10 Best Biotech Stocks with Highest Upside Potential

9. Beam Therapeutics Inc. (NASDAQ:BEAM)

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.

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.