Regenxbio reports promising three-year follow-up data for its innovative single-dose gene therapy targeting diabetic retinopathy, highlighting sustained efficacy and safety, as it advances to larger clinical trials.
REGENXBIO has reported new three-year follow-up data suggesting that its single-dose gene therapy for diabetic retinopathy can continue to hold up well over time, with the latest results presented at the Retina Society’s 59th Annual Scientific Meeting in Los Angeles. The company said the findings come from the Phase II ALTITUDE study of surabgene lomparvovec, also known as ABBV-RGX-314, delivered through a suprachoroidal injection, a route that places treatment between the sclera and the choroid at the back of the eye.
According to REGENXBIO, 60% of participants in dose level 3 who reached the three-year mark achieved more than a two-step improvement on the Diabetic Retinopathy Severity Scale without needing any further treatment. The company also said there were no vision-threatening events in that group. In a separate safety finding, no intraocular inflammation was seen over three years among 17 patients who had received short-course preventative steroid eye drops.
The new data extend earlier reports on the same programme. REGENXBIO had previously said the treatment showed durable results at two years, and later long-term updates pointed to sustained benefit without intraocular inflammation under the same steroid regimen. The company has said dose level 3, at 1.0 x 10^12 gene copies per eye, is now being assessed in the Phase IIb/III NAAVIGATE trial, reflecting the push to test whether an in-office gene therapy could offer a longer-lasting option for people with diabetic retinopathy.
Diabetic retinopathy, a diabetes-related eye disease that can damage blood vessels in the retina and threaten sight, remains a major cause of vision loss in working-age adults. That makes durable treatments particularly attractive if they can reduce the need for repeated injections or other ongoing care. REGENXBIO’s latest results do not prove the therapy will become standard treatment, but they do add to the case for continued study as larger trials try to confirm both efficacy and safety.
Disclaimer: This content is for informational purposes only and is not intended to be a substitute for professional medical judgment, advice, diagnosis, or treatment.
