FDA approved Gene Therapies for Sickle Cell Disease | Cromos Pharma

FDA Greenlights Groundbreaking Gene Therapies for Sickle Cell Disease

The FDA has given the nod to two pioneering treatments, Casgevy and Lyfgenia, marking a significant milestone as the first cell-based gene therapies for sickle cell disease (SCD) in patients aged 12 and older. Notably, Casgevy stands out as the first FDA-approved treatment employing a novel genome editing technology, CRISPR/Cas9, marking a major leap forward in gene therapy.

Sickle cell disease, impacting approximately 100,000 people in the U.S., predominantly affects African and Hispanic Americans due to a genetic mutation in hemoglobin, a crucial protein delivering oxygen to the body’s tissues.

Casgevy, a CRISPR/Cas9-utilizing gene therapy, is greenlit for patients aged 12 and above experiencing recurrent vaso-occlusive crises. The therapy involves modifying patients’ hematopoietic stem cells using CRISPR/Cas9 technology.

Lyfgenia, another cell-based gene therapy, utilizes a lentiviral vector for genetic modification and is approved for patients aged 12 and older with sickle cell disease and a history of vaso-occlusive events. It modifies blood stem cells to produce HbAT87Q, a gene-derived hemoglobin mimicking the normal adult hemoglobin.

Both therapies are crafted from the patients’ own modified blood stem cells, administered as a one-time infusion as part of a hematopoietic stem cell transplant.

Casgevy’s safety and efficacy were assessed in an ongoing trial, demonstrating a 93.5% success rate in preventing severe vaso-occlusive crises. Lyfgenia’s approval is based on a multicenter study, with 88% of patients achieving complete resolution of vaso-occlusive events between 6 and 18 months post-infusion.

Casgevy and Lyfgenia were developed by Vertex Pharmaceuticals Inc. and Bluebird Bio Inc., respectively. These groundbreaking approvals underscore the potential of innovative cell-based gene therapies in tackling severe diseases and advancing public health.

If you have any questions or want to find out more about how Cromos Pharma can support your next clinical trial please contact us by emailing inquiry@cromospharma.com.



To arrange an introductory meeting and find out how our experience can benefit your next clinical project.