JP2023510186A5 - - Google Patents

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Publication number
JP2023510186A5
JP2023510186A5 JP2022540642A JP2022540642A JP2023510186A5 JP 2023510186 A5 JP2023510186 A5 JP 2023510186A5 JP 2022540642 A JP2022540642 A JP 2022540642A JP 2022540642 A JP2022540642 A JP 2022540642A JP 2023510186 A5 JP2023510186 A5 JP 2023510186A5
Authority
JP
Japan
Prior art keywords
pharmaceutical composition
composition according
crx
vector
promoter
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.)
Granted
Application number
JP2022540642A
Other languages
English (en)
Japanese (ja)
Other versions
JP7703543B2 (ja
JP2023510186A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2021/013733 external-priority patent/WO2021146625A1/en
Publication of JP2023510186A publication Critical patent/JP2023510186A/ja
Publication of JP2023510186A5 publication Critical patent/JP2023510186A5/ja
Application granted granted Critical
Publication of JP7703543B2 publication Critical patent/JP7703543B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

JP2022540642A 2020-01-17 2021-01-15 Crx常染色体優性網膜症の処置のための遺伝子治療 Active JP7703543B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202062962732P 2020-01-17 2020-01-17
US62/962,732 2020-01-17
PCT/US2021/013733 WO2021146625A1 (en) 2020-01-17 2021-01-15 Gene therapy for treatment of crx-autosomal dominant retinopathies

Publications (3)

Publication Number Publication Date
JP2023510186A JP2023510186A (ja) 2023-03-13
JP2023510186A5 true JP2023510186A5 (https=) 2024-01-22
JP7703543B2 JP7703543B2 (ja) 2025-07-07

Family

ID=74595396

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022540642A Active JP7703543B2 (ja) 2020-01-17 2021-01-15 Crx常染色体優性網膜症の処置のための遺伝子治療

Country Status (6)

Country Link
US (1) US20230066585A1 (https=)
EP (1) EP4090381A1 (https=)
JP (1) JP7703543B2 (https=)
AU (1) AU2021208631A1 (https=)
CA (1) CA3165922A1 (https=)
WO (1) WO2021146625A1 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2022217142A1 (en) * 2021-04-09 2022-10-13 Washington University Compositions and methods for treatment of crx-linked retinopathies
GB202205265D0 (en) * 2022-04-11 2022-05-25 Mogrify Ltd Cell conversion

Family Cites Families (31)

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US4683195A (en) 1986-01-30 1987-07-28 Cetus Corporation Process for amplifying, detecting, and/or-cloning nucleic acid sequences
US5091309A (en) 1986-01-16 1992-02-25 Washington University Sindbis virus vectors
US5217879A (en) 1989-01-12 1993-06-08 Washington University Infectious Sindbis virus vectors
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
US5443505A (en) 1993-11-15 1995-08-22 Oculex Pharmaceuticals, Inc. Biocompatible ocular implants
US5725493A (en) 1994-12-12 1998-03-10 Avery; Robert Logan Intravitreal medicine delivery
US5869079A (en) 1995-06-02 1999-02-09 Oculex Pharmaceuticals, Inc. Formulation for controlled release of drugs by combining hydrophilic and hydrophobic agents
US6299895B1 (en) 1997-03-24 2001-10-09 Neurotech S.A. Device and method for treating ophthalmic diseases
US6759050B1 (en) 1998-12-03 2004-07-06 Avigen, Inc. Excipients for use in adeno-associated virus pharmaceutical formulations, and pharmaceutical formulations made therewith
US6416777B1 (en) 1999-10-21 2002-07-09 Alcon Universal Ltd. Ophthalmic drug delivery device
CA2383499C (en) 1999-10-21 2009-11-24 Alcon Universal Ltd. Drug delivery device
EP1339438B1 (en) 2000-11-29 2005-10-19 Allergan Inc. Preventing transplant rejection in the eye
WO2002082904A2 (en) * 2001-04-13 2002-10-24 The Trustees Of The University Of Pennsylvania Method of treating or retarding the development of blindness
GB0125216D0 (en) 2001-10-19 2001-12-12 Univ Strathclyde Dendrimers for use in targeted delivery
CA2466229A1 (en) 2001-11-06 2003-10-30 Eli Lilly And Company Use of il-19, il-22 and il-24 to treat hematopoietic disorders
US6780324B2 (en) 2002-03-18 2004-08-24 Labopharm, Inc. Preparation of sterile stabilized nanodispersions
US7261544B2 (en) 2003-05-21 2007-08-28 Genzyme Corporation Methods for producing preparations of recombinant AAV virions substantially free of empty capsids
GB0613925D0 (en) 2006-07-13 2006-08-23 Unilever Plc Improvements relating to nanodispersions
ES2751999T3 (es) 2008-01-29 2020-04-02 Applied Genetic Tech Corporation Producción recombinante de virus adeno-asociados usando células BHK en suspensión
WO2010114948A2 (en) 2009-04-02 2010-10-07 University Of Florida Research Foundation, Inc. An inducible system for highly efficient production of recombinant adeno-associated virus (raav) vectors
US20120172419A1 (en) * 2009-09-15 2012-07-05 Medical College Of Wisconsin Research Foundation Inc. Reagents and methods for modulating cone photoreceptor activity
US8692332B2 (en) 2010-01-14 2014-04-08 United Microelectronics Corp. Strained-silicon transistor and method of making the same
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
EP2675484B1 (en) 2011-02-14 2018-05-30 The Children's Hospital of Philadelphia Improved aav8 vector with enhanced functional activity and methods of use thereof
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.
EP3800191B1 (en) 2014-03-17 2025-08-20 Adverum Biotechnologies, Inc. Compositions and methods for enhanced gene expression in cone cells
EP3600446A1 (en) * 2017-03-21 2020-02-05 Michalakis, Stylianos Gene therapy for the treatment of cngb1-linked retinitis pigmentosa
US20240263191A1 (en) * 2017-12-21 2024-08-08 Universita Di Napoli Federico Ii Ectopically expressed transcription factors and uses thereof
US20190345516A1 (en) * 2018-03-12 2019-11-14 Musc Foundation For Research Development Methods for treating eye disease
JP7663504B2 (ja) * 2019-03-08 2025-04-16 ユニベルシテ パリ-サクレー 遺伝子置換による希少な眼疾患のための改善された治療方法

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