Patients with X-linked retinoschisis who underwent treatment with adeno-associated virus-based gene therapy developed no treatment-related serious adverse events through 12 months, according to interim safety data from a phase 1/2 trial.
Safety data for 12 of 27 patients treated with adeno-associated virus-based gene therapy (Applied Genetic Technologies Corporation) showed that the treatment is generally well tolerated through 1 year, according to a presentation by Mark E. Pennesi, MD, PhD, at the Macula Society annual meeting in Singapore.
Inflammation was the most common adverse event, typically occurring 3 to 4 weeks after treatment. If treatment for inflammation was necessary, topical and/or oral steroids were administered, according to the presentation.
Two patients developed clinically significant leukocytosis, but it was attributed to steroid treatment. Another patient developed leukocytoclastic vasculitis, but it was unclear if it was related to the study product and may have been a reaction to omeprazole.
The next step will be continued enrollment in a dose expansion phase at high-dose levels and continued evaluation of safety, according to the presentation. – by Robert Linnehan
Pennesi ME. AGTC-RS1-001 phase 1/2 intravitreal gene therapy interim safety results for X-linked retinoschisis. Presented at: Macula Society annual meeting; June 7-10, 2017; Singapore.
Disclosures: Pennesi reports he is a consultant for Ionis Pharmaceuticals, Sparks Therapeutics, ProQR, Editas and AGTC; receives clinical trial support from Sanofi, NightstaRx and AGTC; is on the scientific advisory board for Nacuity Pharmaceuticals; and receives grant support from FFB.