Phase I/II data for its ADC candidates in myotonic dystrophy type 1 and Duchenne muscular dystrophy indicate potential for biomarker-based accelerated approval in both diseases, Dyne asserts.

Public Company Edition: Denali, Crinetics and Ocular Therapeutix grossed $500m, $350m and $325m, respectively, in private placements ahead of data readouts. Also, Viking raised $550m after its positive Phase II obesity data and Abingworth invested $210m in Gilead’s Trodelvy NSCLC trials.

More than 75 industry executives and experts gazed into the crystal ball and shared with Scrip their predictions around transformative technologies. Perhaps unsurprisingly, AI was the dominant theme – but gene editing, cell therapies and new targeting modalities were also among the highlights.

2023 saw a number of gene therapy companies go to the wall, but those that can combine clinical significance and commercial success can build sustainable businesses.