Initial Clinical Data from Editas Medicine’s BRILLIANCE Clinical Trial of EDIT-101 for LCA10 to be Presented at the International Symposium on Retinal Degeneration in September
Data to include patient safety assessments and a preliminary analysis of secondary endpoints to evaluate biological activity
Abstract selected for oral presentation on September 29
Company to host webcast investor event following the presentation on
“We look forward to sharing our Company’s first clinical data at RD2021 and our progress towards developing a transformative gene editing medicine for people living with CEP290-related retinal degeneration. The presentation will include an evaluation of clinical data from the first two adult cohorts as the study continues into the pediatric mid-dose and adult high-dose cohorts,” said
The presentation will include patient safety assessments and a preliminary analysis of secondary endpoints relating to signals of gene editing and clinical benefit. Cumulative data from patients in the adult low-dose and mid-dose cohorts and will be presented by one of the study’s Principal Investigators, Dr.
Full details of the
Title: BRILLIANCE: A Phase 1/2 Single Ascending Dose Study of EDIT-101, an in vivo CRISPR Gene Editing Therapy, in CEP290-Related Retinal Degeneration
Session Title: Platform Session V: Clinical Trials
Date and Time: Wednesday, September 29, 2021, 9:05 –
Investor Event and Webcast Information
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,
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Source: Editas Medicine, Inc.