Two public companies focused on RNA editing therapies, Korro Bio and ProQR, enjoyed impressive stock surges powered by their best-ever one day gains; their share prices lifted by Wave Life Sciences ...

Modifying a protein that controls cell growth can reactivate dormant neural stem cells in fruit flies which could help ...

Researchers suggest research findings might be applicable to other age-related diseases where endothelial dysfunction plays a ...

First patient dosed with ISP-001 shows reduced urinary GAG level, functional improvements surpassing enzyme replacement ...

The disruption of abnormal activity in the gut microbiome may increase our risk for neurological disease, research suggests.

Chief, John Sterling, reviews Tim Harris's latest book, In Pursuit of Unicorns: A Journey through 50 Years of Biotechnology.

Wave Life Sciences' alpha-1 antitrypsin deficiency candidate WVE-006 succeeded in the first-ever clinical demonstration of ...

Researchers used gene therapy to convert harmful fatty acids into beneficial ones to help combat childhood obesity.

The awards celebrate the discovery of a new role for CD4 T cells in the cancer immune response and insights into autoimmune ...

Data from a new study suggests that in the early phase the damage to the brain starts slowly, before speeding up in the ...

The company reflects on their latest IND approval, the evolution of their end-to-end drug discovery platform, and what’s next ...

Results suggest novel roles for LINE1 transposable elements that can be explored in the contexts of diseases, from ...