WO2009134681A3 - Adeno associated viral vectors for targeted transduction of retinal pigment epithetial cells - Google Patents

Adeno associated viral vectors for targeted transduction of retinal pigment epithetial cells Download PDF

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Publication number
WO2009134681A3
WO2009134681A3 PCT/US2009/041606 US2009041606W WO2009134681A3 WO 2009134681 A3 WO2009134681 A3 WO 2009134681A3 US 2009041606 W US2009041606 W US 2009041606W WO 2009134681 A3 WO2009134681 A3 WO 2009134681A3
Authority
WO
WIPO (PCT)
Prior art keywords
cells
retinal pigment
epithetial
viral vectors
rpe
Prior art date
Application number
PCT/US2009/041606
Other languages
French (fr)
Other versions
WO2009134681A2 (en
Inventor
James M. Wilson
Luc H. Vandenberghe
Jean Bennett
Karen Kozarsky
Peter Ertl
Original Assignee
The Trustees Of The University Of Pennsylvania
Smithkline Beecham Corporation
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by The Trustees Of The University Of Pennsylvania, Smithkline Beecham Corporation filed Critical The Trustees Of The University Of Pennsylvania
Publication of WO2009134681A2 publication Critical patent/WO2009134681A2/en
Publication of WO2009134681A3 publication Critical patent/WO2009134681A3/en

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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
    • 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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • C12N2799/00—Uses of viruses
    • C12N2799/02—Uses of viruses as vector
    • C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
    • C12N2799/025—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a parvovirus

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Biotechnology (AREA)
  • Genetics & Genomics (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

A method for specifically targeting a gene product to a retinal pigment epithelial cell is provided, along with adeno-associated viruses (AAVs) useful therein. The method involves delivering to a subject's eye a dose of about 109 genome copies of an AAV7 viral vector and a minigene comprising sequences encoding the gene product targeted to the RPE cells. A synthetic RPE gene 65 is also described. Suitably, this synthetic RPE gene is under the control of regulatory control sequences which direct expression of the RPE65 protein in RPE cells and is delivered via the AAV7 viral vector.
PCT/US2009/041606 2008-04-30 2009-04-24 Aav7 viral vectors for targeted delivery of rpe cells WO2009134681A2 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US12611008P 2008-04-30 2008-04-30
US61/126,110 2008-04-30

Publications (2)

Publication Number Publication Date
WO2009134681A2 WO2009134681A2 (en) 2009-11-05
WO2009134681A3 true WO2009134681A3 (en) 2011-04-28

Family

ID=40974429

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2009/041606 WO2009134681A2 (en) 2008-04-30 2009-04-24 Aav7 viral vectors for targeted delivery of rpe cells

Country Status (1)

Country Link
WO (1) WO2009134681A2 (en)

Families Citing this family (30)

