The FDA approved a gene editing treatment for sickle cell disease to alleviate severe, recurrent pain crises. Casgevy, developed by Vertex Pharmaceuticals (VRTX) and CRISPR Therapeutics (CRSP), is a one-time treatment that harnesses gene editing advancements. However, the therapy faces accessibility hurdles, according to Wedbush Managing Director David Nierengarten. Despite providing a lifelong cure, he believes the complex procedure and recovery alongside the expensive price tag will be challenging to market to patients.
Nierengarten believes uptake will be limited to most acute cases given process difficulties. The length of time for treatment preparation, hospitalization, and healing poses barriers. Low enrollment in clinical trials is “not surprising” per Nierengarten, as “the real barrier here is the procedure itself.”
Accessing facilities able to perform the bone marrow transplant involved also raises logistics issues. He notes trial sites were in areas with small sickle cell populations. “The time it takes to do it and the difficulties involved with it” are key obstacles, Nierengarten told Yahoo Finance. Though transformative for patients, real-world availability of the therapy faces multiple constraints.
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