Animal Studies Demonstrate Safety Profile for RPE Cell Therapy, Advanced Cell Technology (OTCBB: ACTC)
ACT completed key animal studies in connection with its Phase I multi-center study using embryonic stem cell derived Retinal Pigment Epithelium (RPE) cells to treat patients with Stargardt’s Macular Dystrophy (SMD), for which it filed an IND with the FDA in 11/09. The studies demonstrated an excellent safety profile with no safety signals such as tumors or ectopic tissues to address the FDA’s request for additional data on tumorigenicity and biodistribution.
ACT believes that the data will support the FDA granting clearance commence the SMD study in humans later this year. The Phase I trial will be a prospective, open-label study that is designed to determine the safety and tolerability of the retinal pigment epithelium (RPE) cells following sub-retinal transplantation to advanced patients with SMD. 12 patients will be enrolled into the study at 3 clinical sites.
ACT was granted “Orphan Status” for this proposed clinical trial earlier this year. As many as 10 M people in the US have photoreceptor degenerative disease. While most of these patients have Age-Related Macular Degeneration (AMD), a smaller number of patients have SMD.