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EP1381276A4 (en) 2001-04-13 2005-02-02 Univ Pennsylvania Method of treating or retarding the development of blindness
CN110777124A (en) 2011-10-27 2020-02-11 威尔斯达眼科制剂公司 Vectors encoding rod-derived cone viability factors
RU2679843C2 (en) * 2012-07-06 2019-02-13 Юниверсити Оф Айова Рисерч Фаундейшн Modified adeno-associated virus vector compositions
EP2951290B1 (en) 2013-02-01 2017-11-29 The United States of America, as represented by The Secretary, Department of Health and Human Services Method for generating retinal pigment epithelium (rpe) cells from induced pluripotent stem cells (ipscs)
WO2015191508A1 (en) 2014-06-09 2015-12-17 Voyager Therapeutics, Inc. Chimeric capsids
EP3215191A4 (en) 2014-11-05 2018-08-01 Voyager Therapeutics, Inc. Aadc polynucleotides for the treatment of parkinson's disease
CN107109407A (en) 2014-11-14 2017-08-29 沃雅戈治疗公司 Treat the composition and method of amyotrophic lateral sclerosis (ALS)
SG11201703419UA (en) 2014-11-14 2017-05-30 Voyager Therapeutics Inc Modulatory polynucleotides
US11697825B2 (en) 2014-12-12 2023-07-11 Voyager Therapeutics, Inc. Compositions and methods for the production of scAAV
SI3265568T1 (en) * 2015-03-06 2020-10-30 Massachusetts Eye & Ear Infirmary Gene augmentation therapies for inherited retinal degeneration caused by mutations in the prpf31 gene
AU2016362477A1 (en) 2015-12-02 2018-06-14 Voyager Therapeutics, Inc. Assays for the detection of AAV neutralizing antibodies
WO2017189964A2 (en) 2016-04-29 2017-11-02 Voyager Therapeutics, Inc. Compositions for the treatment of disease
CN110214187B (en) 2016-05-18 2024-01-30 沃雅戈治疗公司 Regulatory polynucleotides
CA3024449A1 (en) 2016-05-18 2017-11-23 Voyager Therapeutics, Inc. Compositions and methods of treating huntington's disease
US11298041B2 (en) 2016-08-30 2022-04-12 The Regents Of The University Of California Methods for biomedical targeting and delivery and devices and systems for practicing the same
EP3618839A4 (en) 2017-05-05 2021-06-09 Voyager Therapeutics, Inc. Compositions and methods of treating amyotrophic lateral sclerosis (als)
SG11201909868YA (en) 2017-05-05 2019-11-28 Voyager Therapeutics Inc Compositions and methods of treating huntington's disease
JOP20190269A1 (en) 2017-06-15 2019-11-20 Voyager Therapeutics Inc Aadc polynucleotides for the treatment of parkinson's disease
CN111132626B (en) 2017-07-17 2024-01-30 沃雅戈治疗公司 Track array guidance system
WO2019028306A2 (en) 2017-08-03 2019-02-07 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
AU2018352236A1 (en) 2017-10-16 2020-04-23 The Curators Of The University Of Missouri Treatment of amyotrophic lateral sclerosis (ALS)
WO2019079242A1 (en) 2017-10-16 2019-04-25 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
CN111726985B (en) * 2017-11-15 2022-06-10 弗里德里克·米谢尔生物医学研究所 Primate retinal pigment epithelial cell specific promoter
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
EP3755795A4 (en) 2018-02-19 2022-07-20 Homology Medicines, Inc. Adeno-associated virus compositions for restoring f8 gene function and methods of use thereof
EP3829650A4 (en) * 2018-08-03 2022-05-04 Sangamo Therapeutics, Inc. Improved clinical parameters by expression of factor viii
KR20220106742A (en) * 2019-09-25 2022-07-29 유니버시티 오브 유타 리서치 파운데이션 Methods and compositions for the expression of constitutively active RAP1A from the VMD2 promoter
TW202140791A (en) 2020-01-13 2021-11-01 美商霍蒙拉奇醫藥公司 Methods of treating phenylketonuria
KR20220150906A (en) * 2020-02-07 2022-11-11 더 트러스티스 오브 컬럼비아 유니버시티 인 더 시티 오브 뉴욕 Metabolome reprogramming to delay onset or treat neurodegeneration
MX2023008826A (en) 2021-02-01 2023-09-15 Regenxbio Inc Gene therapy for neuronal ceroid lipofuscinoses.

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2002082904A2 (en) * 2001-04-13 2002-10-24 The Trustees Of The University Of Pennsylvania Method of treating or retarding the development of blindness

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2002082904A2 (en) * 2001-04-13 2002-10-24 The Trustees Of The University Of Pennsylvania Method of treating or retarding the development of blindness

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
BENNICELLI JEANNETTE ET AL: "Reversal of blindness in animal models of leber congenital amaurosis using optimized AAV2-mediated gene transfer.", MOLECULAR THERAPY : THE JOURNAL OF THE AMERICAN SOCIETY OF GENE THERAPY MAR 2008, vol. 16, no. 3, March 2008 (2008-03-01), pages 458 - 465, XP002543023, ISSN: 1525-0024 *
LE MEUR G ET AL: "Restoration of vision in RPE65-deficient Briard dogs using an AAV serotype 4 vector that specifically targets the retinal pigmented epithelium", GENE THERAPY, MACMILLAN PRESS LTD., BASINGSTOKE, GB, vol. 14, no. 4, 1 February 2007 (2007-02-01), pages 292 - 303, XP002493281, ISSN: 0969-7128, [retrieved on 20061005] *
LEBHERZ CORINNA ET AL: "Novel AAV serotypes for improved ocular gene transfer", JOURNAL OF GENE MEDICINE, vol. 10, no. 4, 18 February 2008 (2008-02-18), pages 375 - 382, XP002543022, ISSN: 1521-2254, Retrieved from the Internet <URL:www.interscience.wiley.com> *
PANG ET AL: "Comparative analysis of in vivo and in vitro AAV vector transduction in the neonatal mouse retina: Effects of serotype and site of administration", VISION RESEARCH, PERGAMON PRESS, OXFORD, GB, vol. 48, no. 3, 22 October 2007 (2007-10-22), pages 377 - 385, XP022452232, ISSN: 0042-6989 *

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