The Food and Drug Administration this week granted approval to a new gene therapy for a rare immune disorder, the maker of which is notably not a drug ...

Based on initial positive results from HOPE-B, etranacogene dezaparvovec became the first gene therapy approved for adults ...

A breakthrough therapy is driving aggressive leukaemia into remission. Here’s what the headlines don’t tell you about life after treatment.

Friedreich’s ataxia is a genetic disorder that causes progressive damage to the nervous system and brain, leading to a loss ...

The FDA has issued its stamp of approval to a new, cell-based option to treat Wiskott-Aldrich syndrome (WAS), marking the ...

The three-hit model, developed from more than a decade of systems biology research, proposes that autism develops when three ...

FDA approves 1st gene therapy for Wiskott-Aldrich syndrome, offering a new treatment for patients lacking matched stem cell donors.

Novartis AG (NYSE:NVS) is one of the best pharma stocks to invest in. On December 8, Novartis AG (NYSE:NVS) was upgraded to ...

Backed by Italy-based Fondazione Telethon ETS, Waskyra, for Wiskott-Aldrich syndrome, is the first gene therapy from a ...

EpilepsyGTx, a biotechnology company focused on research and development of cutting-edge gene therapies to treat refractory epilepsy, today announced it has raised $33 million in a Series A financing ...

Breast cancer comes with many variables. A pair of powerful genomic tests can help women and their care teams make personalized care decisions.

A pioneering genetic treatment is saving lives where chemotherapy failed, offering new hope for children and adults with aggressive leukemia. This world-first therapy has kept patients disease-free ...