AR099837A1 - Terapia génica para la retinitis pigmentaria - Google Patents

Terapia génica para la retinitis pigmentaria

Info

Publication number
AR099837A1
AR099837A1 ARP150100879A ARP150100879A AR099837A1 AR 099837 A1 AR099837 A1 AR 099837A1 AR P150100879 A ARP150100879 A AR P150100879A AR P150100879 A ARP150100879 A AR P150100879A AR 099837 A1 AR099837 A1 AR 099837A1
Authority
AR
Argentina
Prior art keywords
gene therapy
pigmentary retinitis
viral particles
particles
retinitis
Prior art date
Application number
ARP150100879A
Other languages
English (en)
Spanish (es)
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=AR099837(A1) "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 AR099837A1 publication Critical patent/AR099837A1/es

Links

Classifications

    • 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
    • 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
    • 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
    • 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
    • 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)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Molecular Biology (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)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (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)
ARP150100879A 2014-03-21 2015-03-25 Terapia génica para la retinitis pigmentaria AR099837A1 (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
AR099837A1 true AR099837A1 (es) 2016-08-24

Family

ID=52829343

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP150100879A AR099837A1 (es) 2014-03-21 2015-03-25 Terapia génica para la retinitis pigmentaria

Country Status (36)

Country Link
US (3) US10383953B2 (OSRAM)
EP (3) EP3628334B1 (OSRAM)
JP (3) JP6669664B2 (OSRAM)
KR (2) KR20230006039A (OSRAM)
CN (2) CN115252823A (OSRAM)
AR (1) AR099837A1 (OSRAM)
AU (3) AU2015230942B2 (OSRAM)
CA (2) CA2943185C (OSRAM)
CL (1) CL2016002333A1 (OSRAM)
CR (1) CR20160480A (OSRAM)
DK (2) DK3119437T3 (OSRAM)
DO (1) DOP2016000237A (OSRAM)
EA (1) EA201691891A1 (OSRAM)
EC (1) ECSP16083000A (OSRAM)
ES (2) ES2957840T3 (OSRAM)
FI (1) FI3628334T3 (OSRAM)
HR (2) HRP20231077T1 (OSRAM)
HU (2) HUE063460T2 (OSRAM)
IL (3) IL247543B (OSRAM)
LT (2) LT3628334T (OSRAM)
MA (1) MA39390B2 (OSRAM)
MX (2) MX376190B (OSRAM)
MY (2) MY190726A (OSRAM)
NZ (1) NZ724622A (OSRAM)
PE (1) PE20161252A1 (OSRAM)
PH (1) PH12016501684B1 (OSRAM)
PL (2) PL3119437T3 (OSRAM)
PT (2) PT3628334T (OSRAM)
RS (2) RS59634B1 (OSRAM)
SG (3) SG11201607005UA (OSRAM)
SI (2) SI3628334T1 (OSRAM)
TW (2) TWI780401B (OSRAM)
UA (1) UA120050C2 (OSRAM)
UY (1) UY36044A (OSRAM)
WO (1) WO2015143418A2 (OSRAM)
ZA (1) ZA201605924B (OSRAM)

