Volume 24 Issue 12, December 2025

Industry is still trawling the kinome for drug targets, 24 years on from the landmark approval of imatinib.

CRISPR-based technologies provide a diverse set of tools to correct pathogenic mutations. This Review describes the promise and the challenges of genome editing therapeutic strategies involving nucleases, base editors and prime editors for patients with inherited haematological disorders.

Systemic lupus erythematosus (SLE) is a complex autoimmune disease with diverse clinical manifestations. This Review discusses advances in understanding its immunopathogenesis, the evolution of targeted therapeutic strategies, and emerging approaches to restore immune tolerance. Challenges and opportunities in achieving durable remission or cure in SLE are also explored.

Multispecific drugs are designed to engage two or more entities to exert their pharmacological effect. This Perspective discusses how a new wave of FDA-approved multispecific molecules have been transformative in overcoming barriers to drug development such as toxicity, rapid clearance, undruggable protein features, and functional redundancy.