Retinitis pigmentosa gene therapy wins European orphan drug status

GenSight Biologics has received orphan drug designation from the European Medicines Agency for their experimental retinitis pigmentosa treatment, GS030.

The company also received Advanced Therapy Medicinal Product classification, which is conferred to groundbreaking innovative opportunities for the treatment of conditions with high unmet needs.

“We are very excited that GS030 has received both Orphan Drug Designation and Advanced Therapy Medicinal Product classification in Europe, recognizing the urgent and unmet medical need for a safe and effective treatment for retinitis pigmentosa patients, and the potential of GS030 to address it, said GenSight CEO Jean-Philippe Combal, Ph.D. “This will allow us to optimize future steps to further advance GS030 toward regulatory approval.”

GS030 is a gene therapy product candidate, currently in preclinical development, that utilizes a modified AAV2 (adeno-associated virus) vector to introduce a light-sensitive rhodopsin protein into the retina of patients with dysfunctional photoreceptors. Once eye cells have been successfully transduced, the patient will wear biomimetic goggles with a real-time sensor that converts images into LED light-amplified signals (optimized for the engineered rhodopsin), which are reflected to the macula.

GenSight has planned a Good Laboratory Practices regulatory toxicity study in primates to commence in September 2016. Depending on these results, a phase I/II clinical trial in retinitis pigmentosa patients has been projected for mid-2017.