jCyte's RPC Therapy Shows Promise in Phase I/IIa Study for Retinitis Pigmentosa

Monday, Aug 25, 2025 2:15 pm ET1min read
ALDX--

jCyte announced the publication of a Phase I/IIa safety study of retinal progenitor cells (RPCs) in retinitis pigmentosa. The study enrolled 28 adults with non-syndromic RP and found that intravitreal RPC injection was well tolerated at all dose levels. Exploratory efficacy data suggested potential vision benefits, including improved BCVA and patient-reported outcomes. The RPC technology has received FDA RMAT designation and ongoing trials will evaluate its efficacy and durability in a larger RP population.

jCyte, Inc. recently announced the publication of a Phase I/IIa safety study of retinal progenitor cells (RPCs) in retinitis pigmentosa (RP). The study enrolled 28 adults with non-syndromic RP and found that intravitreal RPC injection was well tolerated at all dose levels. Exploratory efficacy data suggested potential vision benefits, including improved best-corrected visual acuity (BCVA) and patient-reported outcomes. The RPC technology has received FDA Regenerative Medicine Advanced Therapy (RMAT) designation, and ongoing trials will evaluate its efficacy and durability in a larger RP population [1].

Retinitis pigmentosa is a rare genetic disorder that leads to progressive loss of the rod and cone photoreceptors in the retina, affecting approximately 2 million people worldwide, including about 100,000 individuals in the U.S. Current treatment options are limited, highlighting the significant unmet medical need in this patient population [1].

The Phase I/IIa study is a significant milestone for jCyte, as it demonstrates the safety and potential efficacy of its RPC technology in treating RP. The results provide a foundation for future trials that aim to evaluate the technology's long-term benefits and durability. The company's ongoing efforts to develop a breakthrough treatment for RP are promising, as it continues to address the significant unmet medical needs of patients suffering from this condition.

References:
[1] https://www.biospace.com/press-releases/jcyte-inc-announces-first-patients-treated-in-jc02-88-trial-for-retinitis-pigmentosa
[2] https://www.gurufocus.com/news/3070559/rocket-pharmaceuticals-announces-fda-has-lifted-the-clinical-hold-on-the-pivotal-phase-2-trial-of-rpa501-for-the-treatment-of-danon-disease-rckt-stock-news
[3] https://www.biospace.com/press-releases/aldeyra-therapeutics-receives-fast-track-designation-for-adx-2191-for-the-treatment-of-retinitis-pigmentosa

jCyte's RPC Therapy Shows Promise in Phase I/IIa Study for Retinitis Pigmentosa

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