Henry Ford Health has treated the first Michigan patient outside clinical trials with Roctavian, the newly approved gene ...

The patient, who is in his 30s, got the one-time intravenous infusion at the Infusion and Chemotherapy Treatment Center at ...

Opportunities in the Sickle Cell Disease market include advancing gene-editing therapies like EDIT-301, expanding VOC-reducing treatments such as inclacumab, and exploring novel drug candidates like ...

Sickle cell anemia (HbSS) is the homozygous dominant variant and the most common and severe form of the disease. Whereas patients who only inherited one gene encoding for abnormal HbS and a different ...

Investors have bought into the promise of CRISPR technology for years. The ability to target and edit genes to treat cancer ...

UAE patient with heart failure, thalassemia treated with gene therapy after transplant ...

Despite groundbreaking research, many cell and gene therapies do not make it all the way to the patients. Researchers and clinicians in Lund have now presented a new model for cooperation that will ...

Vertex Pharmaceuticals Incorporated faces slowing Trikafta/Kaftrio sales and a stock-price divergence with peers. Click for ...

KRAS is the most frequently mutated oncogene across all human cancers. Although different KRAS mutations have long been thought to exert the same cancer-driving effects, a new study led by UT ...

Scientists in China have unveiled a breakthrough way to mass-produce powerful cancer-fighting immune cells in the lab. By engineering early-stage stem cells from cord blood—rather than trying to ...