Gene therapy is a medical treatment that works by changing or fixing a person's genes to treat or prevent disease ...

While the existing gene therapy was approved for use in Europe in 2024, it is very expensive, which limits its accessibility. Moreover, this treatment modifies a globin repressor gene, which indeed ...

NEW YORK – Boston Children's Hospital has granted a worldwide license for an investigational gene therapy for sickle cell disease and beta thalassemia to Caring Cross, the nonprofit announced this ...

The nonprofit biotech recently licensed the gene therapy from Boston Children's Hospital and plans to bring it to low- and middle-income countries.

Key HighlightsMotixafortide effectively mobilized sufficient hematopoietic stem cells (HSCs) in patients with sickle cell disease and beta-thalassemia enabling accelerated access to gene therapies73% ...

The U.S. Patent and Trademark Office has accepted a groundbreaking patent from Dei BioPharma Ltd., marking a significant milestone in the treatment of sickle cell disease.Dei BioPharma, a Ugandan ...

Gene therapy for sickle cell disease may help improve a major contributing factor to stroke risk in patients, reports a new study from St. Jude Children's Research Hospital. Many people with sickle ...

Few companies stand better poised than Beam Therapeutics to reap the first fruits of the FDA’s promised flexibility toward cell and gene therapies. | Riding a regulatory win for its base editing ...

Researchers have found a promising new method for gene therapy. They successfully restarted inactive genes by bringing them closer to genetic switches on the DNA called enhancers. The intermediate ...