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PRODYGY Trial with SPVN06 for retinitis pigmentosa","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I\/ Phase II","country":"FRANCE","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"January 2024","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"SparingVision"}]
Find Clinical Drug Development Pipelines & Deals | PipelineProspector
Response to COVID-19 pandemic anticipated to cause interruptions in certain clinical trial operations; updated interim data readout timing to be provided at later date.
First inhaled mRNA therapeutic remains on track to report results from its ongoing multiple-ascending dose (MAD) portion of Phase 1/2 clinical trial in the third quarter of 2020.
SPVN06 is a proprietary, mutation-agnostic, AAV gene therapy approach comprised of one neurotrophic factor (RdCVF) and one enzyme reducing oxidative stress. It is under phase 1/2 clinical development for the treatment of Retinitis Pigmentosa.
ATA-200, is the gene therapy that delivers a normal copy of the gene for production of γ-sarcoglycan, which is investigated for limb-girdle muscular dystrophy Type 2C/R5 (LGMD2C/R5).
SPVN06 is a proprietary, mutation-agnostic, AAV gene therapy approach comprised of one neurotrophic factor and one enzyme reducing oxidative stress used for the treatment of for the treatment of retinitis pigmentosa.
CTx-PDE6b is a clinical stage AAV5-based gene therapy for the treatment of retinitis pigmentosa (RP) caused by bi-allelic mutations of the PDE6b gene. There are currently no approved treatments for PDE6b RP.
GS030 delivered via a single intravitreal injection, introduces a gene encoding for a light-sensitive protein (ChrimsonR-tdT) into retinal ganglion cells, making them responsive to light and bypassing photoreceptors killed off by diseases such as retinitis pigmentosa (RP).
Lysogene has dosed the first patient in France with LYS-GM101 investigational gene therapy at the Armand Trousseau Hospital (AP-HP). This is the third patient treated with LYS-GM101 in the global adaptative-design clinical trial in children with GM1 gangliosidosis.
Lead Product(s):
AAVrh.10 Expressing Beta-galactosidase
Fast Track Designation recieved for DYN101,an investigational antisense product candidate designed to reduce the expression of dynamin 2 protein for the treatment of Myotubular and Centronuclear Myopathies.
LYS-GM101 investigational gene therapy an ‘Innovation Passport’ for the treatment of GM1 gangliosidosis under the United Kingdom’s (UK) Innovative Licensing and Access Pathway (ILAP). LYS-GM101 is currently in clinical development.
Lead Product(s):
AAVrh.10 Expressing beta-galactosidase