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Open science initiatives that harness human genomic and genetic data could help increase the likelihood of successfully developing new drugs for neurodegenerative diseases.
Significant progress has been made in understanding the influence of membrane transporters in drug disposition and response. Here, the International Transporter Consortium provides an update on the current status of membrane transporters in drug development and regulatory requirements, discusses recent scientific advances in the field and highlights future directions and unanswered questions.
The field of nucleic acid therapeutics is rapidly expanding, with mRNA drug and vaccine candidates now under development for diverse indications. However, various safety concerns are associated with the mRNA platform. This Review discusses key challenges and toxicities identified during the development of mRNA drugs and vaccines, assesses models and tools used to limit toxicity, highlighting knowledge gaps and future directions.
Clinical trial costs seem to be inflating excessively, without a clear increase in the value of the information gained. Here, we highlight factors that could be driving this trend and discuss potential solutions.
This Review discusses the mechanisms that regulate stabilization of hypoxia-inducible factors (HIFs), and the pharmacological strategies to activate or inhibit HIFs in diseases such as ischaemia, inflammation, cancer, retinal neovascularization and pulmonary hypertension.
Several challenges hamper the translation of preclinical drug discovery efforts into safe and effective therapies for psychiatric disorders. In their Perspective, Khodosevich and colleagues highlight differences between drug targets in animal models and those in patients as a key reason for failed clinical studies. They present a framework that integrates single-cell and spatial omics data to overcome this loss in translation, which they hope will assist the development of new drugs for diseases such as schizophrenia and major depressive disorder.