ACTC Advanced Cell Technology, Inc., a biotechnology

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Apr 12, 2013 at 7:50 PM

ACTC Advanced Cell Technology, Inc., a biotechnology company, focuses on the development and commercialization of human embryonic, induced Pluripotency (iPS), and adult stem cell technology in the field of regenerative medicine Operations The company’s business is focused on both the development and commercialization of adult stem cell therapies and therapies based on cells derived from embryonic stem (ES) cells or other potentially totipotent stem cell technologies. The company’s adult stem cell-based products are specifically targeted at therapies for heart and other cardiovascular disease and are at a more advanced stage of development than its human ES cell based technologies. Its first human ES cell-based product, retinal pigmented epithelial cells, have entered Phase I clinical trials and various patients have already been treated. The company’s intellectual property includes patent rights and applications for specific applications of stem cell technology in producing retinal pigment epithelium (RPE) cells, hemangioblasts, myoblast stem cells, and various methods and compositions for the use of these technologies and derived cells in treating retinal and other eye disease, inflammatory and autoimmune diseases, and heart disease, as well as to provide agents for wound healing and replacement of blood components. Human ES Cell Programs The company’s research and technologies are focused on solving this obstacle by creating stem cell therapeutics with compatible tissues or which can be used in immunopriveleged or immunosuppressed sites of transplantation in patients. Compatible tissues are referred to as being histocompatible. The company has developed an alternative to inner cell mass (ICM)-derivation of human embryonic cell (hESC) - a method which utilizes single cell biopsy to remove a single blastomere from a 4-8 cell preembryo in a manner which does not result in the destruction of the preembryo. The company’s human ES cell technologies comprise cellular reprogramming and pluripotent stem cell technologies to produce cells that have the proliferative capacity of ES cells with therapeutic application and compatible with the patient. Cell Therapy Research Programs The company’s research in this area includes projects focusing on developing different cell types that may be used in the future to treat a range of diseases. Its researchers have generated stable RPE cell lines for use in its clinical retinal program and are working on projects to generate stable cell lines with particular focus on blood lineage and vascular epithelial cell lines from hemangioblast cells. Its cell therapy research programs include RPE and hemangioblast programs. The hemangioblast program is in preclinical development. Adult Stem Cell Program The company’s adult stem cell-based program is developing an autologous myoblast transplantation therapy delivered using a minimally invasive catheter injection system to restore cardiac function in patients with advanced heart disease. The key target for the therapy would be heart failure patients with New York Heart Association (NYHA) scores Class II to IV. The company's therapy could also benefit patients supported on ventricular assistance devices and potential additional indications, such as acute myocardial infarction, peripheral artery disease, and non-cardiac tissue repair. Intellectual Property The company’s research and development is supported by an intellectual property portfolio. It owns or has licenses to approximately 35 patents and has approximately 170 patent applications pending worldwide in the field of regenerative medicine and stem cell therapy. The company also has non-exclusive rights to a portfolio of patents and patent applications that support its core intellectual property. Collaborative Agreements In 2011, the company entered into a definitive collaborative agreement with Roslin Cells LTD (Roslin Cells) of Scotland. The company would work together to establish a bank of Good Manufacturing Practice (GMP)-grade human ES cell (hESC) lines using its patented, proprietary ‘single-cell blastomere’ technique for deriving hESC lines without destroying embryos. Regulations The company, in addition to safety regulations enforced by the US Food and Drug Administration (FDA), is also subject to regulations under the Occupational Safety and Health Act, the Environmental Protection Act, the Toxic Substances Control Act, and other present and potential future and federal, state, local, and foreign regulations.

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