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editas MEDICINE Editas Medicine Announces First Quarter 2021 Results and Update May 5 , 2021 Strengthening Leadership by Adding Mark S. Shearman , Ph.D. , as Chief Scientific Officer Advancing BRILLIANCE trial of EDIT - 101 for LCA10 ; clinical data expected by year - end RUBY trial of EDIT - 301 for sickle cell disease active and recruiting Preclinical ocular data presented at ARVO supports in vivo gene editing Cash , cash equivalents , and marketable securities of $ 723 million as of March 31 , 2021 CAMBRIDGE , Mass . , May 05 , 2021 ( GLOBE NEWSWIRE ) -- Editas Medicine , Inc. ( Nasdaq : EDIT ) , a leading genome editing company , today reported business highlights and financial results for the first quarter of 2021 . " Our team is making tremendous progress towards discovering , developing , and manufacturing novel genome editing medicines , including excellent forward momentum this quarter for our two clinical - stage medicines , " said James C. Mullen , Chairman , President and Chief Executive Officer , Editas Medicine . " We are pleased to announce Dr. Mark Shearman will be joining Editas as Chief Scientific Officer in June 2021. Mark has an outstanding track record of drug discovery and development , and we are confident that his expertise will help us achieve our near - term milestones and continue to realize the promise of bringing transformative medicines to patients . " Mr. Mullen continued , " In ocular , we advanced the BRILLIANCE trial of EDIT - 101 for LCA10 and have line of sight for initiating the next cohort . We also presented preclinical data at ARVO that further validates our in vivo gene editing platform in USH2A and RP4 . In ex vivo , we advanced the RUBY trial of EDIT - 301 for sickle cell disease , including activating sites for recruitment and finalizing preparations to produce study drug material for dosing patients in the trial . We look forward to maintaining the momentum through the remainder of the year and expect to share Editas ' first clinical data for EDIT - 101 by year - end . " Recent Achievements and Outlook In Vivo Gene Edited Medicines • EDIT - 101 for Leber Congenital Amaurosis 10 ( LCA10 ) Initial clinical data expected by year - end Editas Medicine is progressing the Phase 1/2 BRILLIANCE trial for the treatment of LCA10 . The Company is continuing to dose patients in the second cohort , the adult mid - dose , and results of this cohort will inform the path to initiate dosing of pediatric patients . Clinical data from the first two cohorts is expected to be presented by the end of 2021 . • Other Ocular Programs Preclinical data for USH2A and RP4 presented at ARVO Editas Medicine presented preclinical data at the Association for Research in Vision and Ophthalmology ( ARVO ) 2021 Annual Meeting for Usher Syndrome 2A ( USH2A ) and retinitis pigmentosa 4 ( RP4 ) . For USH2A , the Company demonstrated that gene editing restored protein expression and rescued deficits in photoreceptor morphology in human retinal organoids . For RP4 , the Company demonstrated that a dual adeno - associated virus editing system , which simultaneously knocks out and replaces the rhodopsin mutation of RP4 , produced clinically relevant editing levels with no off - target editing . The Company expects to declare a development candidate for its RP4 program by the end of 2021 . Ex Vivo Gene Edited Medicines • EDIT - 301 for Sickle Cell Disease On track to dose first patient in RUBY trial by year - end Editas Medicine is developing EDIT - 301 using Cas12a ( Cpf1 ) , a proprietary enzyme , as a potentially best - in - class medicine to treat sickle cell disease . The Phase 1/2 RUBY trial for the treatment of sickle cell disease is active and recruitment of patients has started . The Company remains on track to begin patient dosing in the RUBY trial by the end of 2021 . • EDIT - 301 for Beta - Thalassemia