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.

More than 100 biopharma executives and experts told Scrip their predictions for therapeutic area advances in the coming year. The recent commercial success of GLP-1s in diabetes and obesity and their potential in further disease areas fuelled excitement around the metabolic space. Expectations were also positive in neurology following the launch of Leqembi for Alzheimer’s disease in 2023, while the multiple opportunities to improve cancer treatment kept oncology top of the pile overall.

The company claims its gene editing tech is the most potent yet – and is joining others in making established cardiovascular disease gene PCSK9 its first therapeutic target

Biotech entrepreneurs tend to be an optimistic group, and there were many pockets of hope for 2024. However, the consensus remains that depressed valuations and paucity of fresh funds will continue to pose a challenge for the sector, and companies will need to prioritize carefully and work even harder to demonstrate and create value.