ACT receives patent for RPE cells to treat retinal degeneration

Sep 15 2010

Advanced Cell Technology, Inc. ("ACT"; OTCBB: ACTC), a leader in the field of regenerative medicine, announced today that it will be issued U.S. Patent Numbers 7,795,025 and 7,794,704 on Tuesday, September 14th, which continue to extend the company's patent portfolio covering its retinal pigment epithelial (RPE) cell programs. In particular, the claims that will issue in patent number 7,794,704 broadly cover methods for treating retinal degeneration using human RPE cells differentiated from human embryonic stem cells (hESCs). Issuing with 68 claims, this patent covers methods of treatment with hESC-derived RPE cells that includes, but is not limited to, Stargardt's disease, retinitis pigmentosa, and macular degeneration. The 41 claims issuing in US Patent 7,795,025 contribute to the development of the company's protection of the processes for manufacturing RPE cells from human ES cells. The patent covers fundamental methods for generating transplantable cells for treatment of human patients.

“A valuable addition to our strong intellectual property portfolio, these patents should help position ACT as the dominant player in this potentially very large market. Our RPE technology is safe and scalable, and has tremendous potential for treating some 200 or more diseases of the retina.”

"Another wonderful milestone for ACT! These patents continue the recognition of the inventions and innovations resulting from our scientific team's ongoing research, and further protects the platform technology underlying our RPE program, one of our key therapeutic programs," said William M. Caldwell IV, ACT's Chairman and CEO. "A valuable addition to our strong intellectual property portfolio, these patents should help position ACT as the dominant player in this potentially very large market. Our RPE technology is safe and scalable, and has tremendous potential for treating some 200 or more diseases of the retina."

"ACT is developing first-in-class treatments for degenerative disorders of the retina," said Robert Lanza, M.D., ACT's Chief Scientific Officer. "The use of RPE cells created from human embryonic stem cells should open the door to potential treatments for many diseases of the retina that impact sight. According to the World Health Organization, macular degeneration alone is known to affect 30-40 million people worldwide, and this represents only a handful of the 200 diseases that may be treated using our RPE cells. We have worked hard to develop an efficient method for producing a renewable source of transplantable RPE cells that can be used to target diseases such as Stargardt's Disease and Age-related Macular Degeneration. For many of these patients there are no available treatments. We have demonstrated that our stem cell-derived RPE cells can rescue visual function in animals that otherwise would have gone blind. We are looking forward to starting our clinical trials with the hope that these cells will be similarly efficacious in patients."

"We believe that these patents are especially important as they extend the company's patent coverage of the scalable manufacturing of human RPE cells for therapeutic use, which are core to our technology and product portfolio," continued Mr. Caldwell. "This IP further expands our patent estate with respect to protecting the use of RPE cells in a wide range of treatments, offering additional validation of the strength and breadth of our patent portfolio. This development also dovetails nicely with the prospect of initiating our human clinical trial for our RPE program. We are optimistic that the methods-of-treatments and the culturing processes covered by these two new patents, along with the Company's proprietary detection technique for final product release, will establish a formidable barrier to entry for any potential competitors. Once we have begun to treat Stargardt patients, we plan to initiate another clinical trial relating to the use of RPE cells in the treatment of dry Age-Related Macular Degeneration (AMD). At present there is no approved treatment for dry AMD, despite the fact that it represents a $20-30 billion potential market."

ACT's Single Blastomere technology used for isolating hESCs does not require the destruction of embryos. The RPE and other hESC-derived cells the company intends to produce for clinical use all begin with these "embryo-safe" stem cells. ACT does not rely on government funding for any of its research or development efforts, and accordingly has not been impacted by the recent court injunction against federal funding of hESC research. Nevertheless, the company's hESC lines and cells made for those lines (such as RPE cells) should fall outside the scope of the court order. While the injunction has been stayed, it is widely believed that this is only a temporary reprieve, with a permanent injunction a real possibility perhaps as early as the end of this month. Should that come to pass, ACT stands ready to offer its human stem cell lines to the research community, pending their approval by the National Institutes of Health (NIH).