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Advances in Gene-Editing Therapy Show Promise for Sickle Cell Disease


Shares of Crispr Therapeutics and Vertex Pharmaceuticals Inc. saw an increase in premarket trading following the momentous approval of the world’s first CRISPR-based medicine by a U.K. regulator.

With this groundbreaking regulatory action, the president and CEO of Vertex, Dr. Reshma Kewalramani, expressed her excitement, stating, “Today is a historic day in science and medicine.”

Casgevy functions by editing the defective gene in patients’ bone marrow stem cells, stimulating the production of functional hemoglobin. Hemoglobin is a vital protein in red blood cells responsible for delivering oxygen to tissues. The process involves the extraction of stem cells from the bone marrow, editing them in a laboratory, and subsequently infusing them back into the patient. The Medicines and Healthcare Products Regulatory Agency of the U.K. affirms that patients may require hospitalization for approximately one month to allow the treated cells to integrate into the bone marrow.

Advances in Gene-Editing Therapy Show Promise for Sickle Cell Disease

The field of gene-editing therapy continues to make breakthroughs, with revolutionary treatment options on the horizon. Crispr Therapeutics, a pioneering biotech company, has developed a therapy known as exa-cel that is currently undergoing a review by the U.S. Food and Drug Administration (FDA). The FDA has even granted priority review for exa-cel in the context of sickle cell disease and is set to make a decision by December 8.

The positive reception from an FDA advisory panel has contributed to the soaring success of Crispr Therapeutics shares. While it is important to note that the FDA is not obligated to follow the recommendations of such panels, it typically considers their advice seriously. In this case, the outlook for exa-cel appears promising.

Industry analysts at Evercore ISI have expressed their confidence, stating that exa-cel is highly likely to receive approval from the FDA by early December. Additionally, an FDA decision on exa-cel’s application in thalassemia is expected by March 30.

CEO and chairman of CRISPR Therapeutics, Samarth Kulkarni, expressed his hope for the future impact of this groundbreaking technology on patients with serious diseases. However, it is crucial to remember that while these advancements are exciting, they must go through rigorous testing and approval processes to ensure their safety and efficacy.

As a result of these promising developments, Crispr Therapeutics shares have experienced significant growth. In fact, premarket trading on Thursday saw an increase of over 5%, contributing to a 38% overall increase for the year. Similarly, Vertex shares also saw a slight boost of 0.5% in premarket trading.

It is clear that gene-editing therapy holds tremendous potential and may pave the way for innovative solutions in the treatment of various genetic diseases. While awaiting the FDA’s decision, the medical community eagerly awaits the outcome, hoping that this heralds the beginning of a new era in patient care.

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