AAV mediated RPE65 and Survivin based dual gene therapy
TRL Level:
5
Sustained vision rescue for LCA2 by combination of RPE65/ Survivin
Vision
Develop durable and accessible gene therapies for inherited blindness disorders worldwide.
Problem
LCA2 causes severe childhood blindness. Emerging studies show declining long-term vision rescue and progressive retinal degeneration after gene therapy.
Solution
Optimized dual-AAV therapy (RPE65 and Survivin) restores long term vision by reducing retinal cell death.
Potential
Can be used as a platform technology to be used for treatment of several other inherited blindness disorders (e.g. XLRP, Usher syndrome).
Target Beneficiaries
Children and adults with LCA2 and retinal degeneration, ophthalmology hospitals and retinal gene therapy programs
Current Users
Preclinical validation completed in rd12 mouse model with sustained efficacy for 24 weeks, Technology readiness level 5 (TRL5).
