ES2957840T3 - Tratamiento génico de la retinitis pigmentaria - Google Patents

Tratamiento génico de la retinitis pigmentaria Download PDF

Info

Publication number
ES2957840T3
ES2957840T3 ES19186929T ES19186929T ES2957840T3 ES 2957840 T3 ES2957840 T3 ES 2957840T3 ES 19186929 T ES19186929 T ES 19186929T ES 19186929 T ES19186929 T ES 19186929T ES 2957840 T3 ES2957840 T3 ES 2957840T3
Authority
ES
Spain
Prior art keywords
mir
rhodopsin
nucleic acid
aav
sequence
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
ES19186929T
Other languages
English (en)
Spanish (es)
Inventor
Catherine O'riordan
Matthew Adamowicz
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Genzyme Corp
Original Assignee
Genzyme Corp
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=52829343&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=ES2957840(T3) "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
Application granted granted Critical
Publication of ES2957840T3 publication Critical patent/ES2957840T3/es
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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
    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0048—Eye, e.g. artificial tears
    • 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
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—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
    • C12N2320/34—Allele or polymorphism specific uses
    • 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
    • C12N2330/00—Production
    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
    • C12N2330/51—Specially adapted 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
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Epidemiology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Virology (AREA)
  • Immunology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Dermatology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
ES19186929T 2014-03-21 2015-03-20 Tratamiento génico de la retinitis pigmentaria Active ES2957840T3 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201461969027P 2014-03-21 2014-03-21

Publications (1)

Publication Number Publication Date
ES2957840T3 true ES2957840T3 (es) 2024-01-26

Family

ID=52829343

Family Applications (2)

Application Number Title Priority Date Filing Date
ES19186929T Active ES2957840T3 (es) 2014-03-21 2015-03-20 Tratamiento génico de la retinitis pigmentaria
ES15716232T Active ES2760263T3 (es) 2014-03-21 2015-03-20 Tratamiento génico de la retinitis pigmentaria

Family Applications After (1)

Application Number Title Priority Date Filing Date
ES15716232T Active ES2760263T3 (es) 2014-03-21 2015-03-20 Tratamiento génico de la retinitis pigmentaria

Country Status (36)

Country Link
US (3) US10383953B2 (2)
EP (3) EP3119437B1 (2)
JP (3) JP6669664B2 (2)
KR (3) KR20230006039A (2)
CN (2) CN106456660B (2)
AR (1) AR099837A1 (2)
AU (3) AU2015230942B2 (2)
CA (2) CA2943185C (2)
CL (1) CL2016002333A1 (2)
CR (1) CR20160480A (2)
DK (2) DK3119437T3 (2)
DO (1) DOP2016000237A (2)
EA (1) EA201691891A1 (2)
EC (1) ECSP16083000A (2)
ES (2) ES2957840T3 (2)
FI (1) FI3628334T3 (2)
HR (2) HRP20192141T1 (2)
HU (2) HUE063460T2 (2)
IL (3) IL247543B (2)
LT (2) LT3119437T (2)
MA (1) MA39390B2 (2)
MX (2) MX376190B (2)
MY (2) MY203654A (2)
NZ (1) NZ724622A (2)
PE (1) PE20161252A1 (2)
PH (1) PH12016501684B1 (2)
PL (2) PL3628334T3 (2)
PT (2) PT3119437T (2)
RS (2) RS64611B1 (2)
SG (3) SG11201607005UA (2)
SI (2) SI3628334T1 (2)
TW (2) TWI706789B (2)
UA (1) UA120050C2 (2)
UY (1) UY36044A (2)
WO (1) WO2015143418A2 (2)
ZA (1) ZA201605924B (2)