Families Citing this family (28)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
UA120050C2 (uk) * 2014-03-21 2019-09-25 Джензайм Корпорейшн Генна терапія для лікування пігментного ретиніту
MA40819B1 (fr) 2015-02-10 2020-04-30 Genzyme Corp Variant d'arni
WO2017093936A1 (en) 2015-12-03 2017-06-08 Friedrich Miescher Institute For Biomedical Research Synp162, a promoter for the specific expression of genes in rod photoreceptors
CN108474001B (zh) * 2015-12-03 2022-04-08 弗里德里克·米谢尔生物医学研究所 SynP160，用于基因在视杆光感受器中特异性表达的启动子
KR102312253B1 (ko) * 2015-12-04 2021-10-15 쏘흐본느 유니베흐시테 프로모터 및 이의 용도
IL261014B2 (en) * 2016-03-01 2023-10-01 Univ Florida Aav vectors for treatment of dominant retinitis pigmentosa
SG11201900049QA (en) * 2016-07-05 2019-02-27 Univ Johns Hopkins Crispr/cas9-based compositions and methods for treating retinal degenerations
AU2018238422B2 (en) * 2017-03-21 2025-04-03 Martin Biel Gene therapy for the treatment of CNGB1-linked retinitis pigmentosa
WO2019060726A1 (en) 2017-09-22 2019-03-28 Genzyme Corporation Variant rnai
WO2019067766A1 (en) * 2017-09-27 2019-04-04 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
US12162994B2 (en) 2018-03-02 2024-12-10 Sigilon Therapeutics, Inc. Biocompatible hydrogel capsules and process for preparing same
EP4186921A1 (en) * 2018-03-23 2023-05-31 The Trustees of Columbia University in the City of New York Gene editing for autosomal dominant diseases
UY38160A (es) 2018-04-04 2019-11-29 Sigilon Therapeutics Inc Partículas implantables y métodos relacionados
JP7563694B2 (ja) 2018-06-01 2024-10-08 ユニバーシティー オブ フロリダ リサーチ ファンデーション， インク． 優性網膜色素変性症の処置のための組成物および方法
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
AU2020232692A1 (en) * 2019-03-04 2021-09-30 The Trustees Of The University Of Pennsylvania Neuroprotective gene therapy targeting the AKT pathway
KR20210145180A (ko) * 2019-03-21 2021-12-01 피티씨 테라퓨틱스, 인크. 엔젤만 증후군을 치료하기 위한 벡터 및 방법
BR112022002794A2 (pt) * 2019-08-15 2022-08-09 Childrens Hospital Philadelphia Terapia combinada de transgene e mirna derivado de íntron para tratamento de sca1
EP4087617A4 (en) * 2020-01-10 2024-05-01 Solid Biosciences Inc. VIRAL VECTOR FOR COMBINATION THERAPY
CN115989234A (zh) * 2020-01-29 2023-04-18 建新公司 用于眼部基因疗法的经修饰的腺相关病毒衣壳蛋白及其使用方法
KR102396200B1 (ko) * 2020-07-24 2022-05-12 알지노믹스 주식회사 로돕신 전사체에 특이적인 트랜스-스플라이싱 라이보자임 및 이의 용도
WO2022026632A2 (en) * 2020-07-29 2022-02-03 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
CN120098992A (zh) * 2023-12-05 2025-06-06 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
US6995006B2 (en) 1997-09-05 2006-02-07 Targeted Genetics Corporation Methods for generating high titer helper-free preparations of released recombinant AAV vectors
JP4827353B2 (ja) 1999-08-09 2011-11-30 ターゲティッド ジェネティクス コーポレイション 鎖内塩基対を形成するような配列の設計による、組換えウイルスベクターからの一本鎖の異種ヌクレオチド配列の発現の増大
WO2001092551A2 (en) 2000-06-01 2001-12-06 University Of North Carolina At Chapel Hill Duplexed parvovirus vectors
SG168422A1 (en) 2001-11-13 2011-02-28 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
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
EP2007795B1 (en) 2006-03-30 2016-11-16 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid proteins
ES2907452T3 (es) * 2007-04-12 2022-04-25 The Provost Fellows Found Scholars & The Other Members Of Board Of The College Of The Holy & Undiv T Supresión y reemplazo genético
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
WO2010138263A2 (en) * 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
US9078914B2 (en) 2009-09-10 2015-07-14 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
EP2634253B1 (en) * 2010-10-27 2016-05-11 Jichi Medical University Adeno-associated virus virions for transferring genes into neural cells
SG10202007803QA (en) 2011-02-17 2020-09-29 Univ Pennsylvania Compositions and methods for altering tissue specificity and improving aav9-mediated gene transfer
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
CN104520428B (zh) * 2012-02-17 2018-09-21 费城儿童医院 将基因转移到细胞、器官和组织的aav载体组合物和方法
AU2013243952A1 (en) * 2012-04-02 2014-10-30 Modernatx, Inc. Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
US9127274B2 (en) * 2012-04-26 2015-09-08 Alnylam Pharmaceuticals, Inc. Serpinc1 iRNA compositions and methods of use thereof
SI3401400T1 (sl) * 2012-05-25 2019-10-30 Univ California Postopki in sestavki za RNA usmerjeno modifikacijo tarčne DNA in za RNA usmerjeno modulacijo prepisovanja
JP6199965B2 (ja) * 2012-07-11 2017-09-20 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア Ｒｐｇｒｘ連鎖性網膜変性のａａｖ媒介型遺伝子治療
WO2014066498A1 (en) * 2012-10-23 2014-05-01 Vivek Mittal Treatment of metastatic breast cancer
US9884125B2 (en) 2013-10-04 2018-02-06 Merck Sharp & Dohme Corp. Glucose-responsive insulin conjugates
UA120050C2 (uk) * 2014-03-21 2019-09-25 Джензайм Корпорейшн Генна терапія для лікування пігментного ретиніту

