Name:
Institution:
Type:
PI
Grant Award Details
Phase 2b Clinical Study of Safety and Efficacy of Intravitreal Injection of Retinal Progenitor Cells (jCell) for Treatment of Retinitis Pigmentosa
Grant Type:
Grant Number:
CLIN2-09698
Project Objective:
Complete a Phase 2b Clinical Study of Safety and Efficacy of Intravitreal Injection of Retinal Progenitor Cells (jCell) for Treatment of Retinitis Pigmentosa
Investigator:
Disease Focus:
Retinitis Pigmentosa
Vision Loss
Human Stem Cell Use:
Other
Cell Line Generation:
Adult Stem Cell
Award Value:
$8,295,750
Status:
Active
Grant Application Details
Application Title:
- Phase 2b Clinical Study of Safety and Efficacy of Intravitreal Injection of Retinal Progenitor Cells (jCell) for Treatment of Retinitis Pigmentosa
Public Abstract:
Therapeutic Candidate or Device
Allogeneic human retinal progenitor cells (hRPC)
Indication
Retinitis Pigmentosa (RP)
Therapeutic Mechanism
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. Todate, there is nothing that will restore sight or slow the progression of vision loss in RP. Achieving any measurable benefits would be groundbreaking.
Project Objective
Phase 2 trial completed
Major Proposed Activities
Enrollment of patients in a Phase 2b clinical trial, along with patient follow up and collection of all clinical outcome measures.
Statement of Benefit to California:
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.