Families Citing this family (29)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PL3628334T3 (pl) 2014-03-21 2023-12-18 Genzyme Corporation Terapia genowa w retinopatii barwnikowej
CR20170406A (es) 2015-02-10 2017-11-14 Genzyme Corp Arni variante
EP3383440B1 (en) 2015-12-03 2021-06-30 Friedrich Miescher Institute for Biomedical Research Synp162, a promoter for the specific expression of genes in rod photoreceptors
WO2017093934A1 (en) * 2015-12-03 2017-06-08 Friedrich Miescher Institute For Biomedical Research Synp160, a promoter for the specific expression of genes in rod photoreceptors
WO2017093566A1 (en) 2015-12-04 2017-06-08 Universite Pierre Et Marie Curie (Paris 6) Promoters and uses thereof
CN115896106A (zh) * 2016-03-01 2023-04-04 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aav载体
KR20190039703A (ko) * 2016-07-05 2019-04-15 더 존스 홉킨스 유니버시티 Crispr/cas9-기반 조성물 및 망막 변성을 치료하기 위한 방법
EP3600446A1 (en) * 2017-03-21 2020-02-05 Michalakis, Stylianos Gene therapy for the treatment of cngb1-linked retinitis pigmentosa
CN111356763B (zh) * 2017-09-22 2024-03-12 建新公司 变体RNAi
EP3687580A1 (en) * 2017-09-27 2020-08-05 Sigilon Therapeutics, Inc. Methods, compositions, and implantable elements comprising active cells
CN107961380B (zh) * 2017-11-23 2020-05-05 清华大学 试剂在制备药物中的用途、筛选药物的方法以及药物组合物
US12605486B2 (en) 2018-03-02 2026-04-21 Sigilon Therapeutics, Inc. Afibrotic compounds, devices, and uses thereof
EP3759154A1 (en) 2018-03-02 2021-01-06 Sigilon Therapeutics, Inc. Biocompatible hydrogel capsules and process for preparing same
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
BR112020020084A2 (pt) 2018-04-04 2021-01-05 Sigilon Therapeutics, Inc. Partícula, preparação de uma pluralidade de partículas, método de preparação de uma partícula, e, composição de partículas.
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
GB201817469D0 (en) * 2018-10-26 2018-12-12 Univ Oxford Innovation Ltd Gene therapy for retinal disease
CN111518813B (zh) * 2019-02-03 2023-04-28 武汉纽福斯生物科技有限公司 视紫红质的编码序列、其表达载体构建及其应用
CN113795279B (zh) * 2019-03-04 2024-12-31 宾夕法尼亚州大学信托人 靶向akt通路的神经保护性基因疗法
CN114206393A (zh) * 2019-03-21 2022-03-18 Ptc医疗公司 用于治疗天使综合征的载体和方法
EP4013414A4 (en) * 2019-08-15 2023-09-27 The Children's Hospital of Philadelphia COMBINED INTRON-DERIVED MIRNA AND TRANSGENE THERAPY FOR THE TREATMENT OF SCA1
CA3164335A1 (en) * 2020-01-10 2021-07-15 Fatih Ozsolak Viral vector for combination therapy
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
KR102396200B1 (ko) * 2020-07-24 2022-05-12 알지노믹스 주식회사 로돕신 전사체에 특이적인 트랜스-스플라이싱 라이보자임 및 이의 용도
US20230265455A1 (en) * 2020-07-29 2023-08-24 University Of Florida Research Foundation, Incorporated Improved aav-mediated x-linked retinoschisis therapies
EP4323390A1 (en) * 2021-04-16 2024-02-21 Regeneron Pharmaceuticals, Inc. Treatment of x-linked juvenile retinoschisis
TWI832531B (zh) * 2022-11-02 2024-02-11 慈濟學校財團法人慈濟大學 Rip1抑制劑或mlkl抑制劑用於治療或預防遺傳性視網膜失養症的用途
CN117625619B (zh) * 2023-12-05 2024-11-12 云舟生物科技(广州)股份有限公司 核酸分子及其作为特异启动子的应用
CN120230798A (zh) * 2023-12-29 2025-07-01 上海朗昇生物科技有限公司 用于视网膜基因递送的重组腺相关病毒载体及其应用

