Grant Award Details
Complete a Phase 2 Study of the Safety of Repeat Intravitreal Injection of Human Retinal Progenitor Cells (jCell) in Adult Subjects with Retinitis Pigmentosa
Grant Application Details
- A Phase 2 Study of the Safety of Repeat Intravitreal Injection of Human Retinal Progenitor Cells (jCell) in Adult Subjects with Retinitis Pigmentosa
Therapeutic Candidate or Device
Allogeneic human retinal progenitor cells (hRPC)
Retinitis Pigmentosa (RP)
The cells are intended to remain suspended in the vitreous cavity of the eye and exert a beneficial neurotrophic effect on the degenerating retina.
Unmet Medical Need
RP is an incurable orphan disease. There are no treatments currently available other than a retinal chip for very end stage patients. To date, there is nothing that will restore sight or slow the progression of vision loss in RP. Achieving any measurable benefits would be groundbreaking.
CMC readiness & re-dosing
Major Proposed Activities
- Perform critical path manufacturing activities and conduct an in vitro comparability study of clinical versus commercial processes
- Validate endpoints to ensure that changes in patients’ vision function and functional vision are captured by appropriate and meaningful endpoints
- Assess the safety of a repeated injection of jCell in proposed Phase 2 study
RP is a relentless blinding disease with no current treatment. There are an estimated 10,000 patients with RP in California and all are either visually disabled already or expected to become so in time. Most or all these patients will need to receive healthcare benefits, special living assistance, and will also suffer from decreased economic functionality. Treatment for RP would be a medical breakthrough that may lead to treatment for other currently incurable blinding diseases.