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Stem Cell Therapy Licensed for Genetic Eye Disease

Red-blue-green eye

(Public Domain Pictures, Pixabay)

8 May 2020. A biotechnology company is licensing its stem cell treatments for retinitis pigmentosa, an inherited eye disease, in a deal valued at $252 million. The agreement gives Santen Pharmaceutical, in Osaka, Japan, a drug and medical device maker specializing in ophthalmology, rights to technology developed by jCyte Inc. in Newport Beach, California to further develop and commercialize as products in Europe and Asia.

jCyte’s technology, called jCell, produces retinal progenitor cells, a type of stem cell that transforms into healthy cells found in the retina, the sensory layer lining the back of the eye. The retina has photoreceptor cells that convert light into signals carried by the optic nerve into the visual cortex of the brain for interpretation. The company’s first therapy target is retinitis pigmentosa, a progressive genetic disorder that destroys photoreceptor cells over time leading to total blindness in some cases.

Retinal progenitor cells, says jCyte, give off growth factors, proteins that help limit the damage to photoreceptor cells and slow the progression of retinitis pigmentosa. These progenitor cells also reside in the eye for an extended period of time, allowing the cells to transform into healthy retinal cells and tissue, making it possible to reverse the otherwise unstoppable disease. In addition, jCells also do not provoke an immune response, making them safer for patients and not needing other drugs to suppress the immune system.

The company says jCell treatments consist of a single injection that can be given in a doctor’s office in about 30 minutes. In May 2017, jCells received a regenerative medicine advanced therapy designation from FDA, designed to expedite review of promising regenerative treatments.

jCyte is completing a mid-stage clinical trial of jCells among 84 adults at three sites with retinitis pigmentosa. Participants are randomly assigned to receive an injection with one of two dose levels of jCell or a simulated injection for comparison, then are evaluated for the next 12 months. The study team from jCyte and California Institute for Regenerative Medicine, an early backer of the company, are looking primarily for changes in visual acuity, but also for other vision indicators, changes in mobility and quality of life, and safety signs.

Santen develops and acquires through licensing treatments for a number of eye disorders including glaucoma and ocular hypertension, a contributing factor to glaucoma. The global company also develops digital devices and diagnostics for eye health. Under the agreement with jCyte, Santen acquires a license for developing and commercializing jCells in Japan, elsewhere in Asia, and Europe.

In return, jCyte receives an initial cash payment of $50 million and a convertible note — a short-term loan payable in equity — of $12 million. jCyte also is eligible for $190 million in clinical, regulatory, and sales milestone payments, as well as royalties on sales of products in the licensed regions. Paul Bresge, CEO of jCyte, says in a statement, “We intend to use the proceeds from this transaction to continue development of our lead investigational therapy jCell, to improve the lives of patients with retinitis pigmentosa, as well as other degenerative retinal diseases.”

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