Advanced Cell Technology, Inc., ("ACT" or OTC: ACTC) is a biotechnology company that specializes in the development of cellular therapies for the treatment of diseases and conditions that impact tens of millions of people worldwide. The company applies stem cell-based technologies (both adult and human embryonic stem cells) and other proprietary methods in the field of regenerative medicine to bring patient-specific therapies from the lab bench to the bedside.
ACT's principal laboratory, GMP facility and corporate offices are in Marlborough, Massachusetts. ACT is led by an experienced management team and a world-class scientific team helmed, respectively, by Interim President, CFO and Executive Vice President of Corporate Development Edward Myles and Chief Scientific Officer Robert Lanza, M.D.
ACT is using a combination of embryonic and adult stem cell technologies to develop novel therapies for indications with few or no alternatives. As documented in the journals Nature and Cell Stem Cell, ACT developed and holds in its repertoire the first-ever proven alternative method for successful hESC generation without harm to the embryo, called the "single-cell blastomere" technique, on which it holds broad intellectual property (IP) protection.
ACT has developed an embryonic stem cell-based therapy which provides a promising treatment option for a variety of incurable eye diseases. Age-related macular degeneration (AMD) alone afflicts more than 30 million people worldwide and is the leading cause of blindness in those older than 60 in the United States. Atrophic (dry) AMD accounts for the vast majority of all of those cases, and currently represents a market in Europe and the United States alone of $25-30 billion. Stargardt's disease, meanwhile, typically affects patients before they reach 20 years of age, and the pathology includes the death of photorecepters due to the loss of functional RPE cells, resulting in blindness.
The company is focused on commercializing its human embryonic stem cell (hESC)-based Retinal Pigment Epithelial (RPE) therapy for degenerative retinal disease, for which it is currently conducting clinical trials. ACT is enrolling patients in two Phase 1/2 clinical trials to test the safety of the hESC-derived RPE cellular therapy for Stargardts Macular Dystrophy (SMD) and for Dry Age-Related Macular Degeneration (Dry AMD). Both the FDA and the European Medicines Agency (EMA) have also granted ACTs RPE cells Orphan status for treatment of Stargardts Disease.
ACTs hemangioblast (HG) program for the treatment of blood and cardiovascular diseases is currently in preclinical development. Hemangioblasts have multiple potential therapeutic applications for the repair of vascular tissue and could be a promising treatment option for a variety of diseases including cardiovascular diseases, diabetes and stroke. A paper published in Nature Methods documented ACTs successful generation of functional hemangioblast cells from human embryonic stem cells, and a paper published earlier this year in Cell Research indicated that hESCs could be a potentially unlimited source of platelets for transfusion. |