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Beam THERAPEUTICS Beam Therapeutics Presents LNP Formulation Data at ASGCT and Reports First Quarter 2021 Financial Results May 11 , 2021 Data from Beam's Novel LNP - mR NA Formulation Demonstrates In Vivo Editing in Liver Cells of Non - human Primates Up to 52 % Company On - track to Submit First IND for BEAM - 101 in the Second Half of 2021 CAMBRIDGE , Mass . , May 11 , 2021 ( GLOBE NEWSWIRE ) --Beam Therapeutics Inc. ( Nasdaq : BEAM ) , a biotechnology company developing precision genetic medicines through base editing , today reported recent business highlights and pipeline updates , as well as first quarter 2021 financial results . " As Beam prepares to enter the clinic , we also continue to extend our leadership position in the field of base editing by expanding our platform and delivery capabilities , " said John Evans , chief executive officer of Beam . " The data presented at ASGCT on our proprietary lipid nanoparticle ( LNP ) formulation show efficient in vivo base editing in hepatocytes of non - human primates ( NHPs ) , while our novel analytical assays confirm specificity and efficiency of base editing to directly correct disease - causing mutations . As we look to the rest of 2021 , we are on - track with our plans to submit our first Investigational New Drug ( IND ) application for BEAM - 101 and initiate IND - enabling studies for BEAM - 102 and BEAM - 201 , as well as nominate our first development candidate from our liver portfolio . We are well positioned today to advance our base editing platform and pipeline and remain focused on achieving our vision of providing life - long cures for patients suffering from serious diseases . " " We are pleased to share our development of a proprietary LNP formulation for non - viral in vivo delivery to the liver at ASGCT , " said Giuseppe Ciaramella , Ph.D. , president and chief scientific officer of Beam . “ These data demonstrate that our LNPs are well tolerated in NHPs and can achieve levels of editing of hepatocytes that we expect would be therapeutic for many genetic diseases . Initial data also demonstrated the stability of our LNPS at - 20 degrees Celsius after 8 weeks , which is important for enabling broad clinical and commercial development . We are continuing to work on optimizing our LNP formulations and are on track to nominate our first development candidate from our liver portfolio in the second half of 2021. " Base Editing Progress • Data Demonstrating Optimization of LNP - mRNA Formulation for Liver Editing Presented at the 24th American Society of Gene and Cell Therapy ( ASGCT ) Annual Meeting : Beam's approach to developing a novel lipid nanoparticle ( LNP ) formulation for in vivo liver editing will be presented during a poster session titled , " Using Base Editing and LNP Delivery to Correct Disease - Causing Mutations Underlying Genetic Liver Diseases " at ASGCT . Using an mRNA - encoding adenine base editor ( ABE ) and guide RNA to target the ALAS1 gene , a surrogate payload for genetic liver diseases , Beam evaluated various LNP formulations and mRNA production processes to improve in vivo editing in the livers of NHPs from less than 10 % initially to 52 % at a total RNA dose of 1.5 mg / kg . These formulations were also well tolerated by NHPs at 1.5 mg / kg with mild and transient liver enzyme elevations , and showed promising interim stability , maintaining potency after 8 weeks at -20 ° C . • Data Highlighting Beam's Approach to Confirming Precision Correction Using Novel Analytical Assays Presented at ASGCT : Base editing is emerging as a powerful next - generation editing technology ; however , developing adequate analytical assays to confirm precise base editing at the protein level is critical and has proven historically challenging . In an oral presentation at ASGCT titled , " LC - MS Confirmation of Single Amino Acid Correction by Base Editing , " Beam will describe its approach to using liquid chromatography mass spectrometry ( LC - MS ) for multiple analytical assays to confirm the precise correction of disease - causing mutations at the amino acid level , providing a unique solution for confirmation and quantitation of single amino acid corrections after base editing . • Data Highlighting Ability to Rationally Design Base Editors for Precise Editing Published in The CRISPR Journal : In April 2021 , work describing Beam's approach to developing inlaid base editors ( IBES ) was published in The CRISPR Journal . IBEs are architectural variants of base editors that demonstrate enhanced specificity and altered activity windows relative to foundational base editors . The work highlights Beam's application of its IBES for BEAM - 102 , one of its base editing programs in development for the treatment of sickle cell disease . First Quarter 2021 Financial Results • Cash Position : Cash , cash equivalents and marketable securities were $ 503.5 million as of March 31 , 2021 , compared to $ 253.4 million as of March 31 , 2020 . • Research & Development ( R & D ) Expenses : R & D expenses were $ 190.1 million for the first quarter of 2021 , compared to