Many gene therapies companies have faced R&D and financial problems this year but the longer term potential of the field and low prices might tempt big pharma into deals.

As the first CRISPR-based therapies draw closer to approval, the Flagship-backed biotech believes its gene writing tech is the ‘definitive’ genetic medicine platform.

The first gene-edited therapy will be submitted for sickle cell disease and transfusion-dependent beta thalassemia; it will compete with bluebird in both indications, but analysts anticipate blockbuster sales in excess of $2bn.

Unsuccessful attempts to use real-world evidence for approvals highlight the need to demonstrate relationship between real-world data and clinical trial patients. The Pink Sheet examines what went wrong for Bluebird/Bristol’s Abecma, Ultragenyx’s Dojolvi, and Karyopharm’s Xpovio.