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Polaryx Therapeutics Marks Rare Disease Day, Reaffirming Commitment to Patients with Rare Pediatric Lysosomal Storage Disorders
PARAMUS, NJ, Feb. 27, 2026 (GLOBE NEWSWIRE) -- Polaryx Therapeutics (Nasdaq: PLYX), a clinical-stage biotechnology company developing novel, disease-modifying
About this update from Polaryx Therapeutics, Inc.
[{"type":"text","content":"PARAMUS, NJ, Feb. 27, 2026 (GLOBE NEWSWIRE) -- Polaryx Therapeutics (Nasdaq: PLYX), a clinical-stage biotechnology company developing novel, disease-modifying therapies for rare, pediatric lysosomal storage disorders (“LSDs”), joins the global rare disease community in raising awareness and supporting patients, families, caregivers, and healthcare providers. “On Rare Disease Day, Polaryx proudly stands alongside the rare disease community - patients, families, caregivers, researchers, and advocacy partners to underscore the urgent need for new and transformative treatments,” said Alex Yang, Chairman and Chief Executive Officer of Polaryx Therapeutics, Inc. “We are deeply committed to raising awareness of rare pediatric lysosomal storage disorders and developing disease-modifying, family-friendly therapies across multiple LSDs.” Polaryx continues to pursue its mission of addressing significant unmet medical needs in rare pediatric disorders by advancing the launch of SOTERIA, a phase 2, open-label, single-arm trial designed to evaluate the safety, tolerability, and clinical activity of Polaryx’s lead candidate, PLX-200, across four rare LSDs, including CLN2, CLN3, Krabbe disease and Sandhoff disease. LSDs are a heterogeneous group of more than 50 inherited rare metabolic diseases caused by mutations in genes encoding lysosomal enzymes or associated proteins. These mutations result in the accumulation of undegraded substrates within lysosomes, leading to cellular dysfunction, chronic inflammation, and cell apoptosis. LSDs often manifest in infancy or early childhood and are associated with severe clinical outcomes, including developmental regression, seizures, blindness, motor impairment, and premature death. LSDs affect approximately 1 in 5,000 births. Among the indications, the SOTERIA trial specifically targets rare subtypes, including: CLN2 disease, also known as late infantile neuronal ceroid lipofuscinosis (“LINCL”), is associated with mutations in the CLN2 gene, which encodes lysosomal tripeptidyl-tripeptidase I (“TPP1”). This mutation in the CLN2 gene results in a deficiency and/or loss of function of the TPP1 protein that leads to intralysosomal accumulation of auto-fluorescent lipopigments known as ceroid-lipofuscin. Globally, the classical form of CLN2 disease has a prevalence of 0.6 to 0.7 per million inhabitants. CLN2...