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Latest From bluebird bio
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.
For an industry increasingly dominated by these drugs, commercial programs need to take on the risk once reserved for development.
- Drug Delivery
- Gene Therapy, Cell Therapy
- Large Molecule
- Other Names / Subsidiaries
- Genetix Pharmaceuticals
- Precision Genome Engineering, Inc. (Pregenen)
- Oncology Newco