SG11201607991WA - Aav vectors for retinal and cns gene therapy - Google Patents

Aav vectors for retinal and cns gene therapy

Info

Publication number
SG11201607991WA
SG11201607991WA SG11201607991WA SG11201607991WA SG11201607991WA SG 11201607991W A SG11201607991W A SG 11201607991WA SG 11201607991W A SG11201607991W A SG 11201607991WA SG 11201607991W A SG11201607991W A SG 11201607991WA SG 11201607991W A SG11201607991W A SG 11201607991WA
Authority
SG
Singapore
Prior art keywords
retinal
gene therapy
aav vectors
cns gene
cns
Prior art date
Application number
SG11201607991WA
Other languages
English (en)
Inventor
Abraham Scaria
Jennifer Sullivan
Lisa M Stanek
Original Assignee
Genzyme Corp
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
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=SG11201607991W(A) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Genzyme Corp filed Critical Genzyme Corp
Publication of SG11201607991WA publication Critical patent/SG11201607991WA/en

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 N.A.
    • 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
SG11201607991WA 2014-05-02 2015-05-02 Aav vectors for retinal and cns gene therapy SG11201607991WA (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201461988131P 2014-05-02 2014-05-02
US201562114575P 2015-02-10 2015-02-10
PCT/US2015/028966 WO2015168666A2 (fr) 2014-05-02 2015-05-02 Vecteurs aav pour thérapie génique de la rétine et du snc

Publications (1)

Publication Number Publication Date
SG11201607991WA true SG11201607991WA (en) 2016-10-28

Family

ID=53189202

Family Applications (3)

Application Number Title Priority Date Filing Date
SG10201912977RA SG10201912977RA (en) 2014-05-02 2015-05-02 Aav vectors for retinal and cns gene therapy
SG11201607991WA SG11201607991WA (en) 2014-05-02 2015-05-02 Aav vectors for retinal and cns gene therapy
SG10201809739QA SG10201809739QA (en) 2014-05-02 2015-05-02 Aav vectors for retinal and cns gene therapy

Family Applications Before (1)

Application Number Title Priority Date Filing Date
SG10201912977RA SG10201912977RA (en) 2014-05-02 2015-05-02 Aav vectors for retinal and cns gene therapy

Family Applications After (1)

Application Number Title Priority Date Filing Date
SG10201809739QA SG10201809739QA (en) 2014-05-02 2015-05-02 Aav vectors for retinal and cns gene therapy

Country Status (32)

Country Link
US (2) US10982228B2 (fr)
EP (2) EP3137497B1 (fr)
JP (3) JP6741591B2 (fr)
KR (2) KR20160147974A (fr)
CN (2) CN106661591B (fr)
AU (3) AU2015252797C1 (fr)
BR (1) BR112016025263A2 (fr)
CA (1) CA2946593A1 (fr)
CL (2) CL2016002713A1 (fr)
CR (1) CR20160555A (fr)
DK (1) DK3137497T3 (fr)
DO (1) DOP2016000280A (fr)
EA (1) EA034355B1 (fr)
ES (1) ES2879636T3 (fr)
GT (1) GT201600227A (fr)
HR (1) HRP20211024T1 (fr)
HU (1) HUE054768T2 (fr)
IL (2) IL248102B (fr)
LT (1) LT3137497T (fr)
MX (2) MX2016014220A (fr)
MY (1) MY188987A (fr)
PE (1) PE20170260A1 (fr)
PH (1) PH12016502046A1 (fr)
PL (1) PL3137497T4 (fr)
PT (1) PT3137497T (fr)
RS (1) RS62078B1 (fr)
SG (3) SG10201912977RA (fr)
SI (1) SI3137497T1 (fr)
TW (1) TWI686476B (fr)
UY (1) UY36106A (fr)
WO (1) WO2015168666A2 (fr)
ZA (1) ZA201606763B (fr)

Families Citing this family (144)

