The third iteration of Vertex and Crispr’s collaboration over CTX001, a gene-editing therapy for rare blood clotting disorders, shows how hopes for the project have ballooned. Vertex is paying $900m up front, plus a potential $200m milestone on first approval, to up its share of profits and costs from 50% to 60%. If Vertex is valuing 10% of profits at $1.1bn, an admittedly simplistic calculation suggests that it thinks CTX001 is now worth $11bn, a punchy sum that will raise expectations ahead of readouts later this year from sickle cell and beta-thalassaemia studies. Such blockbuster forecasts seem particularly optimistic on the day that Bluebird admitted defeat with its beta-thalassaemia gene therapy Zynteglo in Germany, where payers have baulked at the price; Vertex is apparently convinced that CTX001 is a more persuasive offering. The big US biotech is certainly cash rich, although some investors will probably be asking whether that billion dollars might be better deployed in a spreading of bets, rather than doubling down on an asset that still has much to prove. A further is question is what the move says about the chances of success for VX-864, on which data are also due.
|The evolution of a deal|
|Oct 2015||Gene editing collaboration established; Vertex gains rights to license up to six projects||Vertex pays $105m up front (incl $30m equity purchase); agrees to pay milestones of up to $420m on any project licensed in; costs and sales to be shared equally on any project taken forward in haemoglobinopathies, with Crispr leading US commercialisation|
|Dec 2017||CTX001 selected as first gene-edited therapy to be developed||Any new terms undisclosed|
|Apr 2021||Deal amended to give Vertex global lead on development, manufacturing and commercialisation, and a 60% share of costs and profits||Vertex pays $900m plus potential $200m milestone on first regulatory approval for additional 10% profit share|
|Source: company press releases.|