Beam Therapeutics Names First CAR-T Base Editing Development Candidate for the Treatment of T-ALL and Presents New Data at SITC 2020 - Seite 2

Details of Beam’s SITC 2020 presentation of BEAM-201 are as follows:

Title: Highly efficient multiplexed base editing enables the development of investigational universal CD7-targeting CAR-T Cells to treat T-ALL
Publication Number: 111
Category: Cellular Therapies
Data Summary:

• In vitro characterization of the effects of base editing in BEAM-201 demonstrated:
  • Simultaneous base editing by a cytosine base editor (CBE) at four target loci in primary human T cells using a clinical-scale process produced 96-99% on-target editing of each of the four genes as measured by next-generation sequencing and flow cytometry;
  • Simultaneous quad base editing of T cells resulted in no detected genomic rearrangements resulting from the editing process;
  • Multiplex base editing did not negatively affect cell expansion during manufacturing;
  • CBE-edited cells decreased expression of the four target genes with minimal effect on other genes, including key members of the p53 pathway that are upregulated in response to DNA double-stranded breaks produced by multiplex editing with nucleases.
• Further characterization of BEAM-201 in vitro and in a tumor mouse model demonstrated:
  • Beam’s GMP-compliant, clinical-scale process resulted in final BEAM-201 CAR-T cell populations with on-target editing efficiencies between 96-99.9% at each of the four target loci, and 85% CAR-expressing cells. As a result, Beam estimates that 91% of cells are bi-allelically quad base edited and 77% of cells have all 5 genetic modifications. The company believes this is the highest level and uniformity of CAR expression and simultaneous editing across four target sites reported at clinical scale to date.
  • BEAM-201 cells demonstrated robust in vitro CD7-dependent cytokine production, and rapid in vitro cytotoxicity.
  • BEAM-201 cells also demonstrated dose-dependent clearance or control, across a 25-fold dose range, of an aggressive disseminated CCRF-CEM T-ALL tumor mouse model. 

About Beam Therapeutics
Beam Therapeutics (Nasdaq: BEAM) is a biotechnology company developing precision genetic medicines through the use of base editing. Beam’s proprietary base editors create precise, predictable and efficient single base changes, at targeted genomic sequences, without making double-stranded breaks in the DNA. This enables a wide range of potential therapeutic editing strategies that Beam is using to advance a diversified portfolio of base editing programs. Beam is a values-driven organization committed to its people, cutting-edge science, and a vision of providing life-long cures to patients suffering from serious diseases. For more information, visit www.beamtx.com.