PYC Therapeutics: Advancing a Breakthrough for Phelan-McDermid Syndrome

PYC Therapeutics (ASX: PYC) is an Australian clinical-stage biotech company developing precision RNA therapies for genetic disorders. Its newest therapeutic candidate, PYC-002, is designed to address Phelan-McDermid Syndrome (PMS)—a rare brain development disorder stemming from reduced activity of the SHANK3 gene. PMS affects approximately 1 in 10,000 children and currently lacks effective treatments. PYC-002 has shown promising results in vitro by restoring SHANK3 protein levels and improving neuron structure, synapse density, and neuronal activity in PMS patient-derived cells. In animal studies, a single low dose of PYC-002 successfully elevated SHANK3 expression across multiple brain regions, including the hippocampus and prefrontal cortex. The therapy is administered intrathecally (via lumbar puncture) to bypass the blood-brain barrier. With a strong preclinical safety and efficacy profile, PYC-002 is expected to enter human trials in 2026, potentially offering the first disease-modifying treatment for PMS.

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