Gene Therapy Trials Show Promise in Treating Age-Related Macular Degeneration

CHICAGO, IL -- November 1, 2005 -- Using what is known as an RNA duplex called small interfering RNA (siRNA), investigators are moving one step closer toward using gene therapy to combat age-related macular degeneration.No particular safety concerns with Cand5 were identified, and the most commonly reported adverse events were related to the injection procedure. No increases in intraocular pressure were observed.Based on these findings, Dr. Thompson said, a phase 2 trial has been approved by the FDA and is now enrolling patients.Another study using a similar siRNA (Sirna-027, Sirna Therapeutics, San Francisco, CA) reported improvements in ETDRS scores of 5 letters for those who received 100 mcg doses, and 10 letters for patients who received the 200 mcg dose. Follow-up for those patients was 84 days and 56 days respectively, he said.

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