AU2015230942B2 - Gene therapy for retinitis pigmentosa - Google Patents

Gene therapy for retinitis pigmentosa Download PDF

Info

Publication number
AU2015230942B2
AU2015230942B2 AU2015230942A AU2015230942A AU2015230942B2 AU 2015230942 B2 AU2015230942 B2 AU 2015230942B2 AU 2015230942 A AU2015230942 A AU 2015230942A AU 2015230942 A AU2015230942 A AU 2015230942A AU 2015230942 B2 AU2015230942 B2 AU 2015230942B2
Authority
AU
Australia
Prior art keywords
mir
rhodopsin
nucleic acid
seq
aav
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
AU2015230942A
Other languages
English (en)
Other versions
AU2015230942A1 (en
Inventor
Matthew ADAMOWICZ
Catherine O'riordan
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=AU2015230942(B2) "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 AU2015230942A1 publication Critical patent/AU2015230942A1/en
Application granted granted Critical
Publication of AU2015230942B2 publication Critical patent/AU2015230942B2/en
Priority to AU2021200988A priority Critical patent/AU2021200988B2/en
Priority to AU2024227776A priority patent/AU2024227776A1/en
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
    • 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
    • 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
    • A61K48/0066—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)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (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)
  • 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)
AU2015230942A 2014-03-21 2015-03-20 Gene therapy for retinitis pigmentosa Active AU2015230942B2 (en)

Priority Applications (2)

Application Number Priority Date Filing Date Title
AU2021200988A AU2021200988B2 (en) 2014-03-21 2021-02-15 Gene therapy for retinitis pigmentosa
AU2024227776A AU2024227776A1 (en) 2014-03-21 2024-10-30 Gene therapy for retinitis pigmentosa

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201461969027P 2014-03-21 2014-03-21
US61/969,027 2014-03-21
PCT/US2015/021896 WO2015143418A2 (en) 2014-03-21 2015-03-20 Gene therapy for retinitis pigmentosa

Related Child Applications (1)

Application Number Title Priority Date Filing Date
AU2021200988A Division AU2021200988B2 (en) 2014-03-21 2021-02-15 Gene therapy for retinitis pigmentosa

Publications (2)

Publication Number Publication Date
AU2015230942A1 AU2015230942A1 (en) 2016-10-13
AU2015230942B2 true AU2015230942B2 (en) 2020-11-19

Family

ID=52829343

Family Applications (3)

Application Number Title Priority Date Filing Date
AU2015230942A Active AU2015230942B2 (en) 2014-03-21 2015-03-20 Gene therapy for retinitis pigmentosa
AU2021200988A Active AU2021200988B2 (en) 2014-03-21 2021-02-15 Gene therapy for retinitis pigmentosa
AU2024227776A Pending AU2024227776A1 (en) 2014-03-21 2024-10-30 Gene therapy for retinitis pigmentosa

Family Applications After (2)

Application Number Title Priority Date Filing Date
AU2021200988A Active AU2021200988B2 (en) 2014-03-21 2021-02-15 Gene therapy for retinitis pigmentosa
AU2024227776A Pending AU2024227776A1 (en) 2014-03-21 2024-10-30 Gene therapy for retinitis pigmentosa

Country Status (36)

