Gene therapy represents one of medicine’s most ambitious attempts to treat disease at its root cause by altering the genetic code itself. The approach works by recovering the functions of critical ...

Scientists at St. Jude Children's Research Hospital and Northwestern University identified a previously unknown treatment opportunity for sickle cell disease and β-thalassemia. The discovery, ...

By Yiqing Wang/WHRO State lawmakers have budgeted nearly $15 million over two years to help Virginia Medicaid cover gene ...

"This was the first proof that 'gene therapy in a box' could work." Gene therapies or cell therapies that involve genetically modified cells today are available at only a limited number of research ...

Epilepsy affects more than 50 million people worldwide, making it one of the most common neurological disorders. Although ...

The search for gene therapies to treat rheumatoid arthritis (RA) has moved in fits and starts over the past two decades, with trials starting and stopping, and their focus shifting to osteoarthritis ...

Preliminary results from two trials of the gene therapy exagamglogene autotemcel (exa-cel) suggest the therapy offers an effective cure for beta-thalassemia and sickle cell disease in children younger ...

Up to three in every 1,000 newborns is born with hearing loss in one or both ears. While cochlear implants have long been a life-changing option, they involve surgery and can't fully replicate the ...

The U.S. government has awarded Children’s Hospital of Philadelphia $39 million to develop a scalable platform for treating rare genetic, liver-related diseases in infants and children. The federal ...

Dr. Donald Kohn has been developing gene therapies for rare pediatric immune disorders for over 30 years. This week, his role in a clinical trial has culminated in the first-ever U.S. Food and Drug ...

From data lakes to streamlined production, Kriya, Opus, and Epicrispr discuss how technology is improving their operations.