GSK’s Arexvy and Pfizer’s Abrysvo provide older adults with a much-needed vaccine for respiratory syncytial virus (RSV) — and raise hopes for an option for infants too.

Many diseases involve reduced or absent levels of a particular protein and would benefit from therapies that increase gene expression. In their Review, Khorkova et al. discuss the growing range of RNA-targeted therapies in development that aim to boost gene expression, including nucleic acid-based therapeutics targeting the complex regulatory network of non-coding RNA species.

The FDA approved tofersen for amyotrophic lateral sclerosis based on the drug’s ability to lower blood levels of neurofilament light (NfL) — establishing a proof of potential for this neuroscience biomarker that could have implications for other diseases of the brain.

Joni Rutter, director of the National Center for Advancing Translational Sciences (NCATS), discusses the centre’s audacious goal to have 25% of diseases covered by the experimental drug pipeline by 2032.

GSK’s Arexvy and Pfizer’s Abrysvo provide older adults with a much-needed vaccine for respiratory syncytial virus (RSV) — and raise hopes for an option for infants too.

The FDA approved tofersen for amyotrophic lateral sclerosis based on the drug’s ability to lower blood levels of neurofilament light (NfL) — establishing a proof of potential for this neuroscience biomarker that could have implications for other diseases of the brain.

Joni Rutter, director of the National Center for Advancing Translational Sciences (NCATS), discusses the centre’s audacious goal to have 25% of diseases covered by the experimental drug pipeline by 2032.