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Imaging Analysis Suggests Cell Engraftment and Likely Biological Response
“We are encouraged by the results and look forward to the next steps in
moving this therapeutic through the clinic and addressing the needs of
the millions of patients with dry-AMD,” said
The presentation at ARVO reported new clinical trial data with two patients that were treated in cohort 2, where they received a dose of 200,000 cells. Imaging analysis suggests the transplanted OpRegen cells remained in place (engrafted) in an area of the scar that was completely depleted of retinal pigment epithelium (RPE) because of the advanced stages of the disease. Cell engraftment occurred in four of the five patients treated thus far. There was also possible evidence of a biological response with some areas appearing to show structural improvement (a thickening of the thinned area of retina above the scar) without any signs of retinal edema, a fluid build-up that can further compromise vision.
“I am excited to see that the second cohort is showing evidence of cell
survival and early signs suggesting a biological response,” stated Dr.
Best corrected visual acuity (BCVA) remained stable in all treated eyes and importantly showed no signs of deterioration, which could be expected if no treatment was given. The data also continues to show that the procedure is well tolerated in all patients, including those patients with follow-up for more than one year after treatment.
“We continue to be encouraged by the lack of serious adverse events in
the trial during both the surgical procedure required for cell
transplantation and long-term post-operative monitoring,” stated
OpRegen is the lead product of BioTime’s ophthalmology subsidiary
OpRegen is an investigational therapy in which retinal pigment epithelial (RPE) cells are transplanted into the subretinal space, where they are intended to replace missing RPE cells. The ongoing trial is a Phase I/IIa dose-escalation study evaluating the safety and efficacy of three different dose regimens.
More information about the data presented at ARVO is available here.
OpRegen® for the treatment of the dry form of age-related macular degeneration (AMD), consists of a suspension of Retinal Pigment Epithelial (RPE) cells that are delivered subretinally during a simple intraocular injection. RPE cells are essential components of the back lining of the retina, and function to help nourish the retina including photoreceptors. A proprietary process that drives the differentiation of human pluripotent stem cells is used to generate high purity OpRegen® RPE cells. OpRegen® RPE cells are also “xeno-free," meaning that no animal products are used either at any point in the derivation and production process. The avoidance of the use of animal products eliminates some potential safety concerns. Preclinical studies in rats have shown that following a single subretinal injection of OpRegen®, the cells can rapidly organize into its natural monolayer structure in the subretinal space and survive throughout the lifetime of the animal. OpRegen® is designed to be an “off-the-shelf” allogeneic (non-patient specific) product. Unlike treatments that require multiple, frequent injections into the eye, it is expected that OpRegen® would be administered in a single procedure. OpRegen® was granted Fast Track designation from FDA which allows more frequent interactions with the agency, and eligibility for accelerated approval and priority review. OpRegen® is a registered trademark of Cell Cure Neurosciences Ltd., a majority-owned subsidiary of BioTime, Inc.
About Dry Age-Related Macular Degeneration (Dry-AMD)
Macular degeneration affects approximately 11 million people in the U.S. and is the leading cause of blindness in people over the age of 60. Approximately 90 percent of these patients suffer from the dry form, for which there are no FDA-approved therapies. In dry-AMD, there is a loss or dysfunction of the layer of retinal pigment epithelial (RPE) cells generally in the region of the eye called the macula, which is the part of the retina responsible for sharp, central vision that is important for facial recognition, reading and driving. These RPE cells support the light detecting photoreceptor cells that are so critical to vision. When we look at something, the photoreceptors (rods and cones) detect the light and send the information to the brain allowing us to perceive our surroundings. The age-dependent loss of the RPE cells therefore leads to degeneration of nearby photoreceptors and this can lead to severe vision loss or even legal blindness. Generally, the damage caused by the “dry” form is not as severe or rapid as that of the “wet” form. However, in the advanced stage of dry macular degeneration widespread loss of RPE and photoreceptors in the macular area, called geographic atrophy, leads to severe vision loss. While therapeutics are available to treat the wet form of AMD, there are currently no FDA-approved therapies for dry-AMD.
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