Genetic diseases

Metrion Biosciences, a specialist ion channel contract research and drug discovery company, and The KCNC1 Foundation, a not-for-profit organisation focused on the development of a treatment for an ultra-rare genetic disorder, have entered a collaboration to progress a hit identification research project for small molecule modulators of the potassium ion channel Kv3.1, targeting KCNC1-related disorders […]

Scientists have unlocked key insights into virus evolution, revealing new information that could help develop treatments for a wide variety of genetic diseases.  The research focuses on a group of small, ubiquitous viruses called ‘parvoviruses’ (from the Latin word ‘parvus’ meaning ‘small’, ‘puny’ or ‘unimportant’). It was led by scientists at the MRC-University of Glasgow […]

Michael Henderson, MD, Chief Business Officer of BridgeBio Pharma, tells Lu Rahman about the company’s pursuit of transformative medicines to treat genetic diseases. BridgeBio’s mission is to discover, create, test and deliver transformative medicines to treat patients who suffer from genetic diseases and cancers with clear genetic drivers as quickly and safely as possible. “To do […]