Vertex and partner CRISPR Therapeutics hope CDX001 can offer a single-use curative regimen for sickle cell disease and beta thalassemia patients. A regulatory filing is possible within 18-24 months.

Only nine weeks after US approval, Trikafta, which in theory can treat 90% of all patients with cystic fibrosis, is already Vertex's top-selling medicine.

Given the transformative nature of regenerative medicines, treatments yielding greatly improved patient responses that now exist as viable products on the market, cell and gene drug developers over the past several years have been drawing deal attention. Not only through collaborative partnerships, but also as acquisition targets.

Tender deadline for Roche acquisition of Spark pushed back to 30 October; 21.4% of Spark shares tendered so far. Vertex further diversifies with fourth deal of 2019 – an RNA-targeted collaboration with Ribometrix.