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Beam THERAPEUTICS Beam Therapeutics Provides Business and Pipeline Updates and Reports Second Quarter 2021 Financial Results August 10 , 2021 Company On - track to Submit First IND for BEAM - 101 in the Second Half of 2021 Continued Progress Across Base Editing Portfolio , Including Initiation of IND - Enabling Studies for BEAM - 201 CAMBRIDGE , Mass . , Aug. 10 , 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 second quarter 2021 financial results . " We have made meaningful progress in advancing our base editing programs in the first half of the year , and importantly , remain on track to submit our first investigational new drug ( IND ) for BEAM - 101 in the second half of this year , " said John Evans , chief executive officer of Beam . " With the initiation of IND - enabling studies for BEAM - 201 , we are now bringing the versatility and precision of base editing to a second therapeutic area , targeting the high unmet need in T - cell cancers with the first quad - edited cell therapy . We've also continued to expand our innovative collaborator network , most recently through our partnership with Apellis to apply base editing to the more biologically complex disease area of the complement pathway . With a strong balance sheet , we are well positioned to advance our robust pipeline of novel base editing programs through IND filings and into the clinic as we strive to provide potentially life - long cures for patients suffering from serious diseases . " Base Editing Pipeline Progress • IND - Enabling Studies for BEAM - 201 Initiated : Beam has initiated IND - enabling studies for BEAM - 201 for the treatment of relapsed / refractory T - cell acute lymphoblastic leukemia ( T - ALL ) . BEAM - 201 is a potent and specific allogeneic CAR - T targeting CD7 + malignancies , which uses multiplex base editing to simultaneously make four knockout edits at over 96 % efficiency for each gene . Relapsed / refractory T - ALL is a severe disease affecting children and adults with a five - year overall survival rate of less than 25 % . • Additional Upcoming Milestones in Ex Vivo and In Vivo Base Editing Pipeline On Track : Beam anticipates filing an IND for BEAM - 101 , which reproduces single base changes seen in individuals with Hereditary Persistence of Fetal Hemoglobin , or HPFH , to potentially protect them from the effects of mutations causing sickle cell disease ( SCD ) , and initiating IND - enabling studies for BEAM - 102 , which directly corrects the causative mutation in SCD by recreating a naturally - occurring normal human hemoglobin variant , both in the second half of 2021. Beam is also on track to nominate its first Development Candidate for in vivo base editing of the liver using LNP delivery by the end of 2021 . • Data Highlighting Base Editing Approach for the Treatment of Alpha - 1 Antitrypsin Deficiency Published in Molecular Therapy : In July 2021 , data from a preclinical study led by the Center for Regenerative Medicine at Boston Medical Center and Boston University evaluating Beam's base editors in induced pluripotent stem cells ( iPS cells ) from patients with alpha - 1 antitrypsin deficiency ( AATD ) were published in Molecular Therapy . As part of the study , the researchers utilized patient - derived liver cells that mimic the biology of liver hepatocytes , the main producers of alpha - 1 antitrypsin protein in the body , that likewise perform important metabolic , endocrine and secretory functions . The data showed that Beam's base editors corrected the mutation in the gene responsible for AATD and reduced the effects of the disease in the hepatocytes , demonstrating successful base editing in human cells . Upcoming Base Editing Data Presentations • Data from Sickle Cell Disease Program to be Presented at Cold Spring Harbor Laboratory's Genome Engineering : CRISPR Frontiers : Beam plans to present preclinical data highlighting its approach to using adenine base editing ( ABE ) for the treatment of sickle cell disease , including updated safety data during an oral session at the Cold Spring Harbor Laboratory's Genome Engineering : CRISPR Frontiers Conference . Details of the presentation are as follows : Title : Adenine base editing strategy for the treatment of sickle cell disease by elimination of the pathogenic globin protein Session : Disease / Therapeutic Date : Friday , August 20 , 2021 , 2:30 -5 : 30 p.m. ET • Updated Data on Optimization of LNP Delivery Approach to be Presented at TIDES 2021 : Beam plans to present