The CEOs of bluebird bio and CRISPR Therapeutics hope to inspire investor confidence that their gene-based therapies for sickle cell disease and beta thalassemia will be widely reimbursed in the US and EU.

The company has pledged to continue developing its Duchenne in vivo gene editing therapy without Lilly, but will remain focused on its lead program in hepatitis B.

In contrast to the SEC’s view that public companies’ regulatory filings give investors all the information needed to make an investment decision, the discount rate used to value a company may not reflect all its risks.

The biotech company’s CEO talked to Scrip about plans to bring the CD20-targeting CAR-T MB-106 into an investigator-sponsored Phase I trial later this year.