Clinical Trials

Date: 2022-04-11

Type of information: Treatment of the first patient

phase: 1-2

Announcement: treatment of the first patient

Company: Editas Medicine (USA - MA)

Product: EDIT-101

Action mechanism: gene editing. EDIT-101 is a CRISPR/Cas9-based experimental medicine under investigation for the treatment of Leber congenital amaurosis 10 (LCA10), a CEP290-related retinal degenerative disorder. EDIT-101 is administered via a subretinal injection to reach and deliver the gene editing machinery directly to photoreceptor cells. EDIT-101 has been granted Rare Pediatric Disease and Orphan Drug designations from the FDA and Orphan Designation from the EMA.

Disease: Leber congenital amaurosis 10

Therapeutic area: Rare diseases - Genetic diseases - Ophthlalmological diseases

Country: USA

Trial details: 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 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. Patients are monitored every three months for a year after dosing and less frequently for an additional two years thereafter. (NCT03872479).

Latest news:

• On April 11, 2022, Editas Medicine, a leading genome editing company, announced the administration of EDIT-101, an experimental CRISPR gene editing medicine, to the first pediatric patient enrolled in the BRILLIANCE clinical trial, which is designed to test the safety of EDIT-101 for the treatment of Leber congenital amaurosis 10 (LCA10), a CEP290-related retinal degenerative disorder. This marks the world’s first in vivo, or inside the body, dosing of a pediatric patient with a CRISPR gene editing experimental medicine. Albert M. Maguire, MD, the F.M. Kirby Professor of Molecular Ophthalmology at Penn and a member of the Center for Advanced Retinal and Ocular Therapeutics, is the surgeon in the trial, in collaboration with Children’s Hospital of Philadelphia (CHOP), the nation's first hospital devoted exclusively to the care of children and the source of many breakthroughs and firsts in pediatric medicine. CHOP’s Clinical In Vivo Gene Therapy (CIGT) program provided the clinical operations support to conduct the work at CHOP. Editas Medicine initiated enrollment in the pediatric mid-dose cohort in the BRILLIANCE trial following the 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 company remains on track to complete testing of the pediatric mid-dose in the first half of 2022 and expects to initiate testing of the pediatric high-dose this year. Previously, Editas Medicine completed dosing of all adult cohorts in its BRILLIANCE study and announced preliminary EDIT-101 clinical results demonstrated a favorable safety profile and encouraging signals of clinical benefit. The company expects to provide a clinical update on the BRILLIANCE trial in the second half of 2022. The update is expected to provide safety and efficacy assessments on all adult patients who have had at least six months of follow-up evaluations, which 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, Editas Medicine is expanding enrollment in one or more of the previously completed adult cohorts to explore dose response and support establishment of registrational trial endpoints, which are anticipated by year-end.   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, Editas Medicine is focused on translating the power and potential of the CRISPR/Cas9 and CRISPR/Cas12a genome editing systems into a robust pipeline of treatments for people living with serious diseases around the world. Editas Medicine aims to discover, develop, manufacture, and commercialize transformative, durable, precision genomic medicines for a broad class of diseases. For the latest information and scientific presentations, please visit

Is general: Yes