PYC Therapeutics (ASX:PYC) has announced that the first patient with Autosomal Dominant Optic Atrophy (ADOA) has received their first dose of PYC-001.
ADOA is a blinding eye disease that begins in childhood and ultimately leads to legal blindness in middle age in most patients. The disease affects around one in every 35,000 people and is caused by insufficient expression of the OPA1 gene in the retina.
There are currently no treatment options available for patients with ADOA.
PYC-001 is a precision therapy that aims to restore the expression of the OPA1 gene to levels required for normal retina function.
The first ADOA patient enrolled in its single ascending dose study of PYC-001 received a 3 µg dose of PYC-001 in one eye via intravitreal administration. The company said two more patients will be enrolled in this initial dose cohort and are expected to receive their first dose of PYC-001 soon.
The company said the study is scheduled to run for around 18 months and will provide initial insights into the safety, tolerability, and efficacy profile of this drug candidate in the first half of 2025. It will inform the design of a multiple ascending dose study scheduled to commence next year and inform the design of a registrational trial expected to commence in 2026.