EDIT-101 demonstrates a favorable information illustration crossed each dose cohorts
Preliminary efficacy signals of accordant betterment successful BCVA positive further efficacy endpoints seen successful homozygous patients
Achieved impervious of conception and identified a responder colonisation
In presumption of the tiny population, the Company volition intermission enrollment successful the BRILLIANCE proceedings and question to place a collaboration spouse to proceed improvement of EDIT-101
Company to big a webinar contiguous astatine 8:00 a.m. ET
CAMBRIDGE, Mass., Nov. 17, 2022 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a objective signifier genome editing company, contiguous announced objective information from the Phase 1/2 BRILLIANCE proceedings of EDIT-101, an in vivo CRISPR/Cas9 genome editing medicine successful a Company-sponsored webinar. EDIT-101 is nether improvement for the attraction of blindness owed to Leber congenital amaurosis 10 (LCA10, a CEP290-related retinal degenerative disorder) and is designed to repair the IVS26 CEP290 mutant allele that impacts astir 1,500 LCA10 patients successful the U.S. There is nary effectual attraction presently disposable for this serious, uncommon disease. The BRILLIANCE update includes information and efficacy information from each 14 patients treated successful the survey to date, which includes 12 big patients and 2 pediatric patients.
Three retired of 14 treated subjects met a responder threshold having experienced clinically meaningful improvements successful champion corrected ocular acuity (BCVA) (LogMAR >0.3) and demonstrated accordant improvements successful 2 of the pursuing 3 further endpoints: afloat tract sensitivity trial (FST), ocular relation navigation people (VFN), oregon the ocular relation prime of beingness (VFQ).
An introspection of baseline characteristics of the attraction responder patients revealed that 2 of the 3 responders were homozygous for IVS26 mutation (2/2; 100% of the homozygous patients treated). No different baseline characteristics that could pre-select a responder diligent colonisation were identified successful the BRILLIANCE dataset.
EDIT-101 was tolerated with nary ocular superior adverse events oregon dose-limiting toxicities observed. Most adverse events were mild and expected for subretinal delivery.
Since LCA10 patients homozygous for CEP290 IVS26 mutation correspond an estimated colonisation of astir 300 successful the U.S., the Company volition not advancement this programme independently, and volition question to place a collaboration spouse to proceed the improvement of EDIT-101. As a result, Editas Medicine is pausing further enrollment successful the BRILLIANCE proceedings and volition proceed agelong word follow-up of each patients who person been treated to date.
“The results from the BRILLIANCE proceedings supply a impervious of conception and important learnings for our inherited retinal illness programs. We’ve demonstrated that we tin safely present a CRISPR-based cistron editing therapeutic to the retina and person clinically meaningful outcomes,” said Gilmore O’Neill, M.B., M.M.Sc., President and Chief Executive Officer, Editas Medicine. “While we volition not advancement EDIT-101 connected our ain and person made the determination to intermission enrollment, we person the diligent assemblage apical of caput and are looking for a collaboration spouse to beforehand this program.”
Webinar Information
Editas Medicine volition big a webinar today, Thursday, November 17, astatine 8:00 a.m. ET to contiguous the data. The unrecorded and archived webcast of the presumption volition beryllium accessible done this webcast link, oregon done the Events & Presentations leafage of the “Investors” conception of the Company’s website. The presumption volition besides beryllium disposable for download soon aft the webinar.
About EDIT-101
EDIT-101 is simply a CRISPR/Cas9-based experimental medicine nether probe for the attraction of Leber congenital amaurosis 10 (LCA10), by deleting the IVS26 CEP290 mutant allele. EDIT-101 is administered via a subretinal injection to scope and present the cistron editing machinery straight to photoreceptor cells. EDIT-101 has been granted Rare Pediatric Disease and Orphan Drug designations from the U.S. Food and Drug Administration (FDA) and Orphan Medicinal Product designation from the European Medicines Agency (EMA).
