Key Takeaways

  • A phase 1/2a study showed that gene therapy can be used to provide sustained delivery of a therapeutic protein in patients with wet AMD.
  • Higher doses of the gene therapy, called ABBV-RGX-314, demonstrated control of blood and fluid leakage from blood vessels and maintained vision in wet AMD patients.

Many patients with wet AMD require frequent eye injections of an anti-VEGF protein to maintain vision and control leakage of blood and fluids from blood vessels, Peter A. Campochiaro, MD, lead author of a new study published in The Lancet, told Healio.  Observational studies have shown that many patients do not get the frequency of injections needed and therefore lose vision, he added. 

ABBV-RGX-314 (Regenxbio, AbbVie) is in development as a gene therapy that would provide patients with a constant supply of anti-VEGF protein, potentially with only one injection. In a phase 1/2a study, 42 subjects with wet AMD were given one of six doses of the gene therapy. Subjects were evaluated 1 day and 1 week after administration of ABBV-RGX-314 and then monthly for 2 years.

According to Campochiaro, the ABBV-RGX-314 gene therapy was safe and well tolerated. Higher doses of the therapy demonstrated sustained anti-VEGF protein expression, controlled fluid and blood leakage from blood vessels, and maintained vision in many patients, while lower doses showed little to no anti-VEGF protein production and no beneficial effects. “The most important message is that gene therapy can be used to provide sustained delivery of a therapeutic protein,” he said. “There are two ongoing phase 3 trials, which, if successful, could lead to regulatory approval of ABBV-RGX-314, making it available for use in clinical practice.” For more information on the ongoing clinical trials, visit https://clinicaltrials.gov/study/NCT04704921 and https://clinicaltrials.gov/study/NCT05407636 or contact AbbVie Patient Advocacy at 866-860-0117 or Patientadvocacy@regenxbio.com. 

Edited by Miriam Kaplan, PhD

Source: Anthony DeFino, Healio Ocular Surgery News, April 10, 2024; see source article