Country Link
US (3) US10383953B2 (OSRAM)
EP (3) EP3119437B1 (OSRAM)
JP (3) JP6669664B2 (OSRAM)
KR (3) KR20230006039A (OSRAM)
CN (2) CN106456660B (OSRAM)
AR (1) AR099837A1 (OSRAM)
AU (3) AU2015230942B2 (OSRAM)
CA (2) CA2943185C (OSRAM)
CL (1) CL2016002333A1 (OSRAM)
CR (1) CR20160480A (OSRAM)
DK (2) DK3628334T5 (OSRAM)
DO (1) DOP2016000237A (OSRAM)
EA (1) EA201691891A1 (OSRAM)
EC (1) ECSP16083000A (OSRAM)
ES (2) ES2760263T3 (OSRAM)
FI (1) FI3628334T3 (OSRAM)
HR (2) HRP20231077T1 (OSRAM)
HU (2) HUE046454T2 (OSRAM)
IL (3) IL247543B (OSRAM)
LT (2) LT3119437T (OSRAM)
MA (1) MA39390B2 (OSRAM)
MX (2) MX376190B (OSRAM)
MY (2) MY203654A (OSRAM)
NZ (1) NZ724622A (OSRAM)
PE (1) PE20161252A1 (OSRAM)
PH (1) PH12016501684B1 (OSRAM)
PL (2) PL3119437T3 (OSRAM)
PT (2) PT3119437T (OSRAM)
RS (2) RS59634B1 (OSRAM)
SG (3) SG10201912968WA (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
MX376190B (es) 2014-03-21 2025-03-07 Genzyme Corp PARTÍCULAS DE rAAV QUE CODIFICAN miR-708 Y USOS DEL MISMO.
MY181458A (en) 2015-02-10 2020-12-22 Genzyme Corp Variant rnai
ES2886664T3 (es) 2015-12-03 2021-12-20 Friedrich Miescher Institute For Biomedical Res SynP162, un promotor para la expresión específica de genes en fotorreceptores de bastón
ES2900486T3 (es) 2015-12-03 2022-03-17 Friedrich Miescher Institute For Biomedical Res SynP160, un promotor para la expresión específica de genes en los fotorreceptores de los bastones
JP6966442B2 (ja) * 2015-12-04 2021-11-17 ソルボンヌ・ユニヴェルシテSorbonne Universite プロモーター及びその使用
JP7272795B2 (ja) * 2016-03-01 2023-05-12 ユニバーシティ オブ フロリダ リサーチ ファンデーション インコーポレーティッド 優性網膜色素変性の治療のためのａａｖベクター
SG10202109385QA (en) * 2016-07-05 2021-10-28 Univ Johns Hopkins Crispr/cas9-based compositions and methods for treating retinal degenerations
JP7257377B2 (ja) * 2017-03-21 2023-04-13 ミヒャラキス，スティリアノス Ｃｎｇｂ１連鎖性網膜色素変性症の治療のための遺伝子療法
JP7248658B2 (ja) 2017-09-22 2023-03-29 ジェンザイム・コーポレーション バリアントＲＮＡｉ
MX2020003351A (es) * 2017-09-27 2020-10-12 Sigilon Therapeutics Inc Metodos, composiciones y elementos implantables que comprenden celulas activas.
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
WO2019169245A1 (en) 2018-03-02 2019-09-06 Sigilon Therapeutics, Inc. Biocompatible hydrogel capsules and process for preparing same
US20210017509A1 (en) * 2018-03-23 2021-01-21 The Trustees Of Columbia University In The City Of New York Gene Editing for Autosomal Dominant Diseases
IL277713B2 (en) 2018-04-04 2024-09-01 Sigilon Therapeutics Inc Implantable particles and related methods
CA3102095A1 (en) 2018-06-01 2019-12-05 University Of Florida Research Foundation, Incorporated Compositions and methods for treatment of dominant retinitis pigmentosa
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
WO2020180886A1 (en) * 2019-03-04 2020-09-10 The Trustees Of The University Of Pennsylvania Neuroprotective gene therapy targeting the akt pathway
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
CA3151122A1 (en) * 2019-08-15 2021-02-18 The Children's Hospital Of Philadelphia Combined transgene and intron-derived mirna therapy for treatment of 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
CN114521214A (zh) * 2020-07-24 2022-05-20 尔知渃米斯股份有限公司 视紫红质转录体特异性反式剪接核酶及其用途
CA3190214A1 (en) * 2020-07-29 2022-02-03 University Of Florida Research Foundation, Incorporated Improved aav-mediated x-linked retinoschisis therapies
US20220331241A1 (en) * 2021-04-16 2022-10-20 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

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2008125846A2 (en) * 2007-04-12 2008-10-23 The Provost, Fellows And Scholars Of The College Of The Holy And Undivided Trinity Of Queen Elizabeth Genetic suppression and replacement
WO2010138263A2 (en) * 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof

Family Cites Families (29)