* 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
WO2010129021A1 (fr) 2009-05-02 2010-11-11 Genzyme Corporation Thérapie génique pour les maladies neurodégénératives
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
WO2011133874A1 (fr) 2010-04-23 2011-10-27 University Of Massachusetts Constructions d'expression à cistrons multiples
WO2011133901A2 (fr) 2010-04-23 2011-10-27 University Of Massachusetts Traitement à base d'aav pour des troubles liés au cholestérol
DK2826860T3 (en) 2010-04-23 2018-12-03 Univ Massachusetts CNS targeting AAV vectors and methods for their use
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 (fr) 2011-04-21 2018-05-09 University of Massachusetts Compositions à base de raav et procédés pour traiter des déficiences en anti-trypsine alpha-1
PL3254703T3 (pl) 2011-04-22 2020-10-05 The Regents Of The University Of California Wiriony wirusa towarzyszącego adenowirusom z różnymi kapsydami i sposoby ich zastosowania
CA2907799A1 (fr) 2013-05-31 2014-12-04 The Regents Of The University Of California Variants de virus adeno-associes et leurs methodes d'utilisation
WO2015038958A1 (fr) 2013-09-13 2015-03-19 California Institute Of Technology Récupération sélective
US10072251B2 (en) 2014-02-19 2018-09-11 University Of Massachusetts Recombinant AAVS having useful transcytosis properties
CA2942776C (fr) 2014-03-17 2023-01-24 Adverum Biotechnologies, Inc. Cassettes polynucleotidiques et vecteurs d'expression pour l'expression d'un gene dans des cones retiniens a l'aide d'un promoteur de m-opsine tronque
EP3750907A3 (fr) 2014-03-18 2021-04-28 University of Massachusetts Compositions à base de raav et procédés pour traiter la sclérose latérale amyotrophique
US10975391B2 (en) 2014-04-25 2021-04-13 University Of Massachusetts Recombinant AAV vectors useful for reducing immunity against transgene products
MY183472A (en) * 2014-05-14 2021-02-19 Esteve Labor Dr Adenoassociated virus vectors for the treatment of lysosomal storage disorders
WO2015187825A2 (fr) 2014-06-03 2015-12-10 University Of Massachusetts Compositions et méthodes de modulation de l'expression de la dysferline
JP6842410B2 (ja) 2014-10-03 2021-03-17 ユニバーシティ オブ マサチューセッツ 新規の高効率ライブラリーにより同定されるａａｖベクター
WO2016054554A1 (fr) 2014-10-03 2016-04-07 University Of Massachusetts Virus adéno-associés greffés au moyen de peptides de ciblage hétérologues
CN107073051B (zh) 2014-10-21 2021-08-24 马萨诸塞大学 重组aav变体及其用途
SG10201907399RA (en) * 2015-02-10 2019-09-27 Genzyme Corp Enhanced delivery of viral particles to the striatum and cortex
AU2016219396B2 (en) 2015-02-10 2022-03-17 Genzyme Corporation Variant RNAi
EP3256170B1 (fr) 2015-02-13 2020-09-23 University of Massachusetts Compositions et procédés pour l'administration transitoire de nucléases
KR20170137730A (ko) 2015-03-02 2017-12-13 애드베룸 바이오테크놀로지스, 인코포레이티드 망막 추상체에 폴리뉴클레오타이드의 유리체 내 전달을 위한 조성물 및 방법
EP3719134B1 (fr) * 2015-03-11 2023-08-23 The United States of America, as represented by The Secretary, Department of Health and Human Services Vecteurs de rp2 pour le traitement de la rétinite pigmentaire liée au chromosome x
AU2016235163B2 (en) 2015-03-24 2022-03-24 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
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
PL3356390T3 (pl) 2015-09-28 2021-07-05 The University Of North Carolina At Chapel Hill Sposoby i kompozycje dla wektorów wirusowych unikających przeciwciał
