[McGovern Institute for Brain Research at MIT]

Genome editing company Editas Medicine announced today the first administration of its therapy candidate EDIT-101 for the treatment of Leber congenital amaurosis 10 (LCA10), a CEP290-related retinal degenerative disorder. The BRILLIANCE clinical trial, designed to test the safety of EDIT-101, marks the first-ever delivery of an experimental CRISPR gene editing medicine to a pediatric patient.

“Administering the experimental medicine to the first pediatric patient in the BRILLIANCE trial marks a significant milestone toward delivering on the potential of CRISPR gene editing medicines being safe and effective in treating LCA10, which often results in significant vision loss and blindness early in life,” said James C. Mullen, chairman, president, and CEO, Editas in a press release. “Currently, there are no approved treatments for LCA10, and we look forward to sharing future updates from the BRILLIANCE trial, including sharing additional clinical data, later this year.”

Editas began enrolling pediatric patients in the mid-dose cohort of the BRILLIANCE trial following an Independent Data Monitoring Committee (IDMC) endorsement based on an analysis of safety data from a clinical trial in adult patients that tested low-dose and mid-dose levels of the experimental medicine. The mid-dose portion of the pediatric cohort is expected to be completed in the first half of this year, with pediatric high-dose testing set to begin before the end of 2022.

“Enrolling this first pediatric patient in the BRILLIANCE trial is an important step toward bringing potentially life-changing treatments to children with genetic retinal diseases. We are excited to be involved in research focused on testing potential new treatments for untreatable diseases like LCA10,” said trial principal investigator for the site, Tomas S. Aleman, MD, the Irene Heinz-Given and John LaPorte Research Associate Professor at the Scheie Eye Institute of the Perelman School of Medicine at the University of Pennsylvania, and a retinal degeneration specialist with the Division of Pediatric Ophthalmology at Children’s Hospital of Philadelphia (CHOP).

Prior to the start of pediatric testing of EDIT-101, Editas had completed dosing of all adult cohorts in its BRILLIANCE study. The clinical results from this arm of the trial demonstrated a favorable safety profile and encouraging signals of clinical benefit. The company said it expects to provide a clinical update on trial in the second half of 2022, which will provide safety and efficacy assessments on all adult patients who have had at least six months of follow-up evaluations. It will include at least 12 months of data on the adult mid-dose cohort, and at least six months of data on the adult high-dose cohort. Additionally, it is expanding enrollment in one or more of the previously completed adult cohorts to explore dose response and support establishment of registrational trial endpoints, also anticipated by year-end.

Editas Medicines was formed in 2013 and included CRISPR pioneers Feng Zhang of the Broad Institute and Jennifer Doudna of the University of California, Berkeley as co-founders, along with Harvard’s George Church, Keith Joung, and David Liu. Doudna later withdrew from the company over patent disputes concerning the CRISPR technology.

Editas is currently developing both in vivo and ex vivo therapeutics leveraging CRISR-Cas9 and CRISPR-Cas12 technologies in the areas of ocular diseases, blood diseases, and oncology. Other therapies in development include EDIT-103 for currently in IND-enabling study for Rhodopsin-Associated Autosomal Dominant Retinitis Pigmentosa (RHO-adRP) and EDIT-102 in Usher Syndrome 2A, EDIT-301 in two separate blood diseases, and EDIT-202: Multiplexed iNK for solid tumors in collaboration with BlueRock Therapeutics.

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