* 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
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
WO2001011034A2 (en) 1999-08-09 2001-02-15 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.
ATE317916T1 (de) 2001-11-13 2006-03-15 Univ Pennsylvania Verfahren zur identifizierung von adeno- assoziiertem virus (aav) sequenzen sowie kit zur ausführung der methode
EP1486567A1 (en) 2003-06-11 2004-12-15 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Improved adeno-associated virus (AAV) vector for gene therapy
EP1717317A4 (en) * 2004-01-22 2009-02-11 Dnavec Research Inc METHOD OF GENERATING VIRAL NEGATIVE STRAND RNA VECTORS USING A HYBRID PROMOTER COMPRISING THE CYTOMEGALOVIRUS ENHANCER AND THE BETA ACTIN PROMOTER FROM CHICKEN
US7765583B2 (en) 2005-02-28 2010-07-27 France Telecom System and method for managing virtual user domains
US8283151B2 (en) 2005-04-29 2012-10-09 The United States Of America, As Represented By The Secretary, Department Of Health And 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
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
EP2475372B2 (en) * 2009-09-10 2020-10-21 Velin-Pharma A/S Method for the preparation of micro-rna and its therapeutic application
ES2680915T3 (es) * 2010-01-28 2018-09-11 The Children's Hospital Of Philadelphia Plataforma de fabricación escalable para la purificación de vectores virales y vectores virales purificados de este modo para su utilización en terapia génica
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
SG10201601110VA (en) 2011-02-17 2016-03-30 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
JP6373763B2 (ja) * 2012-02-17 2018-08-22 ザ・チルドレンズ・ホスピタル・オブ・フィラデルフィアＴｈｅ Ｃｈｉｌｄｒｅｎ’Ｓ Ｈｏｓｐｉｔａｌ Ｏｆ Ｐｈｉｌａｄｅｌｐｈｉａ Ａａｖベクター成分、及び、細胞、臓器並びに組織への遺伝子導入のための方法
AU2013243954A1 (en) * 2012-04-02 2014-10-30 Moderna Therapeutics, Inc. Modified polynucleotides for the production of cosmetic proteins and peptides
US9127274B2 (en) * 2012-04-26 2015-09-08 Alnylam Pharmaceuticals, Inc. Serpinc1 iRNA compositions and methods of use thereof
MY189533A (en) * 2012-05-25 2022-02-16 Univ California Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
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
CA2889020A1 (en) * 2012-10-23 2014-05-01 Cornell University Treatment of metastatic breast cancer
RU2676307C2 (ru) 2013-10-04 2018-12-27 Мерк Шарп И Доум Корп. Чувствительные к глюкозе конъюгаты инсулина
MX376190B (es) * 2014-03-21 2025-03-07 Genzyme Corp PARTÍCULAS DE rAAV QUE CODIFICAN miR-708 Y USOS DEL MISMO.

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2008125846A2 (en) * 2007-04-12 2008-10-23 The Provost, Fellows And Scholars Of The College Of The Holy And Undivided Trinity Of Queen Elizabeth Genetic suppression and replacement
US8617876B2 (en) * 2007-04-12 2013-12-31 The Provost Fellows And Scholars Of The College Of The Holy And Undivided Trinity Of Queen Elizabeth Rhodopsin gene conserved regions in a viral vector enhance expression
WO2010138263A2 (en) * 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
Behrman, S. et al. .J. Cell Biol., 2011, Vol.192, No.6, 919-927 *
GREENWALD D L ET AL, GENE THERAPY, (2013-04-01), vol. 20, no. 4, pages 425 - 434 *
MAO H ET AL, HUMAN GENE THERAPY, (2012-04-01), vol. 23, no. 4, pages 356 - 366 *

