A one-time gene therapy candidate for Gaucher disease, an allogeneic CAR-T cell therapy for difficult-to-treat blood cancers, and a potentially first-in-class drug for treating hyperphenylalaninemia have made it onto the European Medicines Agency’s priority medicines scheme.

Syncona has stepped in to save Freeline and give it more time to prove the value of FLT201, its early-stage Gaucher’s disease gene therapy.

Public Company Edition: PTC sold more of its Evrysdi royalties to Royalty Pharma for $1bn. Abivax gained $235.8m in an IPO and concurrent private placement, while Cerevel grossed $450m and Nuvalent and Ultragenyx raised $300m each in follow-on offerings, but Freeline is reviewing strategic options.

Avrobio is facing up to the reality of investor skepticism about gene therapy platforms and business models – but can it find a buyer or merger partner to start again?