Research explores the advancements, challenges, and economic implications of gene therapy for various diseases, including inherited blood disorders, malignancies treated with CAR-T cells, and diseases targeted by in vivo AAV vectors. The review highlights the transformative potential of gene therapies while acknowledging the complexities in manufacturing, safety, efficacy, and long-term durability.
October 5th is Global Awareness Day for Peroxisome Biogenesis Disorders, a group of rare and terminal diseases. We one family’s story and how they now advocate
The US Food and Drug Administration has granted accelerated approval to the gene therapy elivaldogene autotemcel (Skysona) for the treatment of cerebral adrenoleukodystrophy in boys.
An FDA advisory committee gave a unanimous thumbs up to two new gene therapies, one for beta-thalassemia and the other for a rare but fatal neurologic disorder.