Only a few patients have undergone an intensive gene therapy cure for sickle cell disease since it was approved by the FDA ...

Gene therapies are revolutionizing treatment for hemophilia A and sickle cell disease; however, they are saddled with hefty ...

A phase 3 trial highlights betibeglogene autotemcel as a potentially curative gene therapy for severe transfusion-dependent β ...

The Congressional Budget Office said policies to increase treatment uptake could affect Medicaid, Social Security, and other government programs.

Gene therapy is referred to as a medical technique that involves modifying or manipulating genes to treat or prevent disease.

In individuals undergoing repeated blood exchanges or transfusions, however, the amount of iron introduced into their bodies ...

GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data. Attributes of the drug, ...

From a pig kidney transplant to restoring genetic deafness, 2024 was a year full of medical breakthroughs. The breakthroughs ...

Chad Stevens traveled from Idaho to Omaha's Nebraska Medical Center, where he became the hospital's first patient to receive ...

Anumigilimab is under clinical development by CSL and currently in Phase I for Sickle Cell Disease. According to GlobalData, Phase I drugs for Sickle Cell Disease have an 80% phase transition success ...

The first medical treatment to use Crispr gene editing has been on the market for a year. Its complexity means few patients ...

Catch up on all the latest pharma industry news from the past month, from breakthroughs in gene therapy to promising drug ...