PLX-300

About Polaryx Therapeutics

Polaryx Therapeutics is a clinical-stage biotech company focused on developing disease-modifying treatments for rare pediatric lysosomal storage disorders (LSDs). Its core achievement is advancing PLX-200, an autophagy-inducing small molecule, into a Phase 2 basket trial (SOTERIA) for multiple LSDs, supported by multiple FDA designations including Orphan Drug, Fast Track, and Rare Pediatric Disease. The company's strategy is to build a diversified, multi-modal pipeline—encompassing small molecules and gene therapy—to target both the genetic and downstream pathological features of these diseases, aiming to establish a new standard of care.

Therapeutic Areas