The congress’s late breakers highlight progress across cell and gene platforms and diseases, and Duchenne is in the spotlight as Sarepta’s SRP-9001 could soon gain accelerated approval.

A long-anticipated pruning back of the ev vivo gene therapy pipeline for sickle cell disease has arrived with Novartis, Graphite Bio and Sangamo each dropping projects, but some challengers are looking ahead to a future of in vivo approaches to the condition.

With the planned $487m acquisition of sensorineural-focused Akouos, Lilly makes its second gene therapy M&A play in two years, on top of partnering/financing activity in the space.

SpringWorks is on course to bring nirogacestat to market by next year – and could play a pivotal role in making BCMA-targeting drugs a success.