Volume 22 Issue 6, June 2023

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.

Since the COVID-19 pandemic began, many potential therapeutics that target SARS-CoV-2 and/or human proteins to control viral infection have been investigated, with a few receiving authorization by regulatory agencies. This Review article summarizes progress with COVID-19 drug discovery, and discusses the lessons learned about aspects such as drug repurposing, disease models and clinical development strategies.

Drugs that target angiogenic factors such as vascular endothelial growth factor (VEGF) are approved for clinical use in oncology and ophthalmology, but challenges remain. Cao et al. discuss strategies to enhance therapeutic efficacy, overcome drug resistance, define biomarkers and develop next-generation agents for other diseases.

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.