First gene treatment is now approved by FDA to treat human illness!

In a groundbreaking decision, the Food and Drug Administration (FDA) has given its approval for the first gene-editing treatment aimed at addressing severe sickle cell disease. This approval marks a significant milestone for both sickle cell treatment and the rapidly advancing field of gene therapy, generating excitement for potential applications in treating various diseases.

The FDA’s approval includes two gene therapies specifically for individuals aged 12 and older suffering from the most severe form of sickle cell disease. This blood disorder, often overlooked by medical research, has now gained attention with the introduction of innovative treatments.

Dr. Nicole Verdun, Director of the Office of Therapeutic Products at the FDA’s Center for Biologics Evaluation and Research, expressed enthusiasm about advancing treatments for those whose lives have been severely impacted by sickle cell disease. The gene therapies, developed using the CRISPR technique, hold the promise of more targeted and effective treatments, especially for rare diseases with limited current options.

 

The CRISPR treatment, named CASGEVY, involves extracting cells from a patient’s bone marrow, editing a gene using CRISPR, and then infusing the modified cells back into the patient. While not a cure, this one-time treatment aims to alleviate symptoms for a lifetime by producing fetal hemoglobin, restoring normal red blood cell function.

Data presented to the FDA demonstrated positive outcomes, with the treatment resolving severe pain crises for 96.7% of subjects for at least 18 months. Another gene therapy by bluebird bio inc. was also approved by the FDA for sickle cell disease, employing a different approach that does not involve CRISPR.

However, the elation over these approvals is tempered by concerns about accessibility. The complex and arduous nature of the procedures, along with potential high costs, may pose challenges for widespread adoption. The treatments, requiring multiple hospital visits, bone marrow transplants, and extended hospitalization, might be daunting for many patients, limiting their accessibility.

Despite the transformative potential of gene-editing treatments, there is a need for further efforts to make these groundbreaking therapies widely available. Researchers and advocates emphasize the importance of addressing logistical challenges, ensuring affordability, and conducting thorough research on potential long-term effects. The journey towards accessible gene-editing treatments is ongoing, with hopes that innovations like CRISPR will pave the way for more breakthroughs in medical science.

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