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.

The Japan-based drug maker is hoping to expand into cell and gene therapy as well as enlarge its presence in the North American market, while Orchard awaits an FDA verdict on Libmeldy.

Securing a place on the European Medicines Agency’s priority medicines scheme is not easy, but some companies have managed to make the grade more than once.

Robust natural history data sets with well-chosen outcomes are fundamental in Aetion’s regulatory feasibility tool for real-world evidence, built through analysis of US FDA case studies and guidance.