Clinical Data from Editas Medicine’s Ongoing Phase 1/2 BRILLIANCE Clinical Trial of EDIT-101 for LCA10 to be Presented at the European Society of Gene and Cell Therapy Annual Congress
Editas Medicine announced that initial clinical data from the BRILLIANCE trial of EDIT-101 will be presented at the ESGCT Annual Congress, held virtually from October 19-22, 2021. EDIT-101 targets Leber congenital amaurosis 10 (LCA10), a retinal degenerative disorder associated with CEP290. Early efficacy signals from the mid-dose cohort indicate positive biological activity and a favorable safety profile. The BRILLIANCE trial assesses the safety, tolerability, and efficacy of EDIT-101, enrolling up to 18 patients across multiple dose levels.
- Initial clinical data from the BRILLIANCE trial suggest positive efficacy signals for EDIT-101.
- The trial shows a favorable safety profile for EDIT-101.
- None.
CAMBRIDGE, Mass., Oct. 20, 2021 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a leading genome editing company, today announced that an abstract featuring initial clinical data from the BRILLIANCE clinical trial of EDIT-101 has been selected for an oral presentation at the 2021 European Society of Gene and Cell Therapy (ESGCT) Annual Congress being held virtually October 19 – 22, 2021. EDIT-101 is under development for the treatment of Leber congenital amaurosis 10 (LCA10), a CEP290-related retinal degenerative disorder.
“We are pleased to be working at the forefront of this research with Editas Medicine, evaluating genome editing for the treatment of CEP290-associated retinal disease in the BRILLIANCE trial,” said Eric A. Pierce, M.D., Ph.D., Director of the Ocular Genomics Institute and William F. Chatlos Professor of Ophthalmology at Massachusetts Eye and Ear and Harvard Medical School, and the senior BRILLIANCE principal investigator. “I am highly encouraged by the early efficacy signals in the mid-dose cohort, which suggest positive biological activity and potential early clinical benefits. I am also very pleased that the initial data from the BRILLIANCE trial demonstrate a favorable safety profile. I believe that the trial data support continued EDIT-101 development as well as the evaluation of gene editing approaches for other inherited retinal disorders.”
Details of the Editas Medicine presentation can be accessed on the ESGCT website.
Oral Presentation:
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: Parallel 4b: Gene editing II
Date and Time: Thursday, October 21, 2021, 10:15 – 10:30 a.m. CET
Presenter: Dr. Eric A. Pierce, M.D., Ph.D., Director of the Inherited Retinal Disorders Service, Director of the Ocular Genomics Institute and William F. Chatlos Professor of Ophthalmology at Massachusetts Eye and Ear and Harvard Medical School, and a BRILLIANCE principal investigator.
About EDIT-101
EDIT-101 is a CRISPR-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.
About BRILLIANCE
The BRILLIANCE Phase 1/2 clinical trial of EDIT-101 for the treatment of Leber congenital amaurosis 10 (LCA10), a CEP290-related retinal degenerative disorder, 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. Patients are monitored every three months for a year after dosing and less frequently for an additional two years thereafter. 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 approximately 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 or a CEP290-related retinal degenerative disorder, 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 (also known as Cpf1) 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 www.editasmedicine.com.
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FAQ
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