compositions and methods for convection enhanced delivery of high molecular weight neurotherapeutics
WO2008027084A2 (en) 2006-04-28 2008-03-06 The Trustees Of The University Of Pennsylvania Modified aav vectors having reduced capsid immunogenicity and use thereof
WO2008144585A1 (en) 2007-05-17 2008-11-27 Medgenesis Therapeutix Inc. Convection-enhanced delivery catheter with removable stiffening member and method for using same
US20100081707A1 (en) 2008-02-21 2010-04-01 Ali Robin R Devices and methods for delivering polynucleotides into retinal cells of the macula and fovea
JP2012516357A (ja) 2009-01-29 2012-07-19 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 神経学的障害を治療するための皮質全体に亘る高レベルの治療薬の分布方法
WO2010093784A2 (en) * 2009-02-11 2010-08-19 The University Of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
KR20180000341A (ko) 2009-06-16 2018-01-02 젠자임 코포레이션 재조합 ａａｖ 벡터에 대한 개선된 정제 방법
EP2561073B1 (en) 2010-04-23 2016-08-24 University of Massachusetts Cns targeting aav vectors and methods of use thereof
CN105755044A (zh) * 2011-04-22 2016-07-13 加利福尼亚大学董事会 具有变异衣壳的腺相关病毒病毒体及其使用方法
TWI698240B (zh) * 2012-05-15 2020-07-11 澳大利亞商艾佛蘭屈澳洲私營有限公司 使用腺相關病毒(aav)sflt-1治療老年性黃斑部退化(amd)
WO2015012501A1 (ko) 2013-07-25 2015-01-29 주식회사 스마트캐스터 비공기압 바퀴
GB201403684D0 (en) * 2014-03-03 2014-04-16 King S College London Vector
LT3137497T (lt) 2014-05-02 2021-07-26 Genzyme Corporation Aav vektoriai, skirti tinklainės ir cns genų terapijai

Also Published As

Publication number Publication date
IL248102B (en) 2022-07-01
EA034355B1 (ru) 2020-01-30
LT3137497T (lt) 2021-07-26
TWI686476B (zh) 2020-03-01
UY36106A (es) 2015-11-30
JP2023011812A (ja) 2023-01-24
IL292951A (en) 2022-07-01
PL3137497T4 (pl) 2023-04-11
SG10201809739QA (en) 2018-12-28
JP2020164540A (ja) 2020-10-08
AU2015252797C1 (en) 2021-04-22
DOP2016000280A (es) 2016-11-30
JP6741591B2 (ja) 2020-08-19
KR20250141850A (ko) 2025-09-29
RS62078B1 (sr) 2021-07-30
JP7174011B2 (ja) 2022-11-17
EP4600255A2 (en) 2025-08-13
KR20160147974A (ko) 2016-12-23
SI3137497T2 (sl) 2025-07-31
ES2879636T5 (en) 2025-06-13
DK3137497T4 (da) 2025-05-26
AU2021200242B2 (en) 2023-09-21
BR112016025263A2 (pt) 2018-02-20
CR20160555A (es) 2017-01-27
PE20170260A1 (es) 2017-04-12
WO2015168666A3 (en) 2015-12-23
CN106661591B (zh) 2021-10-29
CL2018000170A1 (es) 2018-06-29
TW201625792A (zh) 2016-07-16
JP7682142B2 (ja) 2025-05-23
EP4600255A3 (en) 2025-10-22
IL292951B1 (en) 2024-06-01
PT3137497T (pt) 2021-07-12
EP3137497B1 (en) 2021-04-07
PH12016502046A1 (en) 2017-02-06
US20170096683A1 (en) 2017-04-06
HUE054768T2 (hu) 2021-09-28
AU2021200242B9 (en) 2023-10-05
CN114231563A (zh) 2022-03-25
FI3137497T4 (fi) 2025-06-02
EP3913061A1 (en) 2021-11-24
JP2017518271A (ja) 2017-07-06
EP3137497B2 (en) 2025-03-05
MX2022012490A (es) 2022-10-27
AU2023285817A1 (en) 2024-01-18
EA201692206A1 (ru) 2017-03-31
ES2879636T3 (es) 2021-11-22
IL248102A0 (en) 2016-11-30
DK3137497T3 (da) 2021-07-12
CL2016002713A1 (es) 2017-08-11
MX2016014220A (es) 2017-02-06
US20210189430A1 (en) 2021-06-24
US10982228B2 (en) 2021-04-20
EP3137497A2 (en) 2017-03-08
PL3137497T3 (pl) 2021-10-25
HRP20211024T1 (hr) 2021-10-01
RS62078B2 (sr) 2025-06-30
SG11201607991WA (en) 2016-10-28
CA2946593A1 (en) 2015-11-05
CN114231563B (zh) 2024-08-16
JP2025065116A (ja) 2025-04-17
AU2015252797B2 (en) 2020-10-15
IL292951B2 (en) 2024-10-01
GT201600227A (es) 2018-01-22
SI3137497T1 (sl) 2021-08-31
US20250320522A1 (en) 2025-10-16
MY188987A (en) 2022-01-17
US12241078B2 (en) 2025-03-04
WO2015168666A2 (en) 2015-11-05
EP3913061B1 (en) 2026-04-01
KR102863726B1 (ko) 2025-09-25
SG10201912977RA (en) 2020-02-27
KR20240032156A (ko) 2024-03-08
AU2021200242A1 (en) 2021-03-18
JP7832294B2 (ja) 2026-03-17
HRP20211024T4 (hr) 2025-06-20
AU2015252797A1 (en) 2016-11-17
CN106661591A (zh) 2017-05-10
ZA201606763B (en) 2023-05-31

Similar Documents

Publication Publication Date Title
ZA201606763B (en) Aav vectors for retinal and cns gene therapy
IL251468A0 (en) aav-based gene therapy
ZA201802462B (en) Gene therapy
PT3612624T (pt) Terapia génica
GB201407322D0 (en) Gene therapy
LT3612237T (lt) Genų terapija
SMT202000453T1 (it) Terapia genica a base di aav per la sclerosi multipla
GB201707212D0 (en) Gene therapy for ciliopathies
GB201701968D0 (en) Gene therapy
IL275613A (en) Gene therapy for eosinophilic disorders
PL3285871T3 (pl) Wektory oraz sposoby terapii regeneracyjnej
GB201513151D0 (en) Functional gene replacement therapy
HK1234076A1 (en) Aav vectors for retinal and cns gene therapy
GB201616821D0 (en) Gene therapy
GB201612104D0 (en) Gene therapy
GB201612105D0 (en) Gene therapy
GB201704634D0 (en) Gene therapy
HK1244501A1 (en) Aav-based gene therapy