Grant Award Details
To enable an FDA pre-IND meeting for PRPE-SF (polarized hESC-derived RPE Soluble Factors), a therapy for Early Stage Dry Age-related Macular Degeneration.
Grant Application Details
- PRPE-SF, polarized hESC-derived RPE Soluble Factors, as a Therapy for Early Stage Dry Age-related Macular Degeneration
PRPE-SF is a preparation of soluble factors from polarized retinal pigment epithelial cells, to support survival of photoreceptors in dry AMD (dAMD).
Area of Impact
dAMD with early geographic atrophy (RPE dysfunction/photoreceptor degeneration) that does not involve the fovea, with visual acuity better than 20/80.
Mechanism of Action
PRPE-SF is composed of multiple neuroprotective and anti-inflammatory factors. The cause of AMD is multifactorial, with both genetic & environmental components. However, not all factors have been defined & targeting the known factors (ex. complement pathway) has not been successful. PRPE-SF does not target one specific mechanism as its multitude of factors may work synergistically (paracrine effect) to provide an optimal microenvironment for photoreceptor survival & function.
Unmet Medical Need
AMD affects over 2 million people nationwide (90% dAMD). The target population for PRPE-SF is patients with dAMD with early geographic atrophy, intended to slow progression of disease. There are no products approved for this target & successful development of PRPE-SF would be a major breakthrough.
To enable an FDA pre-IND meeting for PRPE-SF.
Major Proposed Activities
- Manufacturing Process Dev.
•Finalize manufacturing for transfer to cGMP
•Develop release testing analytics
•Scale PRPE-SF for Phase 1 clinical trial
- Preclinical Dev.
•Assess activity, dose & dose regimen for Phase 1 clinical trial
•Examine pilot distribution & safety of final PRPE-SF drug product.
- Clinical Trial Planning.
• Develop clinical plan & protocol synopsis for clinical trial
• Hold interact meeting with FDA
• Hold pre-IND meeting with FDA
AMD is one of the most common causes of blindness in those 50 or older with an estimated 400,000 Californians projected to suffer from AMD by 2020. AMD is a debilitating disease, which results in loss of independence and productivity, increased injury and dramatic decline in quality of life. With a $3 billion economic burden annually in California, PRPE-SF will be developed by California based companies, creating additional jobs for Californians and a treatment for this devastating disease.