ViGeneron GmbH | MedPath

ViGeneron's VG901 Receives FDA Rare Pediatric Disease Designation and Advances to Dose Escalation in Phase 1b Trial for Retinitis Pigmentosa

4 months ago

• The FDA granted Rare Pediatric Disease Designation to ViGeneron's VG901, a gene therapy for retinitis pigmentosa caused by CNGA1 gene mutations, highlighting the urgent need for new treatments. • VG901 leverages ViGeneron's vgAAV capsid to deliver the functional CNGA1 gene to retinal photoreceptor cells via intravitreal injection, offering a less invasive approach. • The Data Safety Monitoring Board (DSMB) has unanimously approved dose escalation in the ongoing Phase 1b clinical trial, indicating promising early safety results for VG901. • ViGeneron's VG801 receives FDA IND clearance for mRNA trans-splicing gene therapy to treat Stargardt disease and other retinal diseases associated with mutations in the ABCA4 gene.

ViGeneron Doses First Patient in Phase 1b Clinical Trial of VG901 for Retinitis Pigmentosa Treatment

a year ago

ViGeneron GmbH has dosed the first patient in its Phase 1b clinical trial for VG901, a gene therapy aimed at treating retinitis pigmentosa caused by CNGA1 gene mutations. The trial evaluates the safety and efficacy of intravitreal VG901 injections, with results expected by the end of 2025.