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
June 23, 2021 at 07:00 am EDT
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Editas Medicine, Inc. announced that centers are now enrolling the first of two planned pediatric cohorts in the Phase 1/2 BRILLIANCE clinical trial of EDIT-101 following the Independent Data Monitoring Committee (IDMC) endorsement to proceed. The IDMC endorsement is based on an analysis of safety data from the adult low-dose and adult mid-dose cohorts. The Company also announced the completion of dosing of the adult mid-dose cohort. Both the pediatric mid-dose cohort and the adult high-dose cohort will run concurrently. For both cohorts, first dosing is expected this summer and completion is expected in the first half of 2022. EDIT-101 is under development for the treatment of Leber congenital amaurosis 10. 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 planned for September in Nashville.
Editas Medicine, Inc. is a clinical-stage genome editing company. The Company is focused on developing potentially transformative genomic medicines to treat a broad range of serious diseases. It has developed a proprietary gene editing platform based on CRISPR technology. CRISPR uses a protein- ribonucleic acid (RNA) complex composed of an enzyme, including either CRISPR associated protein 9 (Cas9) or Cas12a (CRISPR from Prevotella and Francisella 1, also known as Cpf1), bound to a guide ribonucleic acid (RNA) molecule designed to recognize a particular deoxyribonucleic acid (DNA) sequence. It is engaged in the development of vivo administered gene editing medicines, in which the medicine is injected or infused into the patient to edit the cells inside their body. Its lead program, reni-cel, is an experimental ex vivo gene-edited medicine to treat sickle cell disease (SCD), a severe inherited blood disease that causes premature death, and transfusion-dependent beta thalassemia (TDT).
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