Clinical-stage biotechnology company PYC Therapeutics (ASX:PYC) has announced that the US FDA has designated one of its assets an orphan drug.
The candidate, VP-001, is being developed for patients with Retinitis Pigmentosa type 11 (RP11), a blinding eye disease.
The orphan designation provides tax credits for qualified clinical trials, exemptions from some regulatory fees and the potential for seven years of market exclusivity post-approval.
RP11 is caused by a mutation in one copy of the PRPF31 gene, which leads to protein insufficiency in photoreceptor and Retinal Pigment Epithelial (RPE) cells.
VP-001 increases expression of PRPF31 back to wild-type levels in RP11 patient-derived retinal organoids and iPSC-RPE4 and then into the specific cell type in the eye that is affected by the disease to provide a human model of the disease-affected eye outside of a human.