DK3364997T3 (da) 2015-10-22 2024-04-22 Univ Massachusetts Aspartoacylase genterapi til behandling af canavans sygdom
CA3002980A1 (fr) 2015-10-22 2017-04-27 University Of Massachusetts Vecteurs du serotype de virus adeno-associes ciblant la prostate
US20180230489A1 (en) 2015-10-28 2018-08-16 Voyager Therapeutics, Inc. Regulatable expression using adeno-associated virus (aav)
WO2017100671A1 (fr) * 2015-12-11 2017-06-15 California Institute Of Technology Peptides de ciblage pour diriger des virus adéno-associés (aav)
RU2742612C2 (ru) * 2015-12-15 2021-02-09 Джензим Корпорейшн Векторы на основе аденоассоциированного вируса для лечения муколипидоза типа ii
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 (fr) 2016-02-12 2017-08-17 University Of Massachusetts Agents therapeutiques a base de micro-arn anti-angiogeniques pour l'inhibition de la neovascularisation corneenne
US20190071681A1 (en) * 2016-02-26 2019-03-07 University Of Florida Research Foundation, Incorporated Aav heparin mutants that display significantly improved eye and brain transduction
WO2017176929A1 (fr) 2016-04-05 2017-10-12 University Of Massachusetts Compositions et procédés pour l'inhibition sélective de l'expression d'une protéine de type grainhead
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
RU2764587C2 (ru) * 2016-05-18 2022-01-18 Вояджер Терапьютикс, Инк. Способы и композиции для лечения хореи гентингтона
US11882815B2 (en) 2016-06-15 2024-01-30 University Of Massachusetts Recombinant adeno-associated viruses for delivering gene editing molecules to embryonic cells
KR20230062878A (ko) * 2016-07-08 2023-05-09 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Rdh12가 연루된 장애 및 질환의 치료를 위한 방법 및 조성물
US11458211B2 (en) 2016-07-12 2022-10-04 The University Of Manchester Gene therapy
MX2019001276A (es) 2016-07-29 2019-06-13 Univ California Viriones de virus adenoasociados con cápside variante y métodos para su uso.
EP3500676A4 (fr) * 2016-08-16 2020-07-01 The University of North Carolina at Chapel Hill Procédés et compositions servant au transfert de gènes ciblés
US20200340012A1 (en) * 2016-08-18 2020-10-29 The Regents Of The University Of California Crispr-cas genome engineering via a modular aav delivery system
EP3500279A4 (fr) * 2016-08-19 2020-04-22 Calimmune, Inc. Procédés et compositions pour le traitement de pathologies canins à l'aide d'un virus adéno-associé auto-complémentaire recombinant.
WO2018035451A1 (fr) * 2016-08-19 2018-02-22 Calimmune, Inc. Méthodes et compositions pour le traitement d'affections à l'aide d'un virus adéno-associé recombinant auto-complémentaire
US10457940B2 (en) 2016-09-22 2019-10-29 University Of Massachusetts AAV treatment of Huntington's disease
CA3038292A1 (fr) 2016-09-28 2018-04-05 Cohbar, Inc. Peptides lies a un mots-c therapeutique
EP3518985A4 (fr) * 2016-09-29 2020-08-05 University of Florida Research Foundation, Incorporated Variantes aavrh.10 à capacités d'échappement d'anticorps hôtes et propriétés de ciblage de tissu modifiées
US11578340B2 (en) 2016-10-13 2023-02-14 University Of Massachusetts AAV capsid designs
WO2018075798A1 (fr) 2016-10-19 2018-04-26 Adverum Biotechnologies, Inc. Capsides d'aav modifiées et leurs utilisations
WO2018106956A2 (fr) 2016-12-07 2018-06-14 University Of Florida Research Foundation, Incorporated Adnc d'il-1ra
