Category: Drug Development

Leveraging rapid developments in gene therapy, researchers are designing new ways to target ultra-rare genetic diseases with treatments tailored to a patient’s specific genetic makeup.

A groundbreaking gene editing therapy for sickle cell disease from CRISPR Therapeutics and Vertex Pharmaceuticals has received approval from the U.S. Food and Drug Administration.

Hemophilia is a rare genetic blood disorder characterized by the absence or malfunction of blood clotting factors, which can lead to severe and sometimes life-threatening

Blood cancers like leukemia and lymphoma are caused by mutations in the DNA of blood cells. These mutations cause normal blood cells to change into cancer

Alzheimer’s disease is a major global health concern: the devastating neurodegenerative disorder is responsible for 60-80 percent of all cases of dementia, and it affects

Parkinson’s disease is a progressive neurodegenerative disorder with no known cure. Current treatment options are limited, aiming to alleviate some symptoms through a combination of

The recent release of findings from a small Phase 1 clinical trial not only gives hope to families affected by an ultra-rare childhood disease, but

Cell and gene therapies (CGTs) are quickly making their way from theory to clinical practice, primarily because they target specific disease drivers rather than the