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‘Stop the RNA’ and delay AMD progression
WAILEA, Hawaii — A small interfering RNA compound has delayed the progression of age-related macular degeneration in early clinical testing, according to an investigator speaking here.
Peter K. Kaiser, MD, spoke about ophthalmic applications of short interfering RNA (siRNA) here at Retina 2006, held in conjunction with Hawaiian Eye 2006. He said that RNA proteins are what cause conditions such as AMD.
“But can we stop the protein before it’s made?” he asked rhetorically. “If you stop the RNA, there is no process, and you can turn off certain RNA proteins.”
Dr. Kaiser described Sirna-027 from Sirna Therapeutics, a partially modified RNA that inhibits vascular endothelial growth factor. Sirna has described RNA interference as a natural selective process for turning off genes triggered by short interfering molecules that engage certain cellular proteins.
“If you inject it into the vitreous, it might break down unstable RNA,” Dr. Kaiser said.
In a phase 1 study, Dr. Kaiser said, all 23 patients have “experienced stabilization of visual acuity, and 23% experienced visual improvement of three or more lines of visual acuity within 8 weeks of Sirna-027 injection.”
He said no serious adverse events or dose-limiting toxicities were observed.
Dr. Kaiser said phase 2 studies of the drug are scheduled to begin in the first quarter of 2006. Sirna announced last year that it has partnered with Allergan to develop the compound for ophthalmic use.