At A Race Against Blindness, we closely follow scientific advances that bring new hope to families affected by inherited retinal diseases like Bardet-Biedl Syndrome (BBS) and retinitis pigmentosa (RP). One of the most promising efforts comes from jCyte, Inc., which recently announced that the first patients have been treated in its JC02-88 clinical trial of jCell (famzeretcel) — a potential regenerative therapy for RP.
Why This Trial Matters
Retinitis pigmentosa is a rare genetic condition that causes gradual loss of the rod and cone cells in the retina, leading to progressive vision loss. Symptoms often begin in adolescence, with many individuals becoming legally blind by midlife. More than 2 million people worldwide — including about 100,000 in the U.S. — are affected.
Currently, there are no approved treatments for most RP patients. That’s why this new trial marks such an important milestone.
What Is jCell?
jCell is an innovative allogeneic cell therapy designed to protect and possibly restore vision in people with RP. Unlike many gene therapies that are tailored to specific mutations, jCell could work regardless of genetic subtype. The treatment is delivered through a single intravitreal injection — a minimally invasive procedure performed in the eye clinic.
Inside the JC02-88 Trial
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Phase 2 study enrolling up to 60 patients aged 18–60 with RP.
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Participants will receive either one injection of 8.8 million jCell or a sham treatment (placebo).
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Researchers will track safety, tolerability, and vision outcomes over 6 months.
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All patients are treated at the UC Irvine Gavin Herbert Eye Institute.
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After the main study, an extension trial will allow longer-term monitoring and treatment for those initially assigned to the control group.
This trial also represents the highest dose of jCell tested to date — about 50% higher than previous studies.
Expert Voices on the Trial
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John Sholar, CEO of jCyte: “This marks an important milestone in our mission to bring a breakthrough treatment to the majority of RP patients who currently have limited treatment options.”
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Dr. Paul Sieving, UC Davis & past Director of the National Eye Institute: “This is an exciting development for the RP community, and I am eager to see how this therapy advances toward becoming a novel cell-based treatment.”
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Dr. Henry Klassen, Co-Founder of jCyte: “Our team’s dedication has brought us to this point, and we remain committed to delivering a therapy that can make a meaningful difference in patients’ lives.”
Looking Ahead
If successful, jCell could become the first treatment option for the vast majority of RP patients. With support from the California Institute for Regenerative Medicine (CIRM) and research partners like the Gavin Herbert Eye Institute and Lexitas, this trial is paving the way toward a new era of regenerative therapies.
For more information about the study, visit the official trial page: JC02-88 Clinical Trial.
✨ Why We’re Sharing This:
At A Race Against Blindness, our mission is to accelerate treatments and cures for BBS and related retinal diseases. Breakthroughs like jCell remind us that hope is on the horizon — and every advance in rare retinal research benefits the entire vision loss community.