NEW YORK – Restore Vision on Thursday said it has treated the first patient in a Phase I/II trial with RV-001, the company's lead gene therapy that it is developing for retinitis pigmentosa (RP).
The patient received the gene therapy at Keio University Hospital in Tokyo. Restore Vision, also based in Tokyo, spun out of Keio University in 2016.
RV-001 uses an adeno-associated virus vector to deliver a functional copy of a gene the company engineered to encode chimeric rhodopsin. Restore Vision is betting RV-001 can expand a patient's light sensitivity by activating G proteins to reestablish light activation in retinal interneurons regardless of the genetic mutation causing retinitis pigmentosa.
"Our 'chimeric rhodopsin' approach has broad potential to alter light sensitivity across multiple subtypes of RP patients," Restore Vision CEO Yusaku Katada said in a statement.
Restore Vision is enrolling patients in the trial with advanced retinitis pigmentosa, regardless of genotype, who have limited or no vision. The company plans to monitor safety and exploratory efficacy endpoints and track whether the gene therapy improves patients' light sensitivity and visual function.
So far, the starting dose of RV-001 has been well tolerated in this initial patient, according to Restore Vision.