In a recent clinical trial, CRISPR gene editing was applied to 14 patients suffering from a form of inherited blindness. The treatment proved safe and resulted in measurable vision improvements in 11 of the participants. The trial, named BRILLIANCE, signifies a significant step forward in gene therapy for ocular diseases. Credit: SciTechDaily.com
Results from a groundbreaking clinical trial of CRISPR gene editing in 14 individuals with a form of inherited blindness show that the treatment is safe and led to measurable improvements in 11 of the participants treated. The phase 1/2 trial called BRILLIANCE, was led by principal investigator Eric Pierce, MD, PhD, of Mass Eye and Ear, a member of the Mass General Brigham healthcare system, and sponsored by Editas Medicine, Inc.
Eleven participants demonstrated improvements in at least one of those outcomes, while six demonstrated improvement in two or more. Four participants had clinically meaningful improvement in BCVA. Six participants experienced meaningful improvements in cone-mediated vision as indicated by FSTs, five of whom had improvements in at least one of the three other outcomes. Cone photoreceptors are used for daytime and central vision.
Jason Comander, MD, PhD, director of the Inherited Retinal Disorders Service at Mass Eye and Ear, examines the CRISPR-based medicine prior to performing a surgery of the novel treatment in September 2020, at Mass Eye and Ear in Boston. Credit: Mass Eye and EarThe first patient to receive a CRISPR treatment inside the body took place at the Casey Eye Institute at Oregon Health & Science University , under the leadership of Mark Pennesi, MD, PhD.
Principal investigator of the BRILLIANCE trial, Eric Pierce, MD, PhD, director of Ocular Genomics Institute and Berman-Gund Laboratory for the Study of Retinal Degenerations at Mass Eye and Ear and Harvard Medical School. Credit: Mass Eye and Ear
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