Periodic Reporting for period 1 - GeneVision (Developing a cure for retinitis pigmentosa due to Usher syndrome)
Période du rapport: 2017-04-01 au 2018-09-30
GeneVision objective was to support the clinical development of a novel retinal gene therapy platform based on dual adeno-associated viral (AAV) vectors which has been developed by the PI within his RetGeneTx ERC Consolidator project. GeneVision aims treating the blinding retinitis pigmentosa presented by patients with Usher syndrome type IB (USHIB) by intraocular administrations of dual AAV.
To support dual AAV clinical development GeneVision:
-is defining the dual AAV dose-response in the retina of a mouse model of USHIB: this study is about to be completed and confirms the efficacy of the high dose proposed for a phase I/II clinical trial.
- consolidated the GeneVision IP position and conducted a thorough analysis of competition and company valuation, which lead to discussions with a French venture.
-is evaluating potential partnerships with the above mentioned venture capitalists, who are interested in further developing dual AAV in the retina.
Finally, during the GeneVision project, the PI has applied and obtained as coordinator funding from the EU under its H2020 program to support a phase I/II clinical trial to test the safety and efficacy of dual AAV in the retina of USHIB patients.
In conclusion the work laid by GeneVision allowed to proceed towards the clinical development of dual AAV in the retina and is laying the bases for its future commercialization.
To support dual AAV clinical development GeneVision:
-is defining the dual AAV dose-response in the retina of a mouse model of USHIB: this study is about to be completed and confirms the efficacy of the high dose proposed for a phase I/II clinical trial.
- consolidated the GeneVision IP position and conducted a thorough analysis of competition and company valuation, which lead to discussions with a French venture.
-is evaluating potential partnerships with the above mentioned venture capitalists, who are interested in further developing dual AAV in the retina.
Finally, during the GeneVision project, the PI has applied and obtained as coordinator funding from the EU under its H2020 program to support a phase I/II clinical trial to test the safety and efficacy of dual AAV in the retina of USHIB patients.
In conclusion the work laid by GeneVision allowed to proceed towards the clinical development of dual AAV in the retina and is laying the bases for its future commercialization.