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.

Agency recommendation for randomized controlled trials in ultra-rare pediatric disease is unworkable for various reasons, say advocates and sponsors who seek use of the accelerated approval pathway based on changes in heparan sulfate levels.

Avrobio is facing up to the reality of investor skepticism about gene therapy platforms and business models – but can it find a buyer or merger partner to start again?

Public Company Edition: Also, Johnson & Johnson’s consumer health spin-out Kenvue filed paperwork to get the ball rolling on an initial public offering. In addition, Geron raised $198m and Madrigal accessed more than $300m after positive trial readouts, but layoffs were revealed by Editas, Elevation, Nabriva and others.