In the recently published Molecular Therapy Advances paper, the InGenuiTy F/HN pseuotyped lentviral platform was assessed in non-human primates. There were no safety issues noted over the seven day period of the study. Airway epithelial cell transduction efficiency, as assessed by GFP expression, was ~10% at the dose tested. The study extends earlier findings of rSIV.F/HN-based in vivo gene transfer in mice to NHPs, demonstrating transduction efficiency without relevant toxicity.
These data provide additional confidence for the forward clinical development of our first and potential best-in-class neonatal surfactant protein deficiency candidates AVG-002 and AVG-003 targeting SP-B and ABCA3 respectively. Additional data will be presented in the months ahead.
Notably, AVG-002 already has PRDD and ODD status with a contingent Priority Review Voucher. AlveoGene will submit AVG-003 data to the FDA to achieve PRDD and ODD status later in 2026. This will result in AlveoGene having 2 contingent PRVs, with their value currently trading at approximately $200m per voucher.
AlveoGene plans to undertake accelerated clinical development for both candidates in parallel, leveraging new and emerging regulatory pathways for rare and ultra rare disease, and enabling therapeutic solutions for these areas of unmet clinical needs.