Developing groundbreaking therapies can be maddeningly slow, but jCyte recently took a big step forward, receiving an $8.3 million grant from the California Institute for Regenerative Medicine (CIRM) to help fund a phase 2b trial for our retinitis pigmentosa therapy. The trial will investigate safety and efficacy and will recruit around 70 patients.
Granted orphan drug status by the Food & Drug Administration, our therapy (jCell) uses retinal progenitor cells (RPCs) to rescue diseased retinal cells and possibly regenerate new ones. This non-surgical treatment requires a simple intravitreal injection.
Early results from jCyte’s ongoing phase 1/2a trial, which is also being supported by a CIRM grant, have shown the therapy is safe, with virtually no adverse effects and no evidence of any immune response to the injected RPCs. The new trial will continue to assess the treatment’s safety, as well as its efficacy.
After carefully considering the jCyte proposal, CIRM’s grant review working group gave the therapy a powerful endorsement, with 11 of 15 members saying the application had “exceptional merit and warrants funding.” In a statement released during their December 13 meeting the working group added:
“The proposed treatment approach holds the potential to address a clear and urgent unmet medical need, improve the standard of care and offer a strong value proposition for this patient population. Further, the investigators leading this study are exceptional and have proposed a well-designed trial to generate useful data that will further inform clinical development of this treatment.”