Credit: SciTechDaily.comMass Eye and Ear-led phase 1/2 trial, which included 14 individuals, found that the first-of-its-kind experimental treatment was safe and efficacious.BRILLIANCE trial outcomes revealed 11 out of 14 cured participants experienced some improvements in vision and quality of life measures.CRISPR-based therapy was discovered safe with no dose-limiting toxicities reported.Mass Eye and Ear researchers say their findings support continued research study and clinical trials of CRISPR treatments for acquired retinal disorders.Results from a groundbreaking clinical trial of CRISPR gene modifying in 14 individuals with a form of acquired loss of sight show that the treatment is safe and led to measurable enhancements in 11 of the participants treated. Credit: Mass Eye and EarParticipant Demographics and Trial ProceduresAll 14 trial individuals, consisting of 12 grownups (ages 17 to 63) and two kids (ages 10 and 14), were born with a type of Leber Congenital Amaurosis (LCA) triggered by mutations in the centrosomal protein 290 (CEP290) gene. Jason Comander, MD, PhD, director of the Inherited Retinal Disorders Service at Mass Eye and Ear, takes a look at the CRISPR-based medication prior to performing a surgical treatment of the unique treatment in September 2020, at Mass Eye and Ear in Boston. Additional individuals were treated across three other trial sites: Bascom Palmer Eye Institute, W.K. Kellogg Eye Center, and Scheie Eye Institute at the Childrens Hospital of Philadelphia (CHOP) and the Hospital of the University of Pennsylvania. This research study was likewise supported by the National Institute of Health P30 EY014104 core grant to Mass Eye and Ear, P30 EY010572 core grant, the Malcolm M. Marquis MD Endowed Fund for Innovation, and an unrestricted grants from Research to Prevent Blindness to Casey Eye Institute and the Scheie Eye Institute.
Credit: SciTechDaily.comMass Eye and Ear-led stage 1/2 trial, which consisted of 14 participants, found that the first-of-its-kind experimental treatment was efficacious.brilliance and safe trial outcomes showed 11 out of 14 cured individuals experienced some improvements in vision and quality of life measures.CRISPR-based therapy was found safe with no dose-limiting toxicities reported.Mass Eye and Ear researchers say their findings support continued research study and medical trials of CRISPR treatments for inherited retinal disorders.Results from a groundbreaking medical trial of CRISPR gene modifying in 14 individuals with a kind of inherited blindness show that the treatment is safe and led to quantifiable improvements in 11 of the individuals treated. Jason Comander, MD, PhD, director of the Inherited Retinal Disorders Service at Mass Eye and Ear, takes a look at the CRISPR-based medicine prior to performing a surgery of the novel treatment in September 2020, at Mass Eye and Ear in Boston. Additional participants were treated throughout 3 other trial websites: Bascom Palmer Eye Institute, W.K. Kellogg Eye Center, and Scheie Eye Institute at the Childrens Hospital of Philadelphia (CHOP) and the Hospital of the University of Pennsylvania.
