Beam Therapeutics (BEAM) Reveals Encouraging Results From Risto-Cel Phase 1/2 BEACON Study

Beam Therapeutics Inc. (NASDAQ:BEAM) is one of the 10 best biotech stocks with highest upside potential.

On April 2, Beam Therapeutics Inc. (NASDAQ:BEAM) released results from its Phase 1/2 BEACON study for ristoglogene autogetemcel. This therapy is being developed to treat severe vaso-occlusive crises associated with sickle cell disease, through the New England Journal of Medicine. Risto-cel is a potentially best-in-class autologous cell therapy. Results from the study indicated that the therapy was well-tolerated with safety, consistent with myeloablative conditioning, and showed highly promising efficacy results.

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The study led to better parameters for hemolysis, anemia correction, and the absence of any complications with respect to vaso-occlusion after transplant. Rapid and complete bone marrow reconstitution was obtained in patients. The average values of hemoglobin F exceeded 60%, while those of hemoglobin S were below 40% in most patients.

All hemolysis-related measures were either normal or showed improvement after risto-cel therapy. All sickling measures declined after risto-cel therapy and were like those observed in people suffering from sickle cell trait. There were no major adverse events associated with risto-cel that were not related to busulfan conditioning, autologous hematopoietic stem cell transplantation, and SCD.

Beam Therapeutics Inc. (NASDAQ:BEAM) is a biotechnology company that develops precision genetic medicines and also engages in gene therapies and genome editing research. Since its launch, the company has been spearheading the CRISPR-based editing that facilitates the development of advanced genetic medicines.

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