The Center for Breakthrough Medicines signs deal to manufacture jCyte’s retinal degenerative disease cell therapy
jCell is a regenerative cell therapy for retinitis pigmentosa (RP) and other retinal degenerative diseases, which has received FDA Regenerative Medicine Advanced Therapy (RMAT) designation.
Under the terms of the agreement, CBM will provide Phase 3 clinical trial supplies and commercial drug product following Biologics License Application (BLA) approval of jCyte's proprietary jCell platform technology. CBM will also provide all BLA-enabling Chemistry, Manufacturing and Controls (CMC) work to support the program through regulatory approval and commercialization.
RP is a rare genetic disease that is typically diagnosed in children and adolescents. It causes progressive vision loss leading to legal blindness by early adulthood. Nearly 2 million people suffer from the disease worldwide, including approximately 100,000 people in the US, which makes RP the leading cause of inherited blindness. To date, there is no approved treatment for 99% of RP patients.
jCell is an investigational treatment for RP involving a simple, minimally invasive intravitreal injection, which can be administered within an ophthalmologist's office. The principal mechanism of action is the sustained release of established neurotrophic factors, which have been shown to reduce photoreceptor cell death and promote the function of surviving photoreceptors. The therapy aims to preserve vision by intervening in the disease process at a time when host photoreceptors' function can be protected and potentially restored.
Dr. Shannon Blalock, CEO of California-headquartered jCyte, said: "RP affects approximately one in 3,500 people worldwide. Our goals are to make jCell the first cell therapy approved to address this devastating condition and the first-line agent of choice. Partnering with a patient-focused, fully integrated, mission-centric CDMO like CBM will help ensure we achieve our goals."