PYC Therapeutics combining two complementary platform technologies: - RNA drug design capabilities; and - a proprietary drug delivery technology. Together they are being developed to create a new generation of RNA therapeutics to change the lives of patients with genetic diseases. PYC is adding a third investigational drug program for the treatment of Phelan McDermid Syndrome (PMS) to its pipeline.

PMS is a neurodevelopmental disorder characterised by developmental and speech delays, behavioural problems and compromised ability to perceive pain and regulate body temperature. The creation of a disease-modifying therapy for patients with PMS represents an area of major unmet patient need. Effective treatment of the underlying cause of this disorder would represent a life-changing paradigm for PMS patients and their families.

Consistent with PYC's strategy, PMS is a monogenic disease in which the underlying cause is insufficient expression of the SHANK3 gene in the target cell (neurons) in the brain. The new pipeline addition represents PYC's first program in the Central Nervous System (CNS) - demonstrating the scalability of the Company's platform technology into target tissues beyond the eye. The underlying cause of PMS in the majority of patients is a deletion or mutation affecting one copy of the SHANK3 gene, causing a ~50% decrease in expression of the SHANK3 protein.

It is this deficiency of SHANK3 expression in neuronal cells in the brain that causes PMS. PYC has designed and validated an RNA therapeutic capable of increasing SHANK3 expression in cells by ~2-3 fold. The extent of protein upregulation observed in these in vitro studies is sufficient to correct the underlying SHANK3 protein deficiency that causes PMS.