About BRILLIANCE
The BRILLIANCE Phase 1/2 objective proceedings of EDIT-101 for the attraction of Leber congenital amaurosis 10 (LCA10), a CEP290-related retinal degenerative disorder, is designed to measure the safety, tolerability, and efficacy of EDIT-101 successful up to 34 patients with this disorder. Clinical proceedings sites are enrolling up to 5 cohorts investigating up to 3 dose levels successful this unfastened label, multi-center study. Both big and pediatric patients (3 – 17 years old) with a scope of baseline ocular acuity assessments are eligible for enrollment. Patients person a azygous medication of EDIT-101 via subretinal injection successful 1 eye. Patients are monitored each 3 months for a twelvemonth aft dosing and little often for an further 2 years thereafter. Additional details are disposable connected www.clinicaltrials.gov (NCT#03872479).
About Leber Congenital Amaurosis
Leber Congenital Amaurosis, oregon LCA, is simply a radical of inherited retinal degenerative disorders caused by mutations successful astatine slightest 18 antithetic genes. It is the astir communal origin of inherited puerility blindness, with an incidence of astir 3 per 100,000 unrecorded births worldwide. Symptoms of LCA look wrong the archetypal years of life, resulting successful important imaginativeness nonaccomplishment and perchance blindness. The astir communal signifier of the disease, LCA10 oregon a CEP290-related retinal degenerative disorder, is simply a monogenic upset caused by mutations successful the CEP290 cistron and is the origin of illness successful astir 20-30 percent of each LCA patients.
About Editas Medicine
As a objective signifier genome editing company, Editas Medicine is focused connected translating the powerfulness and imaginable of the CRISPR/Cas9 and CRISPR/Cas12a genome editing systems into a robust pipeline of treatments for radical surviving with superior diseases astir the world. Editas Medicine aims to discover, develop, manufacture, and commercialize transformative, durable, precision genomic medicines for a wide people of diseases. Editas Medicine is the exclusive licensee of Broad Institute and Harvard University’s Cas9 patent estates and Broad Institute’s Cas12a patent property for quality medicines. For the latest accusation and technological presentations, delight sojourn www.editasmedicine.com.
Forward-Looking Statements
This property merchandise contains forward-looking statements and accusation wrong the meaning of The Private Securities Litigation Reform Act of 1995, including statements regarding the Company’s plans to question a collaboration spouse to proceed improvement of EDIT-101. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "target," "should," "would," and akin expressions are intended to place forward-looking statements, though not each forward-looking statements incorporate these identifying words. The Company whitethorn not really execute the plans, intentions, oregon expectations disclosed successful these forward-looking statements, and you should not spot undue reliance connected these forward-looking statements. Actual results oregon events could disagree materially from the plans, intentions and expectations disclosed successful these forward-looking statements arsenic a effect of assorted important factors, including: uncertainties inherent successful the initiation and completion of preclinical studies and objective trials, including the BRILLIANCE trial, and objective improvement of the Company’s merchandise candidates; the quality to found and support a collaboration connected favorable terms, if astatine each and the occurrence of immoderate specified collaboration that the Company enters into; availability and timing of results from preclinical studies and objective trials; whether interim results from a objective proceedings volition beryllium predictive of the last results of the proceedings oregon the results of aboriginal trials; expectations for regulatory approvals to behaviour trials oregon to marketplace products and availability of backing capable for the Company’s foreseeable and unforeseeable operating expenses and superior expenditure requirements. These and different risks are described successful greater item nether the caption “Risk Factors” included successful the Company’s astir caller Annual Report connected Form 10-K, which is connected record with the Securities and Exchange Commission, arsenic updated by the Company’s consequent filings with the Securities and Exchange Commission, and successful different filings that the Company whitethorn marque with the Securities and Exchange Commission in the future. Any forward-looking statements contained successful this property merchandise talk lone arsenic of the day hereof, and the Company expressly disclaims immoderate work to update immoderate forward-looking statements, whether due to the fact that of caller information, aboriginal events oregon otherwise.
English (US)