WO2018145009A1 (fr) * 2017-02-06 2018-08-09 University Of Tennessee Research Foundation Méthodes et compositions à base d'adnzyme pour le traitement de la maladie de huntington
WO2018156892A1 (fr) 2017-02-23 2018-08-30 Adrx, Inc. Inhibiteurs peptidiques de l'agrégation du facteur de transcription
JOP20190200A1 (ar) 2017-02-28 2019-08-27 Univ Pennsylvania تركيبات نافعة في معالجة ضمور العضل النخاعي
WO2018191450A2 (fr) 2017-04-14 2018-10-18 National Taiwan University Hospital Thérapie génique pour une carence en aadc
WO2018200419A1 (fr) * 2017-04-23 2018-11-01 The Trustees Of The University Of Pennsylvania Vecteurs viraux comprenant des capsides d'aav modifié et compositions les contenant
SG11201909868YA (en) * 2017-05-05 2019-11-28 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease
WO2018208972A1 (fr) 2017-05-09 2018-11-15 University Of Massachusetts Méthodes de traitement de la sclérose latérale amyotrophique (sla)
JP7330899B2 (ja) 2017-05-10 2023-08-22 マサチューセッツ アイ アンド イヤー インファーマリー ウイルスのアセンブリ活性化タンパク質（ａａｐ）依存性を改変する方法および組成物
US20230137562A1 (en) 2017-06-07 2023-05-04 Adrx, Inc. Tau aggregation inhibitors
BR112019019015A2 (pt) * 2017-06-30 2020-04-14 Univ California vírions de vírus adeno-associado com capsídeos variantes e seus métodos de uso
KR20200044793A (ko) 2017-08-03 2020-04-29 보이저 테라퓨틱스, 인크. Aav의 전달을 위한 조성물 및 방법
EP3676373A4 (fr) 2017-08-28 2021-06-09 The Regents of The University of California Variants de capside de virus adéno-associé et leurs procédés d'utilisation
WO2019060686A1 (fr) 2017-09-22 2019-03-28 University Of Massachusetts Nouveaux vecteurs d'expression double de sod1 et utilisations associées
KR20200056428A (ko) 2017-09-22 2020-05-22 젠자임 코포레이션 변이체 RNAi
KR20200103634A (ko) * 2017-10-20 2020-09-02 인쎄름 (엥스띠뛰 나씨오날 드 라 쌍떼 에 드 라 흐쉐르슈 메디깔) 치료적으로 유효한 양의 재조합 aav9- 유도 벡터의 망막하 전달을 포함하는 대상체의 원추 광수용체 내 관심의 폴리뉴클레오티드를 발현하는 방법
US11464803B2 (en) 2017-11-14 2022-10-11 Arcellx, Inc. D-domain containing polypeptides and uses thereof
KR20200087176A (ko) 2017-11-14 2020-07-20 아르셀엑스, 인크. 다기능성 면역 세포 요법
AU2018369975B2 (en) * 2017-11-15 2022-05-19 Friedrich Miescher Institute For Biomedical Research Primate retinal pigment epithelium cell-specific promoter
KR20200088385A (ko) * 2017-11-15 2020-07-22 더 리젠츠 오브 더 유니버시티 오브 미시간 Rdh12 암호화 영역을 포함하는 바이러스 벡터 및 망막 이영양증을 치료하는 방법
MA50877A (fr) 2017-11-21 2020-09-30 Bayer Healthcare Llc Matériaux et méthodes pour le traitement de la rétinite pigmentaire autosomique dominante
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
JP7406677B2 (ja) 2018-04-03 2023-12-28 ギンコ バイオワークス インコーポレイテッド 抗体を回避するウイルスベクター
US20210230632A1 (en) 2018-05-15 2021-07-29 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
SG11202011296VA (en) 2018-05-15 2020-12-30 Voyager Therapeutics Inc Compositions and methods for the treatment of parkinson's disease
JP2021522840A (ja) * 2018-05-15 2021-09-02 プレジデント アンド フェローズ オブ ハーバード カレッジ 改善された遺伝子送達特性を示すウイルスベクター
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
EP3830265A1 (fr) 2018-08-03 2021-06-09 Genzyme Corporation Variante d'arni contre l'alpha-synucléine
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
US11149256B2 (en) 2018-09-26 2021-10-19 California Institute Of Technology Adeno-associated virus compositions for targeted gene therapy
KR20210068068A (ko) 2018-09-28 2021-06-08 보이저 테라퓨틱스, 인크. 조작된 프로모터를 갖는 프라탁신 발현 구축물 및 그의 사용 방법
