Benzinga | Jan 9, 2022 11:44AM EST

Beam Therapeutics Announced Progress Across Ex Vivo and In Vivo Pipeline of Base Editing Therapeutics and Outlines Key Anticipated 2022 Milestones Said First Subject Anticipated to be Enrolled in BEAM-101 Phase 1/2 Clinical Trial for the Treatment of Sickle Cell Disease in H2 2022

Beam Therapeutics Inc. (NASDAQ:BEAM), a biotechnology company developing precision genetic medicines through base editing, today outlined anticipated 2022 milestones across its ex vivo programs targeting editing of hematopoietic stem cells (HSCs) and T cells and in vivo programs targeting editing of liver cells leveraging lipid nanoparticles (LNPs) for delivery. Updates include that the company has selected its fourth development candidate and first in vivo base editing candidate, BEAM-301, which aims to correct the R83C mutation for the potential treatment of patients with glycogen storage disorder Ia (GSDIa).

"We made significant progress across our base editing portfolio in 2021, which culminated in U.S. Food and Drug Administration clearance of the first investigational new drug application of a base editing therapeutic, BEAM-101. We also further expanded our platform, particularly with LNP delivery of base editors to the liver and our proprietary technology for accelerating LNP delivery to other tissues, including HSCs," said John Evans, chief executive officer of Beam. "We believe 2022 is set to be our most important year yet, with preparations underway to launch the BEACON-101 clinical trial with BEAM-101 for the treatment of sickle cell disease and to complete our transition to becoming a clinical-stage company. We believe we are well positioned today, with four development candidates, a rich pipeline of earlier stage programs, and an industry-leading platform of editing and delivery technologies enabling us to bring forward a new class of precision genetic medicines. None of this would be possible without the commitment of our remarkable team of fearless innovators. We look forward to the year ahead and continuing our work to bring potentially life-changing medicines to as many patients as possible."

Ex Vivo HSC Programs

BEAM-101 is a patient-specific, autologous HSC investigational therapy, which incorporates base edits that are designed to mimic single nucleotide polymorphisms seen in individuals with hereditary persistence of fetal hemoglobin. BEAM-101 aims to potentially alleviate the effects of mutations causing sickle cell disease (SCD) or beta-thalassemia by leading to increases in fetal hemoglobin, which inhibits hemoglobin S (HbS) polymerization. The BEACON-101 trial is a Phase 1/2 clinical trial designed to assess the safety and efficacy of BEAM-101 for the treatment of SCD. The trial is expected to include an initial "sentinel" cohort of three patients, treated one at a time to confirm successful engraftment, followed by dosing in up to a total of 45 patients. Beam has begun site selection and the institutional review board approval processes for the BEACON-101 trial and plans to enroll the first subject in the second half of 2022.BEAM-102 is designed to treat SCD by directly editing the causative HbS point mutation to recreate a naturally occurring normal human hemoglobin variant, HbG-Makassar. The Makassar variant has been reported to have the same function as the more common HbA variant and does not cause SCD. Beam plans to submit an investigational new drug (IND) application for BEAM-102 in the second half of 2022.Ex Vivo T Cell Programs

BEAM-201 is a multiplex base edited anti-CD7 CAR-T cell investigational therapy designed to treat relapsed/refractory T cell acute lymphoblastic leukemia, a severe disease affecting children and adults. Beam plans to submit an IND application for BEAM-201 in the second half of 2022.Beam plans to nominate a second CAR-T development candidate in 2022.In Vivo LNP Liver-targeting Programs

BEAM-301, the company's newest development candidate, is a liver-targeting LNP formulation of base editing reagents designed to correct the R83C mutation. R83C is the most common disease-causing mutation of GSDIa, a life-altering genetic disease with no approved disease-modifying treatments available today. Beam anticipates initiating IND-enabling studies for BEAM-301 in 2022.Beam plans to nominate a second liver-targeted development candidate in 2022.J.P. Morgan Healthcare ConferenceMr. Evans will present Beam's pipeline and business updates during a presentation at the 40th Annual J.P. Morgan Healthcare Conference on Monday, January 10, 2022, at 2:15 p.m. ET. A live webcast will be available in the investor section of the company's website at www.beamtx.com and will be archived for 60 days following the presentation.