Fierce Pharma February 8, 2023
As Vertex Therapeutics nears completion of a historic FDA submission, the rare disease specialist has depicted a rosy launch picture for what could become the first CRISPR-based gene editing therapy.
Vertex believes a network of about 50 authorized treatments centers in the U.S., and 25 in Europe, should suffice for its sickle cell disease and beta thalassemia gene therapy candidate exagamglogene autotemcel, or exa-cel, Chief Operating Officer Stuart Arbuckle told investors during a conference call Tuesday.
About 32,000 patients in the U.S. and Europe with severe disease will form the target population for the CRISPR Therapeutics-partnered exa-cel, and they are geographically concentrated, Arbuckle said. These patients require multiple hospitalizations annually for vaso-occlusive crises or are dependent on...
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