OHSU Casey Eye Institute grows gene and cell therapy research by performing its first stem cell therapy in the retina

Mark Pennesi, M.D., Ph.D., center right, and staff at OHSU's Casey Eye Institute perform the first-ever in vivo CRISPR gene edit procedure for the BRILLIANCE clinical trial.

The OHSU Casey Eye Institute has used a stem cell therapy in the retina for the first time as part of a Phase 2a clinical trial evaluating the safety and efficacy of an experimental treatment for an inherited blindness-causing disorder.

Above: OHSU Casey Eye Institute performed the first-ever CRISPR gene editing within the human body in early 2020 for the BRILLIANCE clinical trial. (OHSU/Kristyna Wentz-Graff)

The study is exploring the use of human retinal progenitor cells, which are early descendants of stem cells that can differentiate into retinal cells, but can’t divide or reproduce indefinitely. The study sponsor, ReNeuron, is exploring these cells as a potential way to treat retinitis pigmentosa, or RP.

The rare disorder is caused by mutations in over 90 known genes and involves the loss of light-sensitive cells in the back of the eye. Currently there is no effective treatment available for RP, which initially causes individuals to have trouble seeing at night, and later lose their peripheral vision. Patients often become legally blind in their 40s or 50s.

If the investigational therapy works as designed, the retinal progenitor cells will integrate into the retina, where they may secrete neurotrophic factors that could boost the retina’s visual function and slow disease progression. The human retinal progenitor cells could also possibly become new retinal cells that might enable the patient to regain lost vision.

OHSU is the third institution to provide the experimental therapy through their trial, ReNeuron reported in a Jan. 15, 2021, press release.

Mark Pennesi, M.D., Ph.D., chief of the Paul H. Casey Ophthalmic Genetics Division at the Casey Eye Institute, is OHSU’s lead researcher for the trial. Andreas Lauer, M.D., Thiele-Petti chair of  OHSU’s ophthalmology department, performed the procedure.

In early 2020, Pennesi and Lauer also led the first-ever CRISPR gene editing performed within the human body. That procedure was done for the BRILLIANCE clinical trial investigating a potential treatment for a rare form of inherited blindness called Leber congenital amaurosis type 10, OHSU announced in March 2020.

Franny WhiteFranny White
Senior Media Relations Specialist