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Clinical-stage biotechnology company PYC Therapeutics (ASX:PYC) has achieved promising results from a fourth drug discovery program addressing the underlying cause of a severe neurodevelopmental disorder known as Phelan- McDermid Syndrome (PMS)

PMS is a genetic disorder affecting one in every 10,000 people that affects brain development and function and results in a range of intellectual and physical disabilities. There are currently no treatments available for patients with PMS that address the underlying cause of the disorder.

PYC has been able to restore the deficient protein that causes PMS in the brain cells in which the disorder occurs (known as neurons). The neurons in which these results have been achieved were derived from a patient with PMS – demonstrating the utility of PYC’s approach in PMS prior to the initiation of human trials.

“This is a big step forward in this body of work - this is the data that the clinicians have been asking us to generate before we push into the clinic” CEO Dr. Rohan Hockings said.

“Importantly, we have been able to generate the data with two different chemistries of RNA therapy, one of which has already demonstrated clinical benefit in patients with disorders occurring in neurons. This gives us clear line of sight into first in human studies where we believe an RNA therapy offers the greatest potential benefit to PMS patients and their families.”

  • PMS is characterised by intellectual disability, absent or severely delayed speech and behavioural issues - there are no treatments available for this disorder
  • It is caused by a loss of one functional copy of the SHANK3 gene resulting in insufficient SHANK3 protein expression in brain cells known as neurons
  • PYC has been able to restore the missing SHANK3 protein that causes PMS in neurons derived from a PMS patient

The company will now progress towards initiating the studies required to enter human trials (anticipated to commence in 2025).

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