Family Cites Families (31)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5262529A (en) * 1990-01-24 1993-11-16 President And Fellows Of Harvard College Diagnosis of hereditary retinal degenerative diseases
US5985583A (en) * 1992-06-23 1999-11-16 Mount Sinai School Of Medicine Of The City University Of New York Cloning and expression of gonadotropin-releasing hormone receptor
US6566118B1 (en) 1997-09-05 2003-05-20 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US6989264B2 (en) 1997-09-05 2006-01-24 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant 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
MX359371B (es) 2001-11-13 2018-09-25 Univ Pennsylvania Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo.
EP1486567A1 (en) 2003-06-11 2004-12-15 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Improved adeno-associated virus (AAV) vector for gene therapy
CN1934260B (zh) * 2004-01-22 2013-03-13 株式会社载体研究所 利用含有巨细胞病毒增强子和鸡β-肌动蛋白启动子的杂合启动子制造负链RNA病毒载体的方法
US7765583B2 (en) 2005-02-28 2010-07-27 France Telecom System and method for managing virtual user domains
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
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
EP2140004B1 (en) 2007-04-12 2022-01-12 The Provost, Fellows, Foundation Scholars, & the other members of Board, of the College of the Holy & Undiv. Trinity of Queen Elizabeth near Dublin Genetic suppression and replacement
US20090214478A1 (en) * 2008-02-21 2009-08-27 Alberto Auricchio Method of treating ocular diseases by gene therapy
US9217155B2 (en) * 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
US8734809B2 (en) * 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
AU2010294197C9 (en) * 2009-09-10 2018-09-13 Velin-Pharma A/S Method for the preparation of micro-RNA and its therapeutic application
WO2011094198A1 (en) 2010-01-28 2011-08-04 The Children's Hospital Of Philadelphia Research Institute, Abramson Research Center A scalable manufacturing platform for viral vector purification and viral vectors so purified for use in gene therapy
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
WO2012057363A1 (ja) 2010-10-27 2012-05-03 学校法人自治医科大学 神経系細胞への遺伝子導入のためのアデノ随伴ウイルスビリオン
TR201908020T4 (tr) 2011-02-17 2019-06-21 Univ Pennsylvania Doku özgüllüğünü değiştirmek ve aav9-aracılı gen transferini geliştirmek için bileşimler ve yöntemler.
JP5884152B2 (ja) 2011-07-29 2016-03-15 シャープ株式会社 基地局、端末、通信システムおよび通信方法
ITRM20110685A1 (it) * 2011-12-23 2013-06-24 Internat Ct For Genetic En Gineering And Microrna per la rigenerazione cardiaca attraverso l induzione della proliferazione dei miociti cardiaci
MX367100B (es) * 2012-02-17 2019-08-05 The Children´S Hospital Of Philadelphia Composiciones del vector aav y metodos para su transferencia genetica a celulas, organos y tejidos.
WO2013151665A2 (en) * 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of proteins associated with human disease
US9127274B2 (en) * 2012-04-26 2015-09-08 Alnylam Pharmaceuticals, Inc. Serpinc1 iRNA compositions and methods of use thereof
AU2013266968B2 (en) 2012-05-25 2017-06-29 Emmanuelle CHARPENTIER Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription
EP2872183B1 (en) * 2012-07-11 2018-09-26 The Trustees Of The University Of Pennsylvania Aav-mediated gene therapy for rpgr x-linked retinal degeneration
US9512425B2 (en) * 2012-10-23 2016-12-06 Cornell University Inhibiting migration of cancer cells
KR20160065930A (ko) 2013-10-04 2016-06-09 머크 샤프 앤드 돔 코포레이션 글루코스-반응성 인슐린 접합체
PL3628334T3 (pl) 2014-03-21 2023-12-18 Genzyme Corporation Terapia genowa w retinopatii barwnikowej

