We asked three rare disease drug developers about their work, the challenges facing the field, and what they would do with unlimited funds.

A new study reports targeted CRISPR delivery in retinal and neurological disease models – without viral vectors.

The career path of Moncef Slaoui has resulted in billions of doses of lifesaving vaccines. Here he shares his story.

Bluebird bio’s financial situation is a stark reminder of the challenges of developing advanced therapies for rare diseases.

To shareholders – according to an analysis of financial reports from healthcare companies.

How connecting the data dots can lead to better development and clinical trals. 

How precision analytics is driving the future of pharmaceuticals, from small molecules to CRISPR gene editing.

A Lassa fever vaccine candidate shows promise in animal studies, with guinea pigs 100 percent protected from lethal virus exposure.

A regulatory science push in the UK includes centres for digital transformation, AI and more.