Eye On the Cure Research News

Mar 31, 2020

Horama Acquires Licensing Rights to CRB1 Gene Therapy

Company in France targets a clinical trial in 2023

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Horama, a gene therapy developer for rare eye diseases, has obtained exclusive licensing rights to the CRB1 gene therapy program at Leiden University Medical Center (LUMC) in the Netherlands.

Based on current development timelines and regulatory authorization, Horama expects to launch a Phase 1/2 clinical trial for the CRB1 gene therapy in 2023.

We are very pleased to see that the development of gene therapy for the treatment of diseases caused by mutations in the CRB1 gene has attracted commercial backing to advance it toward a clinical trial. LCA caused by CRB1 mutations is a challenging, unmet need.

Brian Mansfield, PhD, executive vice president of research and interim chief scientific officer, Foundation Fighting Blindness

Mutations in CRB1 cause Leber congenital amaurosis (LCA), a retinal disease causing severe vision loss in children, and retinitis pigmentosa, which also causes significant vision loss from retinal degeneration.

The Foundation Fighting Blindness funded Jan Wijnholds, PhD, LUMC, for his extensive CRB1research.

“We are very pleased to see that the development of gene therapy for the treatment of diseases caused by mutations in the CRB1 gene has attracted commercial backing to advance it toward a clinical trial,” says Brian Mansfield, PhD, executive vice president of research and interim chief scientific officer, Foundation Fighting Blindness. “LCA caused by CRB1 mutations is a challenging, unmet need.”

Horama has a Phase 1/2 clinical trial underway for people with retinitis pigmentosa caused by mutations in PDE6B.