FDA Clears Opus Genetics’ IND Application for Gene Therapy Candidate for Rare Inherited Retinal Disease

According to a statement from Opus Genetics, the FDA has approved its investigational new drug (IND) application for a phase 1/2, first-in-human clinical trial of OPGx-001 in patients with Leber congenital amaurosis (LCA) brought on by biallelic mutations in the LCA5 gene (LCA5).

OPGx-001 is an AAV8 vector designed to deliver a functional LCA5 gene to retinal photoreceptors precisely. There are no approved treatments available right now for people who have vision loss caused by LCA5.

“We founded Opus a little more than a year ago to quickly move promising potential treatments into the clinic for patients in need. This FDA clearance of our IND application for OPGx-001 for LCA5 marks a significant milestone for Opus, as our first program to enter the clinic,” Ben Yerxa, PhD, Chief Executive Officer of Opus, said in a company news release. “Preclinical studies in in vitro and in vivo models of LCA5 have provided support for the safety and efficacy of OPGx-001. We look forward to initiating our first-in-human trial of OPGx-001 in early 2023 and to continuing to build and advance our pipeline of gene therapies for unaddressed inherited retinal diseases in parallel.”

Nine adult patients with LCA5 will participate in the phase 1/2, open-label, dose-escalation trial to assess the subretinal administration of OPGx-001. The trial's goal is to assess possible benefits and safety. Opus intends to add a pediatric cohort after safety in adults has been deemed to be sufficient.

About OPGx-001

OPGx-001 is designed to address a form of Leber congenital amaurosis (LCA) due to biallelic mutations in the LCA5 gene (LCA5), which encodes the lebercilin protein. LCA5 is an early-onset severe inherited retinal degeneration.

Studies on LCA5 individuals have found evidence of a separation between retinal structure and visual function in this condition, suggesting a potential therapeutic target for gene therapy. OPGx-001 accurately delivers a functional LCA5 gene to photoreceptors in the retina using an adeno-associated virus 8 (AAV8) vector. Preclinical data, including animal and human iPSC models, have demonstrated preservation of retinal structure and visual function when OPGx-001 was administered prior to peak disease severity.