• FDA grants Rare Pediatric Disease (RPD) designation to PYC's VP-001 drug
• VP-001 targets a blinding eye disease of childhood called Retinitis Pigmentosa type 11
• PYC preparing to initiate a potentially registrational trial for VP-001 in 2025

PYC Therapeutics, a clinical-stage biotechnology company, has announced that the US Food and Drug Administration (FDA) has granted Rare Pediatric Disease (RPD) designation for its VP-001 drug development program. VP-001 is an investigational drug candidate that addresses the underlying cause of a blinding eye disease of childhood known as Retinitis Pigmentosa type 11 (RP11). The company is currently progressing VP-001 through phase 1/2 studies and is preparing to initiate a potentially registrational trial for the drug in 2025. The RPD program aims to incentivise drug development for serious and rare diseases affecting children. PYC Therapeutics is a clinical-stage biotechnology company focused on creating a new generation of RNA therapies to change the lives of patients with genetic diseases. The company's drug development programs target monogenic diseases, which have the highest likelihood of success in clinical development.

PYC Therapeutics is preparing to initiate a potentially registrational trial for its VP-001 drug in 2025, which targets a blinding eye disease of childhood called Retinitis Pigmentosa type 11. The company is also progressing clinical trials for other drug development programs targeting Autosomal Dominant Optic Atrophy, Autosomal Dominant Polycystic Kidney Disease, and Phelan McDermid Syndrome, with human safety and efficacy data anticipated in 2025 and 2026.