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CN106456660A (zh) 2017-02-22
EP4345165A3 (en) 2024-09-25
CR20160480A (es) 2016-12-14
IL275918A (en) 2020-08-31
JP7048563B2 (ja) 2022-04-05
SI3119437T1 (sl) 2020-01-31
HUE063460T2 (hu) 2024-01-28
CA2943185C (en) 2025-09-16
MX2016012201A (es) 2017-01-19
TW202021627A (zh) 2020-06-16
DK3628334T3 (da) 2023-10-02
DK3628334T5 (da) 2024-09-02
UA120050C2 (uk) 2019-09-25
MY203654A (en) 2024-07-11
FI3628334T3 (fi) 2023-09-15
LT3628334T (lt) 2023-09-25
KR20160127832A (ko) 2016-11-04
DOP2016000237A (es) 2016-10-16
US20200046851A1 (en) 2020-02-13
MA39390B2 (fr) 2022-04-29
US10383953B2 (en) 2019-08-20
TWI706789B (zh) 2020-10-11
KR20230006039A (ko) 2023-01-10
WO2015143418A3 (en) 2015-11-19
MX376190B (es) 2025-03-07
IL275918B (en) 2021-07-29
WO2015143418A2 (en) 2015-09-24
MY190726A (en) 2022-05-12
EA201691891A1 (ru) 2017-01-30
HUE046454T2 (hu) 2020-03-30
DK3119437T3 (da) 2019-12-09
SG11201607005UA (en) 2016-09-29
PL3628334T3 (pl) 2023-12-18
PE20161252A1 (es) 2016-11-30
EP4345165A2 (en) 2024-04-03
MX2020010694A (es) 2020-11-06
AU2015230942B2 (en) 2020-11-19
AU2021200988A1 (en) 2021-03-11
SG10201808218YA (en) 2018-10-30
JP2017509632A (ja) 2017-04-06
SI3628334T1 (sl) 2023-11-30
JP6669664B2 (ja) 2020-03-18
US20170173183A1 (en) 2017-06-22
RS64611B1 (sr) 2023-10-31
TWI780401B (zh) 2022-10-11
ES2957840T3 (es) 2024-01-26
EP3628334B1 (en) 2023-06-28
CL2016002333A1 (es) 2017-05-26
PL3119437T3 (pl) 2020-04-30
EP3119437B1 (en) 2019-09-04
UY36044A (es) 2015-10-30
PH12016501684B1 (en) 2023-03-17
JP2022084810A (ja) 2022-06-07
SG10201912968WA (en) 2020-02-27
CA2943185A1 (en) 2015-09-24
JP7534348B2 (ja) 2024-08-14
RS59634B1 (sr) 2020-01-31
US12201698B2 (en) 2025-01-21
IL284741B (en) 2022-10-01
IL247543A0 (en) 2016-11-30
EP3119437A2 (en) 2017-01-25
PT3119437T (pt) 2019-12-12
LT3119437T (lt) 2019-12-27
HRP20192141T1 (hr) 2020-02-21
NZ762841A (en) 2023-11-24
BR112016021017A2 (pt) 2017-10-03
US20220054657A1 (en) 2022-02-24
PH12016501684A1 (en) 2016-10-03
MA39390A1 (fr) 2017-12-29
AU2021200988B2 (en) 2024-08-01
HRP20231077T1 (hr) 2023-12-22
ZA201605924B (en) 2017-09-27
JP2020059737A (ja) 2020-04-16
AU2015230942A1 (en) 2016-10-13
CA3254798A1 (en) 2025-12-01
EP3628334A1 (en) 2020-04-01
IL284741A (en) 2021-08-31
AU2024227776A1 (en) 2024-11-28
ES2760263T3 (es) 2020-05-13
CN115252823A (zh) 2022-11-01
CN106456660B (zh) 2022-05-31
US11103598B2 (en) 2021-08-31
TW201622752A (zh) 2016-07-01
PT3628334T (pt) 2023-09-26
ECSP16083000A (es) 2017-02-24
IL284741B2 (en) 2023-02-01
IL247543B (en) 2020-07-30
NZ724622A (en) 2022-05-27

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