In tandem with buying the rest of Kleo, Tonix licenses molecular degradation technology from Yale. Neurogene, University of Dundee partner on cell therapies for neurological disorders.

With $1.1bn in cash plus revenue from its existing commercial products and royalties from Roche’s sales of newly approved Evrysdi to invest in R&D, PTC Therapeutics may achieve profitability in 2023.

Jeffrey Aronin, who presided over Marathon Pharmaceuticals during a drug-pricing kerfuffle that drew congressional scrutiny, has built a portfolio of new drug companies at Paragon Biosciences.

Patient-driven drug development meets the old saying of "where there's a will, there's a way" in China, potentially the world's largest market for rare disease treatments.