Advanced Cell Technology, Inc. (“ACT” OTCBB: ACTC) is a biotechnology company that specializes in the development of cellular therapies for the treatment of rare and common diseases that impact millions of people worldwide. The company applies stem cell-based technologies (both adult and human embryonic) and other proprietary methods in the field of regenerative medicine to bring patient-specific therapies from the lab to the bedside. Having recently strengthened its balance sheet, ACT has three cellular product platforms based on ground-breaking stem cell technology.
ACT is focused on commercializing its Retinal Pigment Epithelial (RPE) therapy for degenerative retinal disease, for which it has recently secured two US Food and Drug Administration (FDA) clearances for Phase I/II clinical trials, as well as other milestones (see below). The company is also focused on its autologous (meaning “one’s own”) Myoblast stem cell therapy for the treatment of chronic heart failure, advanced cardiac disease, myocardial infarction, and ischemia, and is also developing its Hemangioblast (HG) platform for the treatment of blood and cardiovascular diseases.
ACT’s principal laboratory and GMP facility is located in Marlborough, Massachusetts, and its corporate offices are located in Santa Monica, California. ACT is led by an experienced management team and a world-class scientific team helmed, respectively, by Interim Chairman and CEO Gary Rabin and Chief Scientific Officer Robert Lanza, M.D.
ACT owns or licenses more than 150 patents and patent applications related to stem cell therapy and regenerative medicine.
Notably, ACT recently announced two major developments related to IP: the Company was awarded a broad patent for production of RPE cells; and the Company is now poised to obtain and control key patents related to the generation of iPS cells.
While the Company’s Myoblast therapy for cardiac disease is an autologous adult stem cell therapy, its RPE and HG programs are hESC-(human embryonic stem cell)-based. As documented in the scientific journals NATURE and Cell Stem Cell, ACT developed and holds in its repertoire the first-ever proven alternative method for successful hESC derivation without harm to the original cells, called the single-cell blastomere technique.
RPE Program Advanced Cell Technology has developed a fully-differentiated Retinal Pigmented Epithelium (RPE) cell derived from human embryonic stem cells, which can be used as a cellular therapy to treat retinal degenerative diseases. The FDA recently granted ACT clearance to proceed with two separate Phase I/II clinical trials to test the safety of the hESC-derived RPE cellular therapy for Stargardt’s Macular Dystrophy (SMD), and for Dry Age-Related Macular Degeneration (Dry AMD). Dry AMD, a $25-30 Billion market in the US and Europe alone, and there are no approved therapies currently available for either Dry AMD or SMD.
Additionally, in February of last year the NIH proposed expanding its definition of hESCs for funding purposes, in part to accommodate ACT’s cell lines derived using the single-cell blastomere technique, potentially directly benefiting ACT’s RPE program. Also, last spring the FDA granted ACT’s RPE cells “Orphan” status for treatment of SMD, making the company eligible to receive tax credits, access to grant funding for clinical trials such as the one which was just approved, marketing exclusivity after drug approval for a period of as long as seven years, and accelerated approval. Additionally, in June of last year ACT was awarded a broad patent for production of RPE cells.
Myoblast Program ACT’s Myoblast treatment may prove particularly beneficial for patients who have experienced a serious heart attack and are at risk for heart failure. ACT has successfully completed several Phase I clinical trials and has secured FDA approval to commence with Phase II Clinical Trials. Myoblast has several advantages relative to currently marketed products and other stem cell therapy approaches, including symptom management and disease modification.
Hemangioblast Program ACT’s Hemangioblast program is for the treatment of blood and cardiovascular diseases. A paper published in NATURE Methods revealed the Company’s successful generation of functional Hemangioblast cells from human embryonic stem cells.
Gary Rabin, Interim Chairman and Chief Executive Officer – 23-year career in finance and operations
Dr. Robert Lanza, M.D., Chief Scientific Officer – 25-year career in biomedical & scientific research
Edmund Mickunus, Vice President of Regulatory – 28-year career in biotechnology & medical devices
Roger Gay, PhD, Senior Director of Manufacturing – 28-year career in clinical manufacturing & biotechnology product development
Rita Parker, Director of Operations – 20-year career in business management