PYC Therapeutics Limiteds fourth drug candidate for Phelan McDermid syndrome is effective in human brain cells.

PYC Therapeutics (ASX: PYC) One of the company's main initiatives is a drug discovery program to address Phelan McDermid Syndrome (PMS), a severe neurodevelopmental disorder. PMS affects approximately 1 in every 10,000 people, leading to challenges in brain development and function, resulting in a range of intellectual and physical disabilities. Currently, there are no approved treatments targeting the root cause of PMS.PYC has announced a significant milestone in its efforts to address PMS. The company has successfully restored the deficient protein responsible for PMS in brain cells (neurons) derived from a patient with PMS. This achievement demonstrates the potential effectiveness of PYC's approach, signaling promising progress before the start of human trials.

Furthermore, the company has generated this data using two different RNA therapy chemistries, one of which has shown clinical benefits in patients with neuronal disorders. This positions PYC to progress toward initial human studies with confidence that RNA therapy holds great promise for providing benefits to PMS patients and their families.

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