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.

The COVID-19 pandemic spurred a wave of rapid and collaborative drug discovery efforts. This Perspective article summarizes scientific drivers and considerations behind such antiviral small-molecule discovery programmes and proposes strategies to accelerate future efforts.

There have been significant recent advances in the development of single-cell technologies, providing remarkable opportunities for drug discovery and development. Here, Ferran and colleagues discuss how single-cell technologies, primarily single-cell RNA sequencing methods, are being applied in the drug discovery pipeline, from target identification to clinical decision-making. Ongoing challenges and potential future directions are discussed.

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.