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Gene Therapy for Macular Degeneration positive results

For patients suffering from age-related macular degeneration, gene therapy has the potential to transfigure the treatment options that are available.

Two therapies are currently in clinical trials. One new therapy currently in trials is RGX-314. This new therapy is delivered sub-retinally or suprachoroidally. Another therapy that is undergoing trials is ADVM-022, which is administered intravitreally.

The results of the RGX-314 trials showed that participants were more likely to have stable visual acuity and at least a 70% reduction in anti-VEGF injections. Forty percent of those in the treatment group no longer had to take anti-VEGF injections after 6 months.

Participants in the ADVM-022 trial also had positive outcomes. There were four groups in the study, and each group had different dosing protocols. None of the patients in group 1 needed additional injections after their initial high dose with a 13-day course of oral steroids.  Group 2, which received a low dose along with 13 day course of oral steroids, had 3 of the 6 patients who did not need additional injections. Group 3 patients received a low dose along with 6 weeks of topical steroids, which resulted in only two patients needing additional injections. Finally, group 4 participants had a high dose with six weeks of topical steroids. This group only had 1 patient who needed additional injections.

Researchers are still working on the proper dosing for these therapies and additional gene therapies are forthcoming as well. 

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