JP2022504740A (ja) * 2018-10-12 2022-01-13 ジェンザイム・コーポレーション 肝臓に向けられた遺伝子補充療法によって重度のｐｋｕを処置するための改善されたヒトｐａｈの生成
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
WO2020099956A1 (fr) * 2018-11-13 2020-05-22 Indian Institute Of Technology Kanpur Procédé de production d'une pluralité de vecteurs aav modifiés sur site cible de sumoylation et produit associé
AU2020208467A1 (en) 2019-01-18 2021-08-05 Voyager Therapeutics, Inc. Methods and systems for producing AAV particles
AU2020212026A1 (en) * 2019-01-23 2021-07-22 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
JP2022522776A (ja) * 2019-03-04 2022-04-20 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
WO2020227515A1 (fr) 2019-05-07 2020-11-12 Voyager Therapeutics, Inc. Compositions et méthodes d'augmentation vectorisée de la destruction, de l'expression et/ou de la régulation de protéines
US20220249700A1 (en) * 2019-05-13 2022-08-11 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Polymer-based implant for retinal therapy and methods of making and using the same
US20220226507A1 (en) * 2019-05-17 2022-07-21 Research Institute At Nationwide Children's Hospital Optimized gene therapy targeting retinal cells
CA3142932A1 (fr) * 2019-06-10 2020-12-17 Homology Medicines, Inc. Compositions de virus adeno-associes pour transfert de gene arsa et leurs procedes d'utilisation
WO2020264438A2 (fr) * 2019-06-27 2020-12-30 University Of Florida Research Foundation, Incorporated Amélioration de transduction médiée par aav de tissus oculaires avec de l'acide hyaluronique
US11446084B2 (en) 2019-07-12 2022-09-20 Neuralink Corp. Laser drilling of pia mater
BR112022000724A2 (pt) * 2019-07-15 2022-06-14 Meiragtx Uk Ii Ltd Proteínas de capsídeo de aav modificado para tratamento de doença 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 (fr) * 2019-08-23 2021-03-04 Duke University Compositions et procédés pour le traitement de douleurs et de démangeaisons pathologiques
EP4025258A4 (fr) * 2019-09-03 2023-09-06 University of Cincinnati Méthodes et compositions pour le traitement de la sclérose latérale amyotrophique
WO2021046155A1 (fr) 2019-09-03 2021-03-11 Voyager Therapeutics, Inc. Édition vectorisée d'acides nucléiques pour corriger des mutations manifestes
CA3157700A1 (fr) 2019-10-17 2021-04-22 Stridebio, Inc. Vecteurs de virus adeno-associes pour le traitement de la maladie de niemann-pick de type c
IL292372A (en) * 2019-10-23 2022-06-01 Univ Pennsylvania Preparations for the reduction of transgene expression specifically drg
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
KR20220133941A (ko) 2020-01-29 2022-10-05 젠자임 코포레이션 안과 유전자 치료를 위한 변형 아데노-관련 바이러스 캡시드 단백질 및 이의 사용 방법
WO2021159069A2 (fr) * 2020-02-07 2021-08-12 The Trustees Of Columbia University In The City Of New York Reprogrammation du métabolome pour retarder l'apparition d'une neurodégénérescence ou pour la traiter
AU2021224551A1 (en) * 2020-02-18 2022-07-28 The University Of North Carolina At Chapel Hill AAV capsid-promoter interactions and cell selective gene expression
EP4110913A2 (fr) * 2020-02-28 2023-01-04 University of Massachusetts Oligonucléotides pour la modulation de prnp
IL296262A (en) * 2020-03-11 2022-11-01 Massachusetts Eye & Ear Infirmary Gene therapy for nmnat1-related retinal degeneration
MX2022012279A (es) * 2020-03-31 2023-02-23 Univ Massachusetts Variantes de capsides de aav y usos de los mismos.
