A Phase 2a study that was evaluating a potential treatment for paediatric patients with non-alcoholic fatty liver disease was stopped early in August after the first three patients showed an increase in liver fat and elevations in serum alanine transaminase (ALT) levels, raising concerns about liver toxicity.

Orchard Therapeutics Ltd has raised $150 million in a Series C financing round to advance its portfolio of ex vivo gene therapies, the most advanced of which is in registration trials for the treatment of an ultra-rare disease that damages the immune system.