Timrepigene emparvovec, a gene therapy for choroideremia, has demonstrated safety and efficacy in maintaining visual acuity when administered bilaterally, according to a study.
The therapy was well tolerated with mostly mild to moderate side effects, and no significant increase in serious adverse events was observed with sequential eye treatments.
The multicenter, open-label GEMINI study, 66 participants were treated with timrepigene emparvovec, with 53 completing the full protocol. The therapy was well tolerated, with visual acuity generally preserved in both eyes regardless of the time interval between treatments. Most adverse events were mild or moderate, with serious surgical complications occurring in only 7.6% of cases. Retinal inflammation, a common side effect, was reported in 45.5% of participants but did not correlate with significant vision loss over 12 months.
The study also found no increased risk of serious treatment-emergent adverse events or systemic immune reactions with sequential eye injections. However, the presence of anti-vector neutralizing antibodies at baseline was linked to a higher incidence of ocular inflammation or decreased visual acuity.
Reference
MacLaren RE, Audo I, Fischer MD, et al. An Open-Label Phase II Study Assessing the Safety of Bilateral, Sequential Administration of Retinal Gene Therapy in Participants with Choroideremia: The GEMINI Study. Hum Gene Ther. 2024;doi: 10.1089/hum.2024.017. Epub ahead of print. PMID: 38970425.
Related Content
