Ann: Annual Report to shareholders, page-13

As PYC is about to release rabbit toxicology results for its lead PPMO asset in Retinitis Pigmentosa type 11, the spotlight has once again fallen on the safety profile of gene therapies.

Applied Genetics Technologies Corporation (AGTC), a Florida-based biotech developing AAV vector-based gene therapies for the treatment of inherited retinal diseases, has reported suspected unexpected serious adverse events in one of its ocular therapy trials.

Four paediatric patients who received a high dose in the company’s Phase 1/2 trial in achromatopsia were reported to have experienced significant inflammation in both segments of the eye approximately one month after dosing.

Nonetheless, AGTC has made the decision to continue clinical development of the therapy, one of five it has under development for inherited retinal diseases. Its lead pipeline product is a therapy for X-linked Retinitis Pigmentosa (XLRP). Of the 29 patients in its completed Phase 1/2 XLRP trial, 4 patients suffered retinal detachment from the gene therapy’s subretinal delivery procedure.

The company also has two neurodegenerative candidate therapies and one in otology. Its current market cap is US$138m, a third of the value of its 52 week high.

https://www.fiercebiotech.com/biotech/kids-suffer-serious-adverse-reactions-agtc-gene-therapy-trial

https://ir.agtc.com/events-and-presentations