On December 8, 2023, the U.S. Food and Drug Administration (FDA) approved two innovational gene therapies, Casgevy and Lyfgenia, for treating sickle cell disease (SCD) in patients aged 12 and older. This marks the first approval of cell-based gene therapies for SCD, with Casgevy also being the first to use CRISPR/Cas9 genome editing technology.

SCD, primarily affecting African and Hispanic Americans, involves a mutation in hemoglobin that leads to red blood cells developing a sickle shape. This mutation causes severe pain, organ damage, and potentially life-threatening complications.

Casgevy is designed for patients with recurrent vaso-occlusive crises (VOCs), which employs CRISPR/Cas9 to edit patients’ hematopoietic stem cells. These modified cells, once reinfused into the patient, increase fetal hemoglobin production, reducing red blood cell sickling. Lyfgenia, intended for patients with a history of vaso-occlusive events (VOEs), uses a lentiviral vector to modify stem cells to produce HbAT87Q, a therapeutic hemoglobin that lowers the sickling risk through cell-based gene therapy. Both treatments involve collecting and modifying the patient’s own blood stem cells, followed by a one-time infusion after high-dose chemotherapy (myeloablative conditioning).

Clinical trials demonstrated Casgevy’s efficacy, with over 93% of patients achieving freedom from severe VOCs. Lyfgenia showed 88% of patients had complete resolution of VOEs. Common side effects for both include low blood cell counts and mouth sores. Lyfgenia carries a black box warning for the risk of blood cancer.

These therapies, which received Priority Review and other FDA designations, are developed by Vertex Pharmaceuticals Inc. (Casgevy) and Bluebird Bio Inc. (Lyfgenia). The FDA’s approval highlights its commitment to advancing treatments for severe health conditions.

Source:
https://www.genengnews.com/topics/genome-editing/fda-approves-casgevy-the-first-crispr-therapy-for-sickle-cell-disease/
About PackGene

PackGene is a CRO & CDMO technology company that specializes in packaging recombinant adeno-associated virus (rAAV) vectors. Since its establishment in 2014, PackGene has been a leader in the AAV vector CRO service field, providing tens of thousands of custom batches of AAV samples to customers in over 20 countries. PackGene offers a one-stop CMC solution for the early development, pre-clinical development, clinical trials, and drug approval of rAAV vector drugs for cell and gene therapy (CGT) companies that is fast, cost-effective, high-quality, and scalable. Additionally, the company provides compliant services for the GMP-scale production of AAVs and plasmids for pharmaceutical companies, utilizing five technology platforms, including the π-Alpha 293 cell AAV high-yield platform and the π-Omega plasmid high-yield platform. PackGene's mission is to make gene therapy affordable and accelerate the launch of innovative gene drugs. The company aims to simplify the challenging aspects of gene therapy development and industrialization processes and provide stable, efficient, and economical rAAV Fast Services to accelerate gene and cell therapy development efforts from discovery phase to commercialization.

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