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First Patients Enrolled In Two Clinical Trials Using Embryonic Stem Cells To Treat Stargardt's Disease And Dry Age-Related Macular Degeneration

June 16, 2011

Advanced Cell Technology, Inc. announced today the enrollment of the first patients in its two Phase 1/2 clinical trials for Stargardt's Macular Dystrophy (SMD) and Dry Age-Related Macular Degeneration (Dry AMD) using retinal pigment epithelial (RPE) cells derived from human embryonic stem cells (hESCs). The patients were enrolled at the Jules Stein Eye Institute at the University of California, Los Angeles (UCLA).

The Phase 1/2 trials are prospective, open-label studies primarily designed to determine the safety and tolerability of the RPE cells following sub-retinal transplantation into patients with SMD and Dry AMD. Each study will enroll 12 patients, and the primary endpoint of both studies is to determine the safety and tolerability of these cells at 12 months.

"The enrollment of the first patients in our two clinical trials marks an important step forward for the field of regenerative medicine," said Gary Rabin, interim chairman and CEO of ACT. "We are very pleased with the progress that has been made toward bringing this ground-breaking technology to the patients who need it most. If these therapies work as we hope they will, particularly with small volumes of cells, then we should be in an excellent position to take advantage of our patented techniques for manufacturing large numbers of doses of RPE cells that can be conveniently stored and shipped to clinicians following the basic manufacturing and distribution systems already familiar to pharmaceutical and biotech companies."

Principal investigator Steven Schwartz, M.D., Ahmanson Professor of Ophthalmology at the David Geffen School of Medicine at UCLA and retina division chief at the Jules Stein Eye Institute at UCLA, said, "These trials mark a significant step toward addressing what is one of the largest unmet medical needs of our time—treatments for otherwise untreatable and common forms of legal blindness, Dry AMD, SMD and other forms of atrophic macular degeneration. Dry AMD is the most common form of macular degeneration. It is the leading cause of blindness in the developed world, and is the leading cause of blindness in people over the age of 55. The incidence of Dry AMD is expected to double over the next 20 years as the population ages. This trial will begin the process of understanding whether stem cell-derived RPE cells have the potential to be a safe and effective treatment for these debilitating diseases. We are looking forward to evaluating the safety and tolerability data of these Phase 1/2 trials, and hope that these early trials will also produce key information relating to engraftment and function of the transplanted RPE cells."

The progress of disease in both SMD and Dry AMD includes atrophy or thinning of the layer of RPE cells in the patient's macula at the center of the retina, the region specialized for high acuity vision. With the loss of RPE cells in the macula comes the eventual loss of photoreceptors. Over time, the progressive loss of RPE cells and concomitant loss of photoreceptors can cause severe central visual deterioration and even blindness as the macula becomes less functional and central vision is gradually lost. ACT's SMD and Dry AMD therapeutic programs utilize transplanted RPE cells to treat these conditions by replacing RPE cells in the patient's eyes before all RPE function is lost.

"Initiating these two clinical trials represents an important milestone for embryonic stem cell research," said Robert Lanza, M.D., chief scientific officer of ACT. "After a decade of extensive research and preclinical studies, it is very satisfying to finally be moving into the clinic. We hope that these cells will, in the future, provide a treatment not only for these two untreatable diseases—Stargardt's disease and macular degeneration—but for patients suffering from a range other debilitating eye diseases."

In addition to the Jules Stein Eye Institute at UCLA, the Casey Eye Institute (CEI) at Oregon Health & Science University (OHSU) in Portland, OR, is also open for enrollment of patients with SMD. As additional sites are ready to enroll patients with SMD and dry AMD, they will be listed on the Clinical Trials page on ACT's Web site and on www.clinicaltrials.gov.

Adapted from Advanced Cell Technology

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