The two boys both have Duchenne Muscular Dystrophy, a genetic disorder that typically has children wheelchair bound by the ...

Ryuji Morizane, MD, Ph.D., of the Department of Medicine at Massachusetts General Hospital, is the senior/corresponding ...

Gene Therapy Trial Can Restart After Patient Death, FDA Says. The Sector Remains Depressed. The biotech Rocket ...

Treatment with exagamglogene autotemcel (exa-cel) led to robust and sustained improvements in quality of life for patients ...

Gene therapy biotech Kriya Therapeutics has secured $313 million in a new series fundraise. | Gene therapy biotech Kriya ...

Patients with severe sickle cell disease experienced significant quality of life improvements after receiving exagamglogene autotemcel gene therapy.

An expert discusses how gene therapy represents a breakthrough treatment approach for BCG-unresponsive patients, offering ...

Biomedical engineers at Duke University have successfully conducted experiments to treat damage caused by heart attacks in nonhuman primates using gene therapy for the first time.

The next big strides in gene therapy for rare diseases may come from CRISPR and new approaches to delivery.

Shares of drugmaker Sarepta Therapeutics surged in afterhours trading Monday after the company said it would resume shipping ...

Modern medicine constantly searches for safer and more efficient ways to treat genetic disorders. One cutting-edge solution that has emerged is the use of non-viral vectors.

The FDA approves the first of two gene therapies this month to treat sickle cell disease. Though expensive and difficult, it promises a better life.