Editas Medicine Announces Enrollment of the First Pediatric Cohort in the BRILLIANCE Clinical Trial of EDIT-101 for the Treatment of LCA10 Following IDMC Endorsement
IDMC endorsed proceeding with first pediatric cohort based on a review of clinical safety data from adult low-dose and adult mid-dose cohorts
Concurrently enrolling the adult high-dose cohort in the BRILLIANCE trial
Company plans to report initial EDIT-101 clinical data in September
“We are extremely pleased with the endorsement of the IDMC, and we look forward to bringing EDIT-101 to additional patients in the BRILLIANCE trial. LCA10 is an early onset retinal degenerative disease resulting in significant vision loss and blindness. Positive feedback from the IDMC is a critical step to move to initiate EDIT-101 dosing in pediatric patients,” said
“We have a long history at Mass. Eye and Ear of helping develop life-changing medicines, and we are pleased to be working at the forefront of research on this novel therapy. We are eager to begin studying EDIT-101 in pediatric patients where we believe it can have great impact and the potential to restore vision,” said
The Company plans to present initial clinical data from the BRILLIANCE clinical trial at an upcoming medical meeting and is currently submitting an abstract to the International Symposium on Retinal Degeneration (RD2021) planned for September in
EDIT-101 is a CRISPR-based experimental medicine under investigation for the treatment of Leber congenital amaurosis 10 (LCA10). EDIT-101 is administered via a subretinal injection to reach and deliver the gene editing machinery directly to photoreceptor cells.
The BRILLIANCE Phase 1/2 clinical trial of EDIT-101 for the treatment of Leber congenital amaurosis 10 (LCA10) is designed to assess the safety, tolerability, and efficacy of EDIT-101 in up to 18 patients with this disorder. Clinical trial sites are enrolling up to five cohorts testing up to three dose levels in this open label, multi-center study. Both adult and pediatric patients (3 – 17 years old) with a range of baseline visual acuity assessments are eligible for enrollment. Patients receive a single administration of EDIT-101 via subretinal injection in one eye. Additional details are available on www.clinicaltrials.gov (NCT#03872479).
About Leber Congenital Amaurosis
Leber Congenital Amaurosis, or LCA, is a group of inherited retinal degenerative disorders caused by mutations in at least 18 different genes. It is the most common cause of inherited childhood blindness, with an incidence of two to three per 100,000 live births worldwide. Symptoms of LCA appear within the first years of life, resulting in significant vision loss and potentially blindness. The most common form of the disease, LCA10, is a monogenic disorder caused by mutations in the CEP290 gene and is the cause of disease in approximately 20-30 percent of all LCA patients.
About Editas Medicine
As a leading genome editing company,
This press release contains forward-looking statements and information within the meaning of The Private Securities Litigation Reform Act of 1995. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "target," "should," "would," and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Forward-looking statements in this press release include statements regarding the expected first dosing of both the pediatric mid-dose cohort and the adult high-dose cohort in the BRILLIANCE trial this summer and completion of dosing of these cohorts in the first half of 2022, and the Company’s plan to report initial EDIT-101 clinical data at a medical meeting in
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Source: Editas Medicine, Inc.