Highlights of Beam Therapeutics
- Biotechnology company developing genetic medicine
- Use proprietary CRISPR based editing technology
- Portfolio includes CAR-T cells for leukemia
- Exvivo editing for sickle cell disease
- In vivo editing for Wilson Disease
- Cash or Cash equivalents $202.2 million
Beam Therapeutics (Nasdaq: BEAM)
Platform and Delivery System
The novel base editing approach uses a chemical reaction designed to create precise, predictable, and efficient genetic outcomes at the targeted DNA sequence. By rewriting a single base in the genome, base editors enable a diverse toolkit that could therapeutically intervene to correct disease-causing point mutations, modify genes to create protective genetic variations, activate gene expression, silence gene expression, or “multiplex” and make multiple simultaneous edits.The clinically validated technologies are used for delivery to these genomic medicines. They are
- electroporation,
- non-viral and
- viral delivery modalities
Beam Therapeutics Platform & Therapeutic Areas
Sickle Cell Disease and Beta Thalassemia
Acute Myeloid and Lymphoblastic Leukemia
Liver Diseases
Ocular Diseases
Base Clinical/Research Program Update
Base Clinical/Research Program Update
BEAM-201 for Treatment of T-ALL
Beam is advancing BEAM-201, a potent and specific anti-CD7 CAR-T multiplex editing program for the treatment of relapsed/refractory T-cell acute lymphoblastic leukemia (T-ALL), a severe disease affecting children and adults with five-year overall survival of less than 25%.
BEAM-201 is produced using a GMP-compliant, clinical-scale process in which T-cells derived from healthy donors are simultaneously base edited at four genomic loci then transduced with a lentivirus coding for an anti-CD7 CAR. The resulting cells are universally-compatible, allogeneic (“off the shelf”) CD7-targeting CAR-T cells resistant to both fratricide and immunosuppression.
Glycogen Storage Disease Type Ia
Beam’s approach to treating patients with GSDIa is to deliver an ABE via lipid nanoparticle (LNP) to the liver to repair either the R83C or the Q347X mutations in G6PC. It is estimated that these two-point mutations account for 900 and 500 patients, respectively, in the United States, representing approximately 60% of all GSDIa patients.
In vivo correction of both mutations by ABEs was observed in the livers of two strains of transgenic mice, each carrying one of the two G6PC mutations. Next-generation sequencing data from whole liver extracts reveal significant correction for both R83C and Q347X, with nearly 40% and approximately 70% A-to-G conversion efficiency, respectively, of each mutation back to the normal gene sequence. These significant levels of mutation correction greatly surpass those expected to restore glucose homeostasis, and functional studies are ongoing to correlate pathophysiology to extent of mutation correction by base-editing.
Sickle Cell Disease
Beam is pursuing two differentiated base editing approaches to treat hemoglobinopathies, BEAM-101 and BEAM-102
Beam Therapeutics -Third Quarter 2020 Financial Results
Cash Position:
Cash, cash equivalents, and marketable securities were $202.2 million as of September 30, 2020. This cash balance does not include the proceeds of the October 2020 offering.
Net Loss:
Net Loss:
Net loss attributable to common stockholders was $34.5 million, or $0.69 per share, for the quarter ended September 30, 2020, compared to $22.3 million, or $3.31 per share, for the quarter ended September 30, 2019.
Reference
www.beamtx.com
https://investors.beamtx.com/node/6826/pdf