All articles tagged with #sickle cell
Researchers at UNSW Sydney have developed a safer CRISPR-based epigenetic editing technique that can switch genes on and off without cutting DNA, offering promising new treatments for genetic diseases like Sickle Cell by reactivating silenced genes through removal of methyl groups, potentially reducing risks associated with traditional gene editing.
The Centers for Medicare and Medicaid Services (CMS) will implement a program to reimburse expensive gene therapies for sickle cell disease based on patient outcomes, marking a significant shift in financing high-cost treatments. Starting next year, Medicaid programs will test outcomes-based agreements to pay for two FDA-approved gene therapies, with list prices of $2.2 million and $3.1 million, respectively.
CRISPR Therapeutics, along with two other companies, faced the challenge of choosing which genetic disease to target first with the revolutionary gene-editing tool CRISPR-Cas9. After considering various options, they decided to focus on sickle cell disease, making it the first disease to be treated using CRISPR.
The US Food and Drug Administration (FDA) is considering the approval of exa-cel, a groundbreaking gene editing treatment for sickle cell disease. Developed by Vertex Pharmaceuticals and CRISPR Therapeutics, exa-cel would be the first FDA-approved treatment that uses CRISPR technology. The treatment involves altering the patient's own stem cells with CRISPR to correct the genetic problems causing sickle cell, and then reintroducing the modified stem cells to the patient. In company studies, the treatment showed positive results, with a significant reduction in painful crises experienced by patients. The FDA sought advice from an independent panel to evaluate the treatment's safety and determine the criteria for approval. The FDA is expected to make a decision by December 8.