Most inherited retinal diseases still have no approved therapy despite advances in gene therapy. This article explores why researchers are targeting shared disease mechanisms alongside individual mutations.
Antibody-drug conjugates (ADCs) present unique toxicology challenges. Learn which endpoints matter most, how they inform development decisions and why they are essential for building a robust nonclinical safety strategy.
The blood–brain barrier protects the brain from harmful substances, but it also prevents many medicines from reaching their target. Researchers are investigating whether focused ultrasound could safely improve drug delivery.
Understanding where proteins are expressed throughout the body is critical for selecting better drug targets. Here’s how a new human proteome atlas could improve target selection, predict toxicity and support drug repurposing.
A new gene therapy platform exploiting the brain’s glymphatic transport system and engineered AAV5 vectors could offer a targeted treatment route for multiple sclerosis, Huntington’s disease and rare white matter disorders.
A three-drug combination targeting shared metabolic vulnerabilities in cancer and senescent cells has improved physical performance and extended survival in aged mice, whilst avoiding the thrombocytopenia associated with existing navitoclax-based regimens.
A newly identified mitochondrial microprotein, MENTSH, has been linked to increased Type 2 diabetes susceptibility in Mexican and Mexican American populations, pointing towards a potential precision medicine target.
Scientists at the Fralin Biomedical Research Institute have identified bone morphogenetic protein 3 as a protective factor in lung blood vessels, with preclinical data showing that restoring its levels can reverse hallmark features of pulmonary arterial hypertension.
One receptor can protect antibodies from degradation, extend their half-life and become a drug target itself. Explore the science behind FcRn and how researchers measure its function.
Static cultures can miss critical immune–tumour interactions. Learn how the Mera™ flow-based human tissue model better captures T-cell activity to strengthen preclinical immunotherapy research.
Non-animal methods are already used throughout early drug discovery, yet animal testing continues to dominate regulatory safety assessment. Recent initiatives suggest change is coming, but significant scientific and practical challenges remain.