FDA clears investigational new drug application for Stargardt disease gene therapy
Key takeaways:
- A phase 1/2 trial will assess safety, tolerability and preliminary efficacy for VG801.
- The FDA also selected ViGeneron for its Rare Disease Endpoint Advancement Pilot Program.
The FDA cleared the investigational new drug application for a phase 1/2 study investigating VG801, a treatment for Stargardt disease and other retinal diseases associated with mutations in the ABCA4 gene.
According to a press release from ViGeneron GmbH, this is the first FDA IND clearance for an mRNA trans-splicing gene therapy. VG801 utilizes two gene therapy delivery technology platforms: REVeRT, designed to reconstruct ABCA4 messenger RNA through transduction with dual AAV vectors, and a vgAAV capsid designed to spur widespread retinal transduction.
The phase 1/2 multicenter, open-label, dose-escalation clinical trial will enroll patients globally and evaluate the safety, tolerability and preliminary efficacy of the gene therapy.
Additionally, The FDA selected the program for its Rare Disease Endpoint Advancement Pilot Program, which will grant ViGeneron more opportunities to collaborate with the FDA throughout the development process.
“By delivering full-length functional ABCA4 gene, VG801 has the therapeutic potential to address the genetic root cause of Stargardt disease and other ABCA4-linked retinal dystrophies,” Caroline Man Xu, MBA, PhD, co-founder and CEO of ViGeneron, said in the release. “The VG801 IND clearance, together with our ongoing VG901 clinical trial for retinitis pigmentosa, expands our clinical-stage pipeline to two programs and underscores our commitment to advancing next-generation gene therapies with novel technologies to address significant unmet medical needs.”
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