A team of researchers at Bascom Palmer Eye Institute has begun a phase 2 clinical trial of a novel gene therapy designed to treat choroideremia, according to press releases from Bascom Palmer and NightstaRx.
AAV2-REP1 comprises a viral vector adeno-associated virus (AAV) designed to deliver a copy of the Rab escort protein 1 (REP1) gene into the eye.
Choroideremia is a rare, progressive degenerative disorder of the retina and choroid caused by a defect in the CHM gene, which produces a faulty protein in the retina. Choroideremia affects one in 50,000 people, the majority being young men, and causes a gradual loss of night vision and peripheral vision, leading to complete loss of central vision.
AAV2-REP1 is being provided by NightstaRx, a privately owned biopharmaceutical company.
According to the release, AAV2-REP1 is injected under the retina using a technique approved by the FDA expressly for the trial.
The trial is designed to gauge the safety and efficacy of AAV2-REP1 and will include six patients, the release said.
“We are extremely excited and optimistic about the success of this gene therapy trial,” lead investigator Byron L. Lam, MD, said in the release. “We hope the knowledge gained will help patients with other retinal diseases.”
The clinical trial is being sponsored by Bascom Palmer and several donors. The first clinical trial of AAV2-REP1 to treat choroideremia was conducted at the University of Oxford. Lead Oxford researcher Robert MacLaren, FRCOphth, FRCS, is collaborating with Lam and the team at Bascom Palmer.
In related news, NightstaRx announced a $35 million financing round led by New Enterprise Associates, a venture capital firm. Founding investor Syncona, an independent subsidiary of the Wellcome Trust, is also participating in the venture, according to a separate press release from NightstaRx.
NightstaRx expanded its pipeline with five program licenses from the University of Oxford through its technology commercialization subsidiary, Isis Innovation.
The funding will be used for clinical development of AAV2-REP1 and other gene therapy programs, the release said.