10 Best Gene Therapy Stocks to Buy Right Now

4) CRISPR Therapeutics AG (NASDAQ:CRSP)

Number of Hedge Fund Holders: 27

CRISPR Therapeutics AG (NASDAQ:CRSP) is focused on utilizing the revolutionary CRISPR-Cas9 gene-editing technology to develop transformative therapies for genetic diseases.

CRISPR Therapeutics AG (NASDAQ:CRSP) received a significant boost with the approval of a CRISPR-based therapy, Casgevy, for the treatment of sickle cell disease (SCD) and transfusion-dependent beta-thalassemia in patients aged 12 years and older. CRISPR Therapeutics AG (NASDAQ:CRSP) partnered with Vertex Pharmaceuticals for the development and commercialization of Casgevy, with strong initial uptake of the therapy. Transfusion-dependent beta-thalassemia (TDT) is a severe form of beta-thalassemia, a hereditary blood disorder caused by mutations in the HBB gene, which encodes the beta-globin component of hemoglobin.

TDT and SCD have high unmet medical needs. On a global basis, millions of patients are impacted, with a significant subset eligible for curative gene-editing treatments. Many patients are dependent on regular blood transfusions and face complications, making a potentially one-time curative therapy like Casgevy highly attractive.

Wall Street analysts expect high per-patient costs for Casgevy, which should significantly boost revenue. Furthermore, as a result of a strategic alliance with Vertex Pharmaceuticals, CRISPR Therapeutics AG (NASDAQ:CRSP) can leverage Vertex’s established commercial infrastructure and expertise, ensuring efficient market entry and scalability.

CRISPR Therapeutics AG (NASDAQ:CRSP)’s pipeline extends beyond Casgevy, with numerous promising candidates in various stages of development. Its allogeneic CAR-T cell therapy programs, mainly CTX112 for B-cell malignancies, demonstrated encouraging results.