The privately held German pharma firm is hoping to bring a new generation of pulmonary fibrosis and cancer drugs to market but their commercial success is not yet a certainty.

Regenxbio and Solid Biosciences believe they have a next-generation approach to AAV-based gene therapies – but must distance themselves from Sarepta’s safety and efficacy problems.

A new small-molecule approach to turning on beneficial genes to counteract disease-causing mutations is being pioneered by Insmed’s UK-based scientists.

Anselamimab was expected to complement Alexion's portfolio of amyloid therapies, but regulators will have to be convinced of its benefits to a patient subgroup in the CARES trial.

Big pharma is increasingly looking east to restock its pipelines, a process that is helping to forge a new generation of mid-sized R&D-based companies in China and is reflected in their stock price performance.

Rivals Mineralys could be first to file but AstraZeneca is banking on its cardiovascular market know-how to lead the emerging class.