Also Published As

Publication number Publication date
TWI780401B (zh) 2022-10-11
IL275918A (en) 2020-08-31
IL275918B (en) 2021-07-29
DK3628334T3 (da) 2023-10-02
DK3628334T5 (da) 2024-09-02
RS59634B1 (sr) 2020-01-31
KR20230006039A (ko) 2023-01-10
PH12016501684B1 (en) 2023-03-17
MX2020010694A (es) 2020-11-06
TW202021627A (zh) 2020-06-16
LT3119437T (lt) 2019-12-27
US12201698B2 (en) 2025-01-21
CR20160480A (es) 2016-12-14
US20170173183A1 (en) 2017-06-22
IL247543B (en) 2020-07-30
ECSP16083000A (es) 2017-02-24
HRP20192141T1 (hr) 2020-02-21
PE20161252A1 (es) 2016-11-30
AU2021200988B2 (en) 2024-08-01
TWI706789B (zh) 2020-10-11
EP3119437A2 (en) 2017-01-25
RS64611B1 (sr) 2023-10-31
DOP2016000237A (es) 2016-10-16
MX376190B (es) 2025-03-07
PH12016501684A1 (en) 2016-10-03
NZ762841A (en) 2023-11-24
US20200046851A1 (en) 2020-02-13
CN106456660A (zh) 2017-02-22
IL284741B2 (en) 2023-02-01
KR20250172735A (ko) 2025-12-09
JP7048563B2 (ja) 2022-04-05
MA39390B2 (fr) 2022-04-29
UY36044A (es) 2015-10-30
US20220054657A1 (en) 2022-02-24
AU2021200988A1 (en) 2021-03-11
SI3628334T1 (sl) 2023-11-30
TW201622752A (zh) 2016-07-01
ES2957840T3 (es) 2024-01-26
MA39390A1 (fr) 2017-12-29
CA2943185C (en) 2025-09-16
EP3628334B1 (en) 2023-06-28
MY190726A (en) 2022-05-12
ZA201605924B (en) 2017-09-27
SG10201808218YA (en) 2018-10-30
NZ724622A (en) 2022-05-27
JP6669664B2 (ja) 2020-03-18
IL284741B (en) 2022-10-01
EP3628334A1 (en) 2020-04-01
IL284741A (en) 2021-08-31
IL247543A0 (en) 2016-11-30
WO2015143418A3 (en) 2015-11-19
US11103598B2 (en) 2021-08-31
SG10201912968WA (en) 2020-02-27
US10383953B2 (en) 2019-08-20
CA2943185A1 (en) 2015-09-24
JP7534348B2 (ja) 2024-08-14
AU2015230942A1 (en) 2016-10-13
UA120050C2 (uk) 2019-09-25
CN115252823A (zh) 2022-11-01
EA201691891A1 (ru) 2017-01-30
SG11201607005UA (en) 2016-09-29
HUE063460T2 (hu) 2024-01-28
EP4345165A3 (en) 2024-09-25
MX2016012201A (es) 2017-01-19
DK3119437T3 (da) 2019-12-09
LT3628334T (lt) 2023-09-25
AU2024227776A1 (en) 2024-11-28
PL3628334T3 (pl) 2023-12-18
MY203654A (en) 2024-07-11
AR099837A1 (es) 2016-08-24
FI3628334T3 (fi) 2023-09-15
KR20160127832A (ko) 2016-11-04
EP4345165A2 (en) 2024-04-03
HRP20231077T1 (hr) 2023-12-22
JP2017509632A (ja) 2017-04-06
JP2022084810A (ja) 2022-06-07
BR112016021017A2 (pt) 2017-10-03
CL2016002333A1 (es) 2017-05-26
JP2020059737A (ja) 2020-04-16
EP3119437B1 (en) 2019-09-04
CA3254798A1 (en) 2025-12-01
PT3628334T (pt) 2023-09-26
PT3119437T (pt) 2019-12-12
PL3119437T3 (pl) 2020-04-30
HUE046454T2 (hu) 2020-03-30
ES2760263T3 (es) 2020-05-13
WO2015143418A2 (en) 2015-09-24
CN106456660B (zh) 2022-05-31
SI3119437T1 (sl) 2020-01-31

Similar Documents

Publication Publication Date Title
AU2021200988B2 (en) Gene therapy for retinitis pigmentosa
AU2020260485B2 (en) Gene therapies for lysosomal disorders
AU2023214366B2 (en) Gene therapies for lysosomal disorders
US20050251872A1 (en) Lentiviral vectors, related reagents, and methods of use thereof
CN1938428A (zh) 多基因表达的质粒系统
US6187991B1 (en) Transgenic animal models for type II diabetes mellitus
CN113322281B (zh) 一种高效组织特异性表达rs1蛋白的重组腺相关病毒及应用
CN110305902B (zh) 一种在工具细胞中激活hSyn启动子的方法及其应用
CN101679976A (zh) 核酸和文库
KR20220064647A (ko) 내산성 효모 유전자 기반 합성 프로모터
CN114990163A (zh) 用于干细胞基因修饰的慢病毒载体及其构建方法和应用
HK40048832A (en) Gene therapies for lysosomal disorders
TW202309287A (zh) 調節基因表現之組合物及方法
HK40046514A (en) Gene therapies for lysosomal disorders
KR20210150487A (ko) 리소좀 장애를 위한 유전자 요법
HK40048832B (zh) 用於溶酶体障碍的基因疗法
HK40067825A (zh) 用於溶酶体障碍的基因疗法

Legal Events

Date Code Title Description
FGA Letters patent sealed or granted (standard patent)