Polaryx Therapeutics' pipeline spans both small molecule and gene therapy modalities, addressing lysosomal storage disorders, a group of more than 50 inherited metabolic diseases. Its most advanced candidate, PLX-200, is a repurposed oral small molecule for these disorders, pursued through a 505(b)(2) regulatory pathway and administered as an oral solution. PLX-400 is a preclinical gene therapy candidate designed to deliver lysosomal genes in adeno-associated viral vectors.
Orally available small molecule therapies aim to promote lysosome biogenesis via PPARα-dependent TFEB upregulation, and PLX-200 is planned for an IND-approved Phase 2 SOTERIA basket trial covering CLN2 and CLN3 subtypes of NCL, Krabbe disease, and Sandhoff disease. Preclinical programs include PLX-100, an orally administrable combination of PLX-200 and vitamin A, and PLX-300, an orally administrable combination therapy of unsaturated carboxylic acid and a PPARα agonist. Polaryx Therapeutics focuses on patients and families living with lysosomal storage disorders.
Quick Facts
When was Polaryx Therapeutics' last funding round?
Polaryx Therapeutics' most recent funding round was for $10M (USD) in May 2026.
How much disclosed funding has Polaryx Therapeutics raised?
As of May 28, 2026, Polaryx Therapeutics has disclosed funding of $10M (USD).
