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Biotechnology company Pyc Therapeutics Ltd (ASX:PYC) has announced that it has been able to restore a deficient protein causing neurodevelopmental disorder Phelan McDermid Syndrome (or PMS), as part of Pyc’s wider drug development program which will soon involve human trials.
Around one in 10,000 people is affected by PMS, which is characteried by intellectual disability, absent or delayed speech and behavioural issues, with the condition being caused by the loss of one functioning copy of the SHANK3 gene.
This in turn results in insufficient SHANK3 protein being expressed in the brain.
But Pyc – through its recent work – has been able to restore the missing SHANK3 protein in neurons derived from a patient with the condition using a ribonucleic acid (RNA)-drug candidate, and this takes the company through the preparation studies for human trials, with the latter set to begin next year.
CEO Dr Rohan Hockings said the results set Pyc Therapeutics up strongly for its future investigations into this genetic condition.
“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,” he 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.”
The market was also happy with the news, and at 11:08 AEST, Pyc shares were trading at 10.5 cents, a rise of 4.99 since the market opened.
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