On November 21, shares rose CRISPR therapeutics (NASDAQ: CRSB) Prices fell by 47% from their peak in March. This may be a bit surprising to people who follow this developer of gene therapies. After all, it’s been less than a year since U.S. and European Union regulators approved its first drug, Casgevy, to treat two blood-related disorders.
Casgevy’s initial launch wasn’t as exciting as investors and its partner, Vertex Pharmaceuticals (NASDAQ:VRTX)he hoped. However, less than a year after launch, it’s still too early to turn our backs on this innovative drugmaker. In addition to Kasgeevi, there are five other treatment candidates in clinical phase tests.
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The Food and Drug Administration (FDA) approved Casgevy to treat sickle cell disease (SCD) last December. In January, the agency followed up by approving transfusion-dependent beta-thalassemia (TDT).
Across the Atlantic, European regulators approved Casgevy to treat both SCD and TDT in February. Despite regulatory approvals, the launch is proceeding slower than investors expected.
CRISPR Therapeutics has wisely partnered with Vertex Pharmaceuticals to develop and commercialize Casgevy, but Vertex is having difficulty getting it off the ground. Despite receiving approval in late 2023, Vertex did not record its first sale of Casgevy until the third quarter.
Sales have been slow because it is a complex treatment produced in single batches of a patient’s stem cells. Once reinjected, the CRISPR-edited stem cells should produce functional hemoglobin, so SCD and TDT patients no longer need regular blood transfusions. Unfortunately, reimplanted Kasgeevi cells can only gain a foothold if patients first deplete their immune systems with a serious conditioning regimen.
Recently, a patient with SCD died during his gene therapy trial Beam treatments. The doctors conducting the study did not blame the BIM candidate for the volunteer’s death; They blamed an air conditioning system containing busulfan. Busulfan is also used for the condition of patients with Casgevy.
The lack of treatment options may be to Kasjevi’s advantage. Last year, the European Medicines Agency revoked the conditional approval of a drug to treat sickle cell disease Novartis It was called Adakveo, after it failed to outperform placebo in a confirmatory trial. In September, Pfizer Oxbryta, a daily pill approved to treat patients with SCD, has been withdrawn from the market after it failed a post-marketing study.
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