Year 4

In this past year the team has completed enrollment of the phase 1/2a clinical trial of retinal progenitor cells in retinitis pigmentosa (RP). Enrollment included 2 additional (higher) dose levels beyond the 2 levels originally proposed, with a total of 28 patients dosed. The trial will be completed in 2017.

A subset of enrolled patients have already completed the trial after 12 months of follow-up. An extension trial has been initiated to continue following those patients. To date the progenitor cell treatment has been well tolerated and data obtained has formed the basis for designing a follow-on phase 2b clinical trial.

In support of this next phase of clinical development, a clinical protocol was submitted to the FDA and passed their review. In addition, a CLIN2 grant application was submitted to CIRM and recently approved for funding. In parallel with the CLIN2 application, the spin-off company jCyte raised matching funds to be contributed to the conduct of this project.

The stage is now set for completion of phase 1/2a and initiation of phase 2b trial in 2017. The latter trial will be larger than the former and primarily directed towards demonstrating efficacy of the progenitor cell treatment.