Also Published As

Publication number Publication date
LT3628334T (lt) 2023-09-25
CN106456660B (zh) 2022-05-31
HRP20192141T1 (hr) 2020-02-21
RS59634B1 (sr) 2020-01-31
AU2021200988B2 (en) 2024-08-01
TW202021627A (zh) 2020-06-16
HUE063460T2 (hu) 2024-01-28
ZA201605924B (en) 2017-09-27
JP6669664B2 (ja) 2020-03-18
NZ762841A (en) 2023-11-24
US11103598B2 (en) 2021-08-31
IL247543B (en) 2020-07-30
IL284741B2 (en) 2023-02-01
TWI706789B (zh) 2020-10-11
HUE046454T2 (hu) 2020-03-30
EA201691891A1 (ru) 2017-01-30
SG10201912968WA (en) 2020-02-27
ECSP16083000A (es) 2017-02-24
KR20230006039A (ko) 2023-01-10
IL247543A0 (en) 2016-11-30
MX2020010694A (es) 2020-11-06
CL2016002333A1 (es) 2017-05-26
US20200046851A1 (en) 2020-02-13
US20170173183A1 (en) 2017-06-22
AU2015230942B2 (en) 2020-11-19
PL3628334T3 (pl) 2023-12-18
WO2015143418A2 (en) 2015-09-24
EP3628334A1 (en) 2020-04-01
IL284741A (en) 2021-08-31
JP7534348B2 (ja) 2024-08-14
BR112016021017A2 (pt) 2017-10-03
RS64611B1 (sr) 2023-10-31
PE20161252A1 (es) 2016-11-30
SI3628334T1 (sl) 2023-11-30
MY190726A (en) 2022-05-12
MX2016012201A (es) 2017-01-19
PH12016501684A1 (en) 2016-10-03
MY203654A (en) 2024-07-11
IL275918A (en) 2020-08-31
AU2024227776A1 (en) 2024-11-28
AU2015230942A1 (en) 2016-10-13
CA2943185A1 (en) 2015-09-24
CR20160480A (es) 2016-12-14
DK3119437T3 (da) 2019-12-09
SG11201607005UA (en) 2016-09-29
IL275918B (en) 2021-07-29
UY36044A (es) 2015-10-30
CN106456660A (zh) 2017-02-22
EP4345165A3 (en) 2024-09-25
PL3119437T3 (pl) 2020-04-30
PH12016501684B1 (en) 2023-03-17
CN115252823A (zh) 2022-11-01
EP3119437A2 (en) 2017-01-25
CA2943185C (en) 2025-09-16
JP2022084810A (ja) 2022-06-07
SI3119437T1 (sl) 2020-01-31
MA39390A1 (fr) 2017-12-29
DOP2016000237A (es) 2016-10-16
KR20250172735A (ko) 2025-12-09
HRP20231077T1 (hr) 2023-12-22
AR099837A1 (es) 2016-08-24
KR20160127832A (ko) 2016-11-04
PT3628334T (pt) 2023-09-26
JP7048563B2 (ja) 2022-04-05
TWI780401B (zh) 2022-10-11
TW201622752A (zh) 2016-07-01
DK3628334T3 (da) 2023-10-02
DK3628334T5 (da) 2024-09-02
JP2020059737A (ja) 2020-04-16
MA39390B2 (fr) 2022-04-29
IL284741B (en) 2022-10-01
MX376190B (es) 2025-03-07
EP4345165A2 (en) 2024-04-03
WO2015143418A3 (en) 2015-11-19
UA120050C2 (uk) 2019-09-25
PT3119437T (pt) 2019-12-12
LT3119437T (lt) 2019-12-27
US12201698B2 (en) 2025-01-21
CA3254798A1 (en) 2025-12-01
EP3119437B1 (en) 2019-09-04
FI3628334T3 (fi) 2023-09-15
NZ724622A (en) 2022-05-27
EP3628334B1 (en) 2023-06-28
ES2760263T3 (es) 2020-05-13
US10383953B2 (en) 2019-08-20
US20220054657A1 (en) 2022-02-24
AU2021200988A1 (en) 2021-03-11
SG10201808218YA (en) 2018-10-30
JP2017509632A (ja) 2017-04-06

Similar Documents

Publication Publication Date Title
ES2957840T3 (es) Tratamiento génico de la retinitis pigmentaria
JP7814430B2 (ja) 深部イントロン突然変異の遺伝子編集
JP2020535803A (ja) バリアントＲＮＡｉ
HK40109446A (en) Gene therapy for retinitis pigmentosa
HK40080889A (en) Gene therapy for retinitis pigmentosa
HK40026944B (en) Gene therapy for retinitis pigmentosa
HK40026944A (en) Gene therapy for retinitis pigmentosa
BR112016021017B1 (pt) Partículas de raav, composições e uso das mesmas no tratamento para retinite pigmentosa ou no tratamento do estresse do retículo endoplasmático
HK1233535B (en) Gene therapy for retinitis pigmentosa
HK1233535A1 (en) Gene therapy for retinitis pigmentosa
HK40116478A (zh) 深内含子突变的基因编辑