CRISPR Therapeutics announced that CASGEVY, its gene-editing therapy, received regulatory approvals for patients 12 years of age and older with severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT) in Switzerland and Canada. These approvals expand the global availability of the first CRISPR-based therapy, following earlier approvals in other jurisdictions. The company's collaboration with Vertex Pharmaceuticals involves a 60/40 split of program costs and profits, with Vertex leading commercialization.
Commercialization efforts for CASGEVY are progressing, with 45 authorized treatment centers (ATCs) activated globally as of mid-October 2024. Approximately 40 patients have had their cells collected across all regions, indicating initial patient uptake for the therapy. This operational milestone is crucial for establishing the infrastructure required for broad patient access and future revenue growth.
The company maintains a strong financial position, reporting approximately $1.9 billion in cash, cash equivalents, and marketable securities as of September 30, 2024. This capital provides a substantial runway for ongoing research and development across its diverse pipeline, including CAR T, in vivo gene editing, and Type 1 Diabetes programs. Updates on the Phase 1 dose escalation study of CTX112 in B-cell malignancies are anticipated at the American Society of Hematology (ASH) 2024 Annual Meeting.
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