In 2023, the FDA approved Vertex’s ”Casgevy,” a CRISPR-based therapy for sickle cell disease. This approval marked the first genetically edited therapy to reach the general market. This therapy, which also received approval from UK regulators to treat another blood disorder called beta thalassemia, works by being administered in a single-infusion of genetically modified stem cells to a patient. Clinical study participants that took Casgevy were free from symptoms associated with sickle cell disease for up to a year, and this approval sets the stage for future nods by regulators for other CRISPR-based therapies that can treat things like HIV, cancers and high blood pressure. According to Nicole Verdun, the 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. A scientist can use CRISPR to explore the origins of diseases such as cancer and pave paths for therapeutics and incurable diagnoses. However, there are barriers to using CRISPR in clinical practice due to the lack of ‘’safe delivery systems to target the tissues and cells.’’ Sanjana told Engadget that while there are concerns, ‘’we definitely don’t want any unintended consequences. There are bits of the genome that if you edit them by mistake, it’s probably no big deal. However, there are other genes that are vitally important.’’ According to Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, the agency’s approval of Casgevy follows ‘’rigorous evaluations of the scientific and clinical data.’’ As of right now, researchers are focused on improving the precision and accuracy of gene editing. Base editing, a CRISPR-derived genome editing method that makes targeted changes to DNA sequences, has been around since 2016. Verve Therapeutics developed a gene edited therapy that can lower cholesterol in patients with a single infusion. At higher doses, the treatment has the potential to reduce proteins associated with bad cholesterol for 2.5 years. And there are trials for gene-edited therapies that target certain cells for cancer and autoimmune diseases expected to begin in 2024. In 2025, we will get a better understanding of how Excision BioTherapeutics’ CRISPR-based therapy works to treat HIV. The application of gene editing as a therapeutic for Alzheimer’s is still in the early stages, and University College London researchers proved that CRISPR has promise as a potential therapeutic for treatment-resistant forms of childhood epilepsy in a recent study. The pricing of CRISPR and related therapies in general will be a huge barrier to access, with the average CRISPR-based therapy estimated to cost between $500,000 and $2 million per patient.
The Evolution of CRISPR-Based Gene Editing in 2023: Triumphs and Trials
