FDA Approves the First Cell-Based Gene Therapy for Rare Disease Community

Last week, the FDA approved Zynteglo, the first gene therapy for individuals with beta-thalassemia. This is a monumental step forward for the entire rare disease community.

Beta-thalassemia is caused by damaged or missing genes. Without treatments, individuals living with beta-thalassemia become dependent on blood transfusions, resulting in lifelong challenges. Zynteglo is a hematopoietic progenitor stem cell gene therapy to treat beta-thalassemia. The elegant therapeutic approach utilizes a patient’s bone marrow stem cells from their blood. The stem cells are personally modified to produce the particular gene that beta-thalassemia patients are naturally missing. Zynteglo is the first cell-based gene therapy of its type to receive approval from the FDA.

FAST has been advancing a very similar program to accelerate therapeutics for all individuals living with Angelman syndrome. The approach, currently being tested on an animal model, has been shown to cross into the brain from the bone marrow cells and impact neurons to replace the particular gene that is missing, meanwhile improving all symptoms of Angelman syndrome. Zynteglo’s exciting announcement further paves the way for future advancements in the rare disease community.