Certain retinal diseases are characterized by degeneration of the retinal pigment epithelium (RPE; a pigmented layer of the retina) which in turn results in photoreceptor loss. Typical retinal degeneration diseases include Retinitis Pigmentosa, Stargardt’s disease (Juvenile Macular Degeneration), Age-related Macular Degeneration (AMD), and other related diseases and conditions.
AMD, for example, afflicts more than 30 million people worldwide and is the leading cause of blindness in people over 60 years of age in the United States. Currently, only a few marketed treatments are available, and these are only focused on treatment of “wet,” or neovascular, AMD, which accounts for only ten percent of all cases of AMD, the other 90% being “Dry” AMD.
ACT’s breakthrough technology in stem cell therapy provides a fully differentiated treatment to patients affected by conditions involving the RPE. ACT differs from the competition in providing, for the first time, a cellular therapy that may provide not only a lasting treatment, and perhaps reversal of some vision loss, for AMD, but also a potential therapeutic approach for a host of other degenerative eye diseases.
ACT has published positive data from animal studies that were completed in collaboration with the Casey Eye Institute at Oregon Health and Science University (OHSU). Study results show that well-characterized RPE cells derived from stem cells and manufactured under GMP-compliant conditions survive after transplantation into the subretinal space of Royal College of Surgeons (RCS) rats (the RSC rat is was the first known animal with retinal degeneration) and appear to appropriately engraft in the correct structures of the eye.
Moreover, these cells achieve significant rescue of visual function in a dose-dependent fashion in an animal model of photoreceptor degeneration. The data further suggest that these cells may be effective in limiting and perhaps even reversing the deterioration of vision accompanying RPE-driven photoreceptor degeneration in human disease.
Based on the various animal studies, and working with leading retinal surgeons and eye clinics, ACT has developed clinical programs and filed Investigational New Drug Applications (INDs) with the FDA for treatment of Stargardt’s disease and Dry AMD, and is developing preclinical data for other diseases impacted by RPE cells.
With a proven track record in stem cell research and product development, and a world-class team of researchers and seasoned executives, ACT is uniquely positioned to offer the opportunity to make a meaningful societal impact with its RPE Program. The economic cost of AMD disease is enormous on both a domestic and global scale. Treatment of wet AMD, alone, is expected to account for $1.84 billion in drug revenues in the US market alone in 2010, according to The Eye Digest. The conservatively estimated potential market for AMD generally (wet and dry) in the US alone is projected at over $25 billion.
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Key Milestones in RPE Clinical Program include:
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