Advanced Cell Technology, Inc. (ACTC) to Pursue Cutting Edge Stem Cell Research with Clinical Translation of “Universal” Blood, Platelets and...

Advanced Cell Technology, Inc. (ACTC) to Pursue Cutting Edge Stem Cell Research with Clinical Translation of “Universal” Blood, Platelets and Off-the-Shelf Mesenchymal Stem Cells

Advanced Cell Technology, Inc., a leader in the field of regenerative medicine, recently announced an agreement with CHA Bio & Diostech Co, Ltd. (“CHA Biotech”), a South Korea-based biotechnology company that is designed to enhance the efforts of the two companies’ international joint venture, Stem Cell & Regenerative Medicine International (SCRMI). Under the terms of the agreement, SCRMI exclusively licenses the rights to its hemangioblast program to ACT for North America (United States and Canada) and to CHA Biotech for Korea and Japan. The companies will each work to develop clinical therapies based on the joint venture’s proprietary hemangioblast cell technology.

SCRMI was formed as a joint venture between ACT and CHA Biotech and has been harnessing expertise from both sides of the globe to drive cutting-edge stem cell research. Under the terms of the agreement, the SCRMI scientists involved in hemangioblast research have been reassigned to ACT where they will continue their research and product development efforts as ACT employees. Ten SCRMI employees are being reassigned to ACT, including Shi-Jiang (John) Lu, Ph.D., SCRMI’s Senior Director of Research, Erin Kimbrel, Ph.D., Qiang (Allen) Feng, Ph.D., and their respective teams. SCRMI’s research has focused on differentiating human embryonic stem cells (hESCs) and induced pluripotent stem (iPS) cells into hemangioblasts – a multipotent cell that is a common precursor to blood, immune, and vascular tissue. The ownership in SCMRI remains largely unchanged between ACT and CHA Biotech. The joint venture is ceasing internal research activity and transitioning to a licensing entity.

“The clinical prospects for this technology are immense,” said Robert Lanza, M.D., chief scientific officer of ACT. “Hemangioblasts can be used to provide transfusable blood components, such as red blood cells and platelets, as well as cells capable of affecting vascular repair. We hope that these programs will eventually help address the critical care shortage of blood for emergency situations. In addition, our preclinical research has shown the ability of hemangioblast-derived cells to repair vascular damage when administered systemically, offering potential therapeutic uses to treat vascular ischemic damage, ischemia-reperfusion injury, diabetic vascular disease and peripheral artery disease (PAD), which represent the leading causes of death and/or disability worldwide. Indeed, in preclinical studies, these cells quickly repaired vascular damage, cutting the death rate after a heart attack in half and restoring the blood flow to ischemic limbs that might otherwise have required amputation.”

The proprietary hemangioblast technology is uniquely well-suited for developing stem cell-based therapies because they are highly expandable and can consistently and reproducibly give rise to various cells of the hematopoietic (blood) and endothelial (vascular) lineages. Strong clinical demand for donated platelets, for example, has created interest in generating renewable sources of transfusable platelets. In recent scientific publications, SCRMI scientists showed that it is feasible to generate functional megakaryocytes and platelets from hESCs on a large scale. The hESC-platelets displayed features that were indistinguishable from those of normal blood platelets, and participated in both clot formation and retraction in-vitro.

In September of last year, Dr. Lanza and Kwang-Soo Kim, Ph.D. (Harvard University/McLean Hospital and CHA Stem Cell Institute), won a National Institutes of Health (NIH) Director’s Opportunity Award for research in “Translating Basic Science Discoveries into New and Better Treatments” under the American Recovery & Reinvestment Act of 2009. The NIH Award provides McLean Hospital and SCRMI with $1.9 million to explore the potential of protein-induced pluripotent stem (iPS) cells as a source of universal red blood cells and platelets for transfusion. Drs. Lanza and Kim will continue to serve as principle investigators on this grant.

“We are very excited about this next step in our joint venture,” said Hyung-Min Chung, Ph.D., chief technology officer of CHA Biotech and chief executive officer of SCRMI. “While still permitting us to leverage the combined expertise of our two companies, this new licensing and development arrangement should help to accelerate the development of clinical programs, and open the door to further government-sponsored research than had been available to SCRMI. Fundamentally, we believe that the hemangioblast program has great potential to address many unmet medical needs through the use of stem cell technology, and feel that this realignment in licensing and research efforts is an important step towards CHA Biotech and ACT realizing those goals.”

“Given the progress that has been made by our teams in the development of hemangioblast-derived products, coupled with bringing the scientists from the joint venture into ACT and Dr. Lanza’s devotion to these programs, this new exclusive license affords ACT a unique new pipeline of therapeutic products, some of which are not that far from the clinic,” added Gary Rabin, interim chairman and chief executive officer of ACT. “The market opportunities around diabetes-related diseases alone are vast. Diabetes is the leading cause of blindness, non-traumatic limb amputations, and cardiovascular morbidity and mortality, each of which may be treatable using hemangioblast-derived therapies. We have within our reach the opportunity to make a meaningful impact on the lives of these patients, while building a product pipeline that leverages off our core competencies relating to stem cells.”

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