In the past few months, jCyte has made a number of important announcements. In December, we received an $8.3 million grant from the California Institute for Regenerative Medicine (CIRM). In April, we announced our phase 2b clinical trial. That same month, we received Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food & Drug Administration (FDA). RMAT gives us better access to FDA expertise as we take our investigational retinitis pigmentosa (RP) therapy through clinical trials.
These announcements have generated interest from RP patients around the world and quite a few questions. We’d like to take this moment to give you the quick bullet on where jCyte is in the process and what all this means in the big picture.
Our investigational therapy (jCell) uses retinal progenitor cells to rescue rods and cones from RP progression and possibly generate new retinal cells. The treatment requires a single intravitreal injection, which can be performed in an ophthalmologist’s office with topical anesthetic. The entire procedure takes less than 30 minutes.
We have almost completed a phase 1/2a study to determine the therapy’s safety. Early results have been quite promising. So far, the treatment has been well-tolerated and has not generated a detectable immune response, a major concern when transplanting cells.
The Phase 2b Trial
On the strength of these results, jCyte has launched a phase 2b trial to test the treatment’s efficacy. The trial is enrolling 70 patients. After the trial is completed, we can determine next steps, which may include a phase 3 trial.
In addition to RMAT, jCell has received Orphan Drug designation from the FDA. These designations underscore the FDA’s interest in finding a therapy for RP, which currently has no medical treatments. The FDA and CIRM have been great partners in advancing jCell.
Many have asked how they can enroll in the clinical trial. Keep in mind, the study is being conducted exclusively in Southern California, and participants will need to come back to the clinic every three months to see if the therapy is working. If you have RP and are interested in the trial, you can send us an email or call 714-509-2151.
The Bottom Line
For people who have been waiting for an RP therapy for years or even decades, patience must seem like an obsolete concept. We get your emails and read your Facebook posts – you’re ready now.
However, we still need to go through the clinical trial process to determine whether jCell is an effective therapy. Our founders, Drs. Klassen and Yang, have been working on this problem for 20 years, and are determined to get jCell over the finish line.
How long will that take? We will know more soon. Stay tuned.