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-Data from patients in Cohort 1 and Cohort 2 to be presented-
The abstract, titled “Phase I/IIa clinical trial of human embryonic stem cell (hESC)-derived retinal pigmented epithelium (RPE, OpRegen®) transplantation in advanced dry form age-related macular degeneration (AMD): interim results”, will be presented by Professor
As previously announced, upcoming program milestones are expected to include:
- Expansion of OpRegen® into the initial U.S. clinical sites in the coming weeks,
- Further data from cohort 2 in the first half of this year,
- Beginning enrollment of cohort 3 of this trial in the coming months,
- Completing enrollment of cohort 3 and 4 this year and
- Reporting data from cohort 3 of the trial this year.
About Dry Age-Related Macular Degeneration (Dry–AMD)
Macular degeneration affects approximately 11 million people in the U.S. It 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
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. 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 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 the
About the OpRegen® Clinical Trial
The Phase I/IIa open label safety and efficacy clinical trial is designed to evaluate three different dose regimens of OpRegen® in four cohorts. Cohort 1 includes three patients each receiving 50,000 cells, cohort 2 includes three patients each receiving 200,000 cells, upon DSMB approval cohort 3 will include three patients each receiving 500,000 cells, and upon DSMB approval cohort 4 will include six patients each receiving 500,000 cells.
Following transplantation, the patients will be followed for 12 months at specified intervals in order to evaluate the safety and tolerability of the product. Following the initial 12 month period, patients will continue to be monitored at longer intervals.
A secondary objective of the clinical trial will be to examine the ability of transplanted OpRegen® to engraft, survive, and modulate disease progression in the patients. In addition to thorough characterization of visual function, a battery of vision tests will be used to quantify improvements in reducing the progression of the disease.
Certain statements contained in this release are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Statements pertaining to future financial and/or operating results, future growth in research, technology, clinical development, and potential opportunities for
To receive ongoing