BR112022020753A2 (pt) 2020-04-15 2022-12-20 Voyager Therapeutics Inc Compostos de ligação a tau
WO2021230987A1 (fr) 2020-05-13 2021-11-18 Voyager Therapeutics, Inc. Redirection de tropisme de capsides de vaa
WO2021247995A2 (fr) 2020-06-04 2021-12-09 Voyager Therapeutics, Inc. Compositions et méthodes de traitement de la douleur neuropathique
JP2023529503A (ja) * 2020-06-13 2023-07-10 オクロジェネクス インコーポレイテッド 網膜症のためのａａｖ媒介性遺伝子導入
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 (fr) 2020-07-23 2022-01-27 Ucl Business Ltd Vecteur de thérapie génique pour eef1a2 et ses utilisations
WO2022026410A2 (fr) 2020-07-27 2022-02-03 Voyager Therapeutics, Inc Compositions et méthodes pour le traitement de la maladie de niemann-pick de type c1
US20230227802A1 (en) 2020-07-27 2023-07-20 Voyager Therapeutics, Inc. Compositions and methods for the treatment of neurological disorders related to glucosylceramidase beta deficiency
JP2023536618A (ja) 2020-08-05 2023-08-28 スペースクラフト セブン リミテッド ライアビリティ カンパニー Ｃｓｒｐ３（システイン及びグリシンリッチタンパク質３）遺伝子治療
AU2021320902A1 (en) 2020-08-07 2023-04-06 Spacecraft Seven, Llc Plakophilin-2 (PKP2) gene therapy using AAV vector
US20230330267A1 (en) * 2020-09-04 2023-10-19 Ohio State Innovation Foundation Novel engineered capsid serotype of recombinant adeno-associated viral vector with enhanced transduction efficiency and widespread distribution in the brain
US20230392166A1 (en) * 2020-10-21 2023-12-07 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
AU2022224667A1 (en) * 2021-02-22 2023-09-07 The Regents Of The University Of Colorado, A Body Corporate Viral vector-based gene therapy for ocular conditions
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 (fr) * 2021-04-26 2022-11-03 Brendan BEAHM Compositions et methodes de traitement d'une deficience de ngyl1
AU2022340595A1 (en) * 2021-09-06 2024-04-18 Huidagene Therapeutics (Singapore) Pte. Ltd. Treatment of rpe65-associated eye diseases and disorders
WO2023091949A2 (fr) 2021-11-17 2023-05-25 Voyager Therapeutics, Inc. Compositions et méthodes de traitement de troubles neurologiques liés à un déficit en bêta glucosylcéramidase
WO2023183623A1 (fr) * 2022-03-25 2023-09-28 Regenxbio Inc. Thérapie génique du virus adéno-associé à facteur de nécrose tumorale alpha dominant-négatif
WO2023212521A2 (fr) * 2022-04-25 2023-11-02 Myrtelle Inc. Traitements de troubles de la myéline
WO2023211857A1 (fr) 2022-04-25 2023-11-02 Lineage Cell Therapeutics, Inc. Procédés et compositions pour le traitement de la perte de vision
WO2023214346A1 (fr) 2022-05-06 2023-11-09 Novartis Ag Nouveaux polypeptides de fusion vp2 d'aav recombinants
WO2023240220A1 (fr) * 2022-06-09 2023-12-14 The University Of North Carolina At Chapel Hill Vecteurs aav-sgsh pour le traitement de la mucopolysaccharidose iiia
WO2023240236A1 (fr) 2022-06-10 2023-12-14 Voyager Therapeutics, Inc. Compositions et procédés pour le traitement de troubles liés à l'amyotrophie spinale
EP4303226A1 (fr) 2022-07-08 2024-01-10 Ospedale San Raffaele S.r.l. Cassettes transgéniques et silencieux épigénétiques pour le traitement de troubles
WO2024008950A1 (fr) 2022-07-08 2024-01-11 Ospedale San Raffaele S.R.L. Cassettes transgéniques
WO2024015794A2 (fr) * 2022-07-12 2024-01-18 The Research Institute At Nationwide Children's Hospital Produits et procédés de thérapie génique par virus adéno-associé

