When the German major bought AskBio a couple of years ago, one of its assets was a gene therapy for Huntington’s, a devastating disease that leads to complete physical and mental deterioration. That asset, BV-101, is about to enter the clinic.

Public Company Edition: The stock market has been unkind to biotech firms but investors rewarded some drug developers for clinical trial successes, including Scholar Rock and Day One, which raised $205m and $172.5m, respectively. However, some SPAC deals and an IPO never got off the ground.

Many industry leaders expect broad progress in the field of RNA technologies following the rapid and successful roll-out of mRNA vaccines against COVID-19 last year. Genetic medicines of various types, emerging platforms for tackling previously “undruggable” targets and the microbiome were among other areas singled out as areas to watch in 2022.

Despite disappointment in its Phase III study, Roche is preparing a new trial of the antisense therapy in patients with a lower disease burden.