Develops small-molecule and gene-therapy candidates for patients with rare pediatric lysosomal storage disorders.
10-K for FY2025, filed Mar 24, 2026We are a clinical-stage biotechnology company committed to the discovery, development, and commercialization of novel, disease-modifying…View ↗
PLX-200 (gemfibrozil), our most advanced drug candidate, is an oral small molecule for the treatment of LSDs. PLX-200 is a repurposed…View ↗
PLX-300 (cinnamic acid) is a novel, oral small molecule therapy in IND-enabling studies for the treatment of LSDs. PLX-300 is an…View ↗
PLX-100 is a preclinical stage orally administrable combination therapy comprised of our proliferator-activated receptor alpha (“PPARα”)…View ↗
PLX-400 is a preclinical stage novel gene therapy being developed for the treatment of LSDs. We are exploring PLX-400 as a monotherapy or…View ↗
No revenue reported yet.
10-K for FY2025, filed Mar 24, 2026
Competes with Tern Therapeutics, THX Pharma, IntraBio, and Forge Biologics
10-K for FY2025, filed Mar 24, 2026With respect to products and product candidates in the treatment of the 13 sub-types of NCLs, there is currently one established standard…View ↗
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