Family Cites Families (31)

* 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
WO1998010088A1 (fr) 1996-09-06 1998-03-12 Trustees Of The University Of Pennsylvania Procede inductible de production de virus adeno-associes recombines au moyen de la polymerase t7
US6225291B1 (en) 1997-04-21 2001-05-01 University Of Florida Rod opsin mRNA-specific ribozyme compositions and methods for the 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
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6995006B2 (en) 1997-09-05 2006-02-07 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
ATE316576T1 (de) 1998-05-27 2006-02-15 Avigen Inc Konvektion-erhöhte verabreichung aadc-kodierende aav vektoren
AU781958C (en) 1999-08-09 2006-03-30 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 intrastrand base pairs
ES2256265T3 (es) 2000-06-01 2006-07-16 University Of North Carolina At Chapel Hill Vectores de parvovirus duplicados.
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
ATE405295T1 (de) * 2002-05-01 2008-09-15 Univ Florida Verbesserte raav-expressionssysteme für die genetische modifikation spezifischer capsidproteine
PT1625210E (pt) 2003-05-21 2011-03-15 Genzyme Corp Métodos para produzir preparações de vírions de aav recombinantes substancialmente livres de capsídeos vazios
EP1486567A1 (fr) 2003-06-11 2004-12-15 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Vecteur derivé d'un virus associé aux adenovirus pour la thérapie génique
DK3235827T3 (da) * 2003-06-19 2021-04-19 Genzyme Corp Aav-virioner med reduceret immunreaktivitet og anvendelser deraf
CN102626336A (zh) 2004-10-05 2012-08-08 建新公司 台阶式插管
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
US9089667B2 (en) 2005-08-23 2015-07-28 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
ES2618787T5 (es) 2006-04-25 2022-10-21 Univ California Administración de factores de crecimiento para el tratamiento de trastornos del SNC
WO2007127839A2 (fr) 2006-04-26 2007-11-08 The Regents Of The University Of California Compositions et méthodes pour l'administration de neurothérapeutiques de haut poids moléculaire améliorée par convection
CN101495624A (zh) * 2006-04-28 2009-07-29 宾夕法尼亚州立大学托管会 衣壳免疫原性降低的经修饰aav载体及其用途
ES2557482T3 (es) 2007-05-17 2016-01-26 Medgenesis Therapeutix Inc. Catéter de entrega mejorada por convección con un miembro de rigidización eliminable
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
WO2010088560A1 (fr) 2009-01-29 2010-08-05 University Of California, San Francisco Procédés de délivrance de taux élevés d'agents thérapeutiques dans la totalité du cortex pour le traitement des troubles neurologiques
US8889641B2 (en) * 2009-02-11 2014-11-18 The University Of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
HUE028341T2 (en) 2009-06-16 2016-12-28 Genzyme Corp Improved methods for purifying recombinant AAV vectors
DK2826860T3 (en) 2010-04-23 2018-12-03 Univ Massachusetts CNS targeting AAV vectors and methods for their use
PL3254703T3 (pl) * 2011-04-22 2020-10-05 The Regents Of The University Of California Wiriony wirusa towarzyszącego adenowirusom z różnymi kapsydami i sposoby ich zastosowania
TWI775096B (zh) * 2012-05-15 2022-08-21 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)

Also Published As

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

Similar Documents

Publication Publication Date Title
ZA201606763B (en) Aav vectors for retinal and cns gene therapy
IL251468A0 (en) aav-based gene therapy
HK1257801A1 (zh) 用於基因治療的腺伴隨病毒載體的鞘內給藥
IL284577A (en) Garden healing
SG11201703193XA (en) Treatment for depression and depressive disorders
HK1258518A1 (zh) 用於眼病的基因療法
GB201407322D0 (en) Gene therapy
PL3612237T3 (pl) Terapia genowa
SI3133923T1 (sl) Genska terapija za multiplo sklerozo na osnovi AAV
GB201707212D0 (en) Gene therapy for ciliopathies
HUE055902T2 (hu) Vektorok és eljárások regeneratív terápiára
IL275613A (en) Gene therapy for eosinophilic disorders
GB201701968D0 (en) Gene therapy
GB201513151D0 (en) Functional gene replacement therapy
IL263085A (en) Garden healing
GB201616821D0 (en) Gene therapy
GB201612104D0 (en) Gene therapy
GB201704634D0 (en) Gene therapy