Synapses are crucial nodes for communication between neurons and are frequently damaged or dysfunctional in a range of neurodegenerative diseases. In their Review, Dejanovic, Sheng and Hanson outline the current understanding of pathological mechanisms operating at the synapse. They discuss several synapse-targeted approaches in preclinical and clinical development, which could be particularly valuable in combination with other therapeutic strategies.

Stuart Orkin, of Harvard Medical School and HHMI, discusses how the discovery of BCL11A paved the way for Vertex and CRISPR Therapeutics’ exa-cel therapy for haemoglobinopathies.

Ventus has overcome medicinal chemistry challenges to advance a first inhibitor of cGAS-STING into the clinic, as drug developers build the case for novel anti-inflammatory agents in autoimmune diseases including lupus.

This Review discusses current research strategies to overcome resistance to CAR-T cell therapy in blood cancers, including optimization of CAR design, improvement of T cell function and persistence, modulation of the immunosuppressive tumour microenvironment and synergistic combination strategies.

Stuart Orkin, of Harvard Medical School and HHMI, discusses how the discovery of BCL11A paved the way for Vertex and CRISPR Therapeutics’ exa-cel therapy for haemoglobinopathies.

Ventus has overcome medicinal chemistry challenges to advance a first inhibitor of cGAS-STING into the clinic, as drug developers build the case for novel anti-inflammatory agents in autoimmune diseases including lupus.