Oxford BioMedica's RetinoStat achieves 'favourable safety profile' in tests

AIM-listed pharmaceutical Oxford BioMedica’s RetinoStat, the first ocular lentiviral gene therapy to be administered in humans, demonstrated a “favourable safety profile with no serious adverse events” in its phase one study.

Results of the study, which were previously published in the peer-reviewed Human Gene Therapy journal in May, found from RetinoStat that therapeutic transgenes was directly measured in patient humor samples and showed a dose response which was stable and persistent in all patients.

Chief executive John Dawson, said: "Like the ProSavin trial before it, the RetinoStat first-in-man study was a clinical trial of 'firsts': the first ever trial to directly administer a lentiviral vector-based product to the eye, the first directly administered lentiviral vector trial in the US, the first direct measurement of an ocular gene therapy transgene during a study and the first reporting of data showing direct demonstration of long-lasting expression of an ocular gene therapy in human subjects.

"These peer-reviewed published results further validate the ground-breaking utility of our LentiVector delivery platform for the treatment of chronic disease."

The phase one study, conducted on 21 participants with highly fibrotic retinas, was to evaluate the safety and tolerability of the therapy for the treatment of severe wet age-related muscular degencration following a single subretinal injection, the first time a lentiviral based vector had been administered to the eye.

The company, which spun out from the University of Oxford in 1995, said it was currently evaluating how to progress clinical development for RetinoStat.

Shares in Oxford BioMedica were up 1.27% to 3.19p at 1422 BST.