Dr. Stephan Grupp, a leading expert in cellular therapy and transplantation at the Children’s Hospital of Philadelphia, has hailed the use of gene editing as a “miracle” in the treatment of sickle cell disease. However, he acknowledges that there are significant challenges that need to be overcome before widespread adoption can occur.
One of the main obstacles is the limited number of authorized medical centers that are equipped to perform this innovative treatment. Additionally, since gene editing needs to be personalized for each patient, it requires onerous procedures that can be time-consuming and costly.
Furthermore, potential insurance barriers make it difficult for patients to access this life-changing treatment. Experts estimate that only a small fraction of sickle cell patients in the United States will be able to receive this new therapy.
The situation is even more dire for sickle cell patients overseas, particularly in Africa, where the treatment is expected to be completely out of reach for millions of individuals.
The breakthrough treatment, named Exa-cel and marketed as Casgevy, is a result of a collaboration between Vertex Pharmaceuticals and CRISPR Therapeutics. Casgevy is the first treatment that has been approved for use with the Nobel Prize-winning gene editing tool CRISPR.
Although clinical trials have shown promising results, with Casgevy correcting the effects of the sickle cell mutation in a small number of subjects, it is important to note that patients will also require costly intensive medical care and an extended hospitalization in addition to the gene editing treatment.
While the use of gene editing in treating sickle cell disease presents a potential solution to a devastating condition, it is clear that there are significant hurdles that need to be overcome before this treatment can be widely accessible to patients in need.
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