from Today's $tockhead. PYC Therapeutics (ASX:PYC) PYC also...

from Today's $tockhead.
PYC Therapeutics (ASXYC)
PYC also received an Orphan Drug Designation (ODD) from the US FDA in late May for its drug candidate, PYC-001, aimed at treating OPA1-associated vision loss.
PYC-001 is said to be a significant advancement in precision therapy. The drug was designed to restore the expression of the OPA1 gene to levels necessary for optimal retinal function.
Utilising the company’s proprietary drug-delivery technology, PYC has tackled a major hurdle for RNA drugs by ensuring effective delivery to the targeted cells affected by ADOA (Autosomal Dominant Optic Atrophy).
ADOA is a progressive and irreversible blinding eye disease. It affects approximately 1 in every 35,000 people representing a market size of ~$2 billion per annum.
ADOA is caused by a mutation in one copy of the OPA1 gene, which results in inadequate levels of OPA1 gene expression, impacting the normal function of retinal ganglion cells.