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Beam THERAPEUTICS Beam Therapeutics Provides Business and Pipeline Updates and Reports Third Quarter 2021 Financial Results November 8 , 2021 BEAM - 101 IND Cleared by FDA for Evaluation as a Treatment for Sickle Cell Disease BEAM - 102 IND - Enabling Studies Also Underway CAMBRIDGE , Mass . , Nov. 08 , 2021 ( GLOBE NEWSWIRE ) Beam Therapeutics Inc. ( Nasdaq : BEAM ) , a biotechnology company developing precision genetic medicines through base editing , today reported recent business and pipeline updates , as well as third quarter 2021 financial results . As part of today's update , Beam announced that its Investigational New Drug ( IND ) application for BEAM - 101 for the treatment of sickle cell disease was cleared by the U.S. Food and Drug Administration ( FDA ) . BEAM - 101 , the company's lead ex vivo base editing product candidate , is a patient specific , autologous hematopoietic investigational cell therapy which incorporates base edits that mimic single nucleotide polymorphisms seen in individuals with hereditary persistence of fetal hemoglobin ( HPFH ) to potentially alleviate the effects of mutations causing sickle cell disease or beta - thalassemia . This is the first open IND for base editing technology , a next - generation form of CRISPR capable of making single base changes without creating double strand breaks in the DNA . Beam is preparing to initiate a Phase 1/2 clinical trial designed to assess the safety and efficacy of BEAM - 101 for the treatment of sickle cell disease , which Beam refers to as the BEACON - 101 trial . -- " We are thrilled to share that the FDA has cleared our first IND . BEAM - 101 has the potential to offer a one - time treatment for patients with sickle cell disease , and this clearance enables the important transition from a preclinical to a clinical - stage company , bringing us closer to our ultimate goal of helping patients , " said John Evans , chief executive officer of Beam . " As leaders in the field of base editing , this milestone underscores the expertise of our team and the significant potential of our technology . We are grateful to the team members who have dedicated countless hours to this effort , and we look forward to this program's continued evaluation in the clinic . As we look toward 2022 , we believe we are well - positioned both financially and organizationally to execute on our vision . " " In addition to the clearance of our IND for BEAM - 101 , we are pleased to announce that we have initiated IND - enabling studies for BEAM - 102 , our Makassar base editing approach for the treatment of sickle cell disease , " said Giuseppe Ciaramella , Ph.D. , president and chief scientific officer of Beam . " We have also made continued progress on BEAM - 201 , our multiplexed base editing approach for the treatment of relapsed / refractory T - cell acute lymphoblastic leukemia and T - cell lymphoblastic leukemia as well as continued advancement across our robust portfolio , as showcased by a number of recent and upcoming data presentations demonstrating our leadership in base editing as well as optimized delivery technologies for our programs . Importantly , we also expect to nominate our first development candidate for in vivo base editing in the liver using LNP delivery for the treatment of patients with glycogen storage disease type la ( R83C mutation ) by the end of the year . We've made extraordinary progress as a company this year and remain committed to advancing novel science so that we may reach as many patients as possible . " Upcoming Base Editing Data Presentations • Preclinical data highlighting potential of CAR T multiplex base editing approach targeting CD5 at 2021 SITC Annual Meeting . At the Society for Immunotherapy of Cancer's ( SITC ) 36th Annual Meeting , Beam will present preclinical data from its multiplex edited allogeneic CAR T research targeting CD5 - positive hematologic malignancies in a poster titled " CD5 knockout enhances the potency of multiplex base - edited allogeneic anti - CD5 CAR T - cell therapy for the treatment of T - cell malignancies . " • Multiple abstracts accepted for presentation at the 63rd American Society of Hematology Annual Meeting & Exposition ( ASH ) . At this year's ASH meeting , Beam will present preclinical data highlighting its base editing approach to address sickle cell disease , including new preclinical data for BEAM - 102 , and data highlighting Beam's in vivo high - throughput lipid nanoparticle ( LNP ) screening approach to identify novel LNPs that can deliver base editors to tissues beyond the liver , such as hematopoietic stem cells . In addition , Beam will present an overview on the application of base editing for the treatment of beta - hemoglobinopathies and other genetic blood disorders during a scientific program session . Details of the presentations can be found here . Recent Base Editing Progress & Data Updates • BEAM - 102 IND - enabling studies initiated . Beam has initiated IND - enabling studies for BEAM - 102 for the treatment of sickle cell disease . BEAM - 102 aims to directly correct the causative mutation in sickle cell disease by recreating a naturally - occurring normal human hemoglobin variant , HbG Makassar . • Preclinical data highlighting base editing approach to address GSDla presented at European Society of Gene & Cell Therapy . Beam recently presented preclinical data demonstrating the ability of its liver - targeted base editing approach to directly correct R83C , one of the primary disease - causing mutations of glycogen storage disease type la ( GSDla ) , and