The company is conducting a total of three clinical trials in the U.S. and Europe. Each trial will enroll a total of 12 patients, with cohorts of three patients in an ascending dosage format. These trials are prospective, open-label studies designed to determine the safety and tolerability of hESC-derived RPE cells following sub-retinal transplantation into patients with dry AMD or SMD at 12 months, the study’s primary endpoint. Preliminary results from the two U.S. trials were reported in The Lancet earlier this year.
“Completing the higher-cell dosage in both our U.S. trials is an important milestone in our clinical programs,” said Robert Lanza, M.D., ACT’s chief scientific officer. “We are now halfway through both U.S. trials, and look forward to treating the remaining two patient cohorts in the coming year.”
Added Dr. Eliott, “Dry AMD affects upwards of 30 million people worldwide. Moreover, it has no approved drug treatment available to date. ACT’s hESC-derived RPE cells could address this considerable unmet need and also point the way towards treatments of other forms of macular degeneration. We are pleased to be a part of this important clinical trial.”
