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First Patient Dosed in Phase Ib Trial Studying ViGeneron's Retinitis Pigmentosa Gene Therapy

NEW YORK – ViGeneron on Wednesday said it has dosed the first patient in a Phase Ib clinical trial of its investigational gene therapy for autosomal recessive retinitis pigmentosa, an eye disorder that leads to progressive vision loss.

The Munich, Germany-based company is developing VG901 specifically as a treatment for forms of the inherited retinal disease due to mutations in the CNGA1 gene, which is estimated to cause up to 8 percent of autosomal recessive cases. The gene therapy is designed to deliver a functional copy of CNGA1 to retinal photoceptor target cells and is administered via intravitreal injection using the company's vgAAV vector platform.

Investigators in the open-label, single-arm, dose-escalation Phase Ib trial, which will enroll about six patients, are assessing safety, tolerability, and preliminary efficacy of the gene therapy.

"VG901 offers a therapeutic potential in addressing the genetic root cause for patients with retinitis pigmentosa affected by CNGA1 mutations," Katarina Stingl, head of the clinic for hereditary retinal degenerations in the center for ophthalmology at the University of Tübingen in Germany and principal investigator for the trial, said in a statement. "We are excited to learn about the potential of this novel therapy and hope to make a meaningful difference to patients' lives."