Today FDA announced approval for the first gene therapies for patients with Sickle Cell Disease.

“Sickle cell disease is a rare, debilitating and life-threatening blood disorder with significant unmet need, and we are excited to advance the field especially for individuals whose lives have been severely disrupted by the disease by approving two cell-based gene therapies today,” said Nicole Verdun, M.D., director of the Office of Therapeutic Products within the FDA’s Center for Biologics Evaluation and Research. “Gene therapy holds the promise of delivering more targeted and effective treatments, especially for individuals with rare diseases where the current treatment options are limited.”

Both products are made from the patient’s blood stem cells, which are modified and given back as a one-time, single-dose infusion as part of a hematopoietic (blood) stem cell transplant. Before treatment, a patient’s stem cells are collected, and then the patient must undergo myeloablative conditioning (high-dose chemotherapy), a process that removes cells from the bone marrow so they can be replaced with the modified cells in Casgevy and Lyfgenia. Patients who received Casgevy or Lyfgenia will be followed in a long-term study to evaluate each product’s safety and effectiveness.

“These approvals represent an important medical advance with the use of innovative cell-based gene therapies to target potentially devastating diseases and improve public health,” said Peter Marks, M.D., Ph.D., director of the FDA’s Center for Biologics Evaluation and Research. “Today’s actions follow rigorous evaluations of the scientific and clinical data needed to support approval, reflecting the FDA’s commitment to facilitating development of safe and effective treatments for conditions with severe impacts on human health.”

Casgevy by Vertex Pharma is a cell-based gene therapy for patients 12 years and older with recurrent vaso-occlusive crises. It utilizes CRISPR/Cas9, a genome editing tool that enables removing, adding or altering sections of the DNA sequence.

Lyfgenia by Bluebird Bio is a cell-based gene therapy for patients 12 years and older with sickle cell disease and a history of vaso-occlusive events. Lyfgenia uses a lentiviral vector (gene delivery vehicle) for genetic modification.

Full announcement here.

My earlier blog post on Cell and Gene therapy here.