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CRISPR technology is revolutionizing the development of therapies for genetic disorders. However, the application of CRISPR-based therapeutics is challenged by factors impacting stability, efficiency, delivery and safety. This Review focuses on chemical engineering of CRISPR–Cas systems to address these issues, it assesses next-generation CRISPR–Cas systems, and it highlights CRISPR-based therapies that are approved or in clinical development.
The Open Targets consortium was founded in 2014 to translate insights from genetics and functional genomics into identifying and prioritizing therapeutic targets. We highlight key achievements and insights stemming from this partnership of academic and industry scientists.
Regulatory T cells keep the immune system in check to maintain homeostasis and restrain inflammation. This Review discusses strategies to harness these cells therapeutically for autoimmunity, transplant tolerance or cancer, for example, by boosting their endogenous function, depleting them or administering them as engineered cell-based therapies.
New approaches are needed to streamline clinical trials of drugs for patients with rare diseases. Digital biomarkers offer one such approach, but several challenges must be addressed to realize their potential.
CD8 T cells and natural killer (NK) cells play a key role in cancer immunosurveillance. However, their anticancer activity is rapidly diminished in the tumour microenvironment, associated with a decline in their metabolic function. This Review provides an overview of metabolic programmes in CD8 T cells and NK cells, discusses the dysfunction that can occur in cancer, and assesses therapeutic strategies to overcome this.
Jason Chin, head of Cambridge’s Centre for Chemical & Synthetic Biology and CSO of Constructive Bio, discusses the role for unnatural amino acids in biologic therapies.
Cancer-preventive vaccines can reduce cancer occurrence by inducing a specific immune response against tumours before they can fully develop. This Review discusses results from pioneering clinical studies and potential approaches to design potent prophylactic cancer vaccines in the future.
Driven by chronic overnutrition and associated with obesity, metabolic dysfunction-associated steatotic liver disease is increasing in prevalence. This Review discusses two main therapeutic approaches: improving the metabolism with incretin mimetics such as semaglutide and directly targeting the liver with agents such as the recently approved resmetirom.
