Latest from Andrew McConaghie
Encouraging results in younger children with Duchenne is keeping Regenxbio on track for a potential mid-2026 filing for its gene therapy, RGX-202.
The 75-year-old veteran of rare disease drug development is handing over the reins to a successor, but will stay to help Stoke Therapeutics expand its pipeline.
The potential first-in-class antibody oligonucleotide conjugate could treat a form of the muscle wasting disease, and could be the first of Avidity’s trio of rare disease drugs to gain approval.
The acquisition is worth up to $1bn and adds to AstraZeneca’s broad array of cell therapy technologies.
In the crowded and competitive field of generalized myasthenia gravis, Amgen has faith that Uplizna’s twice-yearly administration will make it the treatment of choice.
Evaluate Pharma’s top 10 list of unpartnered mid-stage obesity assets shows that few credible challengers to Novo’s Wegovy and Lilly’s Zepbound remain available.