CRISPR Therapeutics
NEWS
The first gene therapies approved to treat sickle cell disease in December 2023 are struggling on the market. But there are glimpses of forward momentum as Vertex and Genetix Bio provide updates.
Following over a year of slow uptake, Vertex Pharmaceuticals and CRISPR Therapeutics expect Casgevy revenues to nearly triple in 2026, as patient access to the sickle cell disease and beta thalassemia gene therapy grows.
Data presented at this year’s American Heart Association Scientific Sessions in New Orleans underline rapid advancements in the cardiovascular field.
The partnership with Sirius expands CRISPR Therapeutics’ modality toolkit, especially in the cardiovascular space.
CRISPR Therapeutics’ partner Vertex reported that more than 65 treatment centers have been activated for the gene therapy Casgevy. While Vertex handles the market, CRISPR has been focused on its clinical program.
Having established success in cancer, biopharma is now looking to leverage CAR T therapies against a new target, autoimmune disorders, with several early- to mid-stage readouts expected this year.
While ex vivo genome editing results in highly effective cell therapies, it can lead to off-target effects. Caribou Biosciences has come up with a novel approach for potentially more precise gene editing compared to all-RNA guides.
Infusions of Vertex and CRISPR Therapeutics’ Casgevy and bluebird bio’s Lyfgenia have begun; Moderna targets 10 approvals through 2027; more oral obesity drug data; the latest from ESMO and more.
Last month, Vertex said sickle cell patients had not yet received infusions of its gene therapy Casgevy. That’s now changed, as the company races with bluebird bio’s Lyfgenia.
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