The FDA announced Friday that it had approved two gene therapies based on CRISPR technology for sickle cell disease, the first cell-based treatments for the disease.
The gene-based therapies, Casgevy and Lyfgenia, are for use in patients 12 years old and older. Both of these therapies are produced by using a patient’s own stem cells.
Casgevy is manufactured by Vertex Pharmaceuticals Inc., while Lyfgenia is manufactured by Bluebird Bio Inc.
“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, director of the Office of Therapeutic Products with 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.”
Cells are collected from a patient, modified using CRISPR, and then infused back into the patient’s system in a one-time dose as a “hematopoietic [blood] stem cell transplant.” These modified cells will then multiply in a patient’s bone marrow and increase production of fetal hemoglobin which will aid oxygen delivery and prevent the spread of red blood cells affected by the disease.
The FDA also detailed two studies that were used to verify the effectiveness and safety of each new therapy. In the Casgevy testing, 29 of the 31 patients treated with the therapy achieved remission from sickled red blood cells restricting the flow in blood vessels and limiting oxygen delivery to the body’s tissues. Lyfgenia was similarly effective, with 28 out of 32 patients achieving the same results.
“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, 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.”
Scientists have long been attempting to use genetic editing to deliver personalized medications to treat illnesses and diseases. Researchers announced earlier this year that they were close to developing a cure for glioblastoma, a type of brain cancer, using genetically engineered brain cancer cells, as previously reported by The Dallas Express.
