This project builds a platform biotech that upgrades enzyme replacement therapy by solving its main bottleneck: poor intracellular delivery and high immunogenicity. We use proprietary lipid nanoparticles to transport enzymes efficiently into target cells, increasing efficacy while reducing dose, infusion frequency, and immune side effects. This enables best-in-class therapies for Pompe and other lysosomal storage diseases. Commercially, the same delivery platform can be reused across multiple enzymes, creating a capital-efficient pipeline. The primary business model is to advance 1–2 lead assets to early clinical proof-of-concept and out-license them to rare-disease pharma partners for upfront payments, milestones, and royalties in the $9B LSD market.
