PYC Therapeutics (ASX: PYC) has announced that the first patient with Autosomal Dominant Optic Atrophy (ADOA) has received the initial dose of its investigational treatment, PYC-001. This marks a significant step in the development of a potential therapy for ADOA, a rare genetic eye disease that causes progressive vision loss.
ADOA, which affects approximately 1 in every 35,000 individuals, typically begins in childhood and leads to legal blindness by middle age. The condition is caused by mutations in the OPA1 gene, resulting in insufficient expression of the gene in the retina, crucial for normal visual function. Currently, there are no approved treatments for ADOA.
PYC-001 is a precision medicine designed to restore the OPA1 gene’s expression to levels necessary for proper retinal function. The first patient enrolled in the company’s ongoing single ascending dose study received a 3 μg dose of PYC-001 through intravitreal (eye) injection. Two additional patients are expected to join this initial cohort shortly.
The study, which will run for approximately 18 months, aims to provide critical data on the safety, tolerability, and potential efficacy of PYC-001. Early findings are expected in the first half of 2025. The insights gained from this study will help shape the design of a multiple ascending dose study slated to begin next year, with the goal of progressing toward a registrational trial anticipated to begin in 2026.
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