Gene Therapy Triumph: The FDA approved two gene treatments for sickle cell disease, a medical breakthrough. Casgevy, produced by Vertex Pharmaceuticals and CRISPR Therapeutics, is the first US medication using Nobel Prize-winning CRISPR gene editing technology. The second approved therapy, bluebird bio’s Lyfgenia, also represents a significant breakthrough.
Sickle cell disease (SCD) is a painful and inherited blood disorder that affects approximately 100,000 people in the United States, with a higher prevalence among individuals of Black descent. The condition leads to the production of flawed, sickle-shaped hemoglobin, impairing the proper transport of oxygen by red blood cells.
Casgevy, priced at $2.2 million, and Lyfgenia, priced at $3.1 million, are positioned as one-time treatments. Both therapies aim to address the underlying genetic causes of SCD through innovative approaches. Casgevy utilizes CRISPR, a revolutionary gene-editing technology discovered by Jennifer Doudna and CRISPR Therapeutics co-founder Emmanuelle Charpentier. CRISPR acts like molecular “scissors,” trimming faulty gene parts and replacing them with normal DNA strands. On the other hand, Lyfgenia employs gene therapy by inserting modified genes into the body through disabled viruses.
The approval of these gene therapies represents a significant advancement for the SCD community, historically overlooked and underfunded. Clinical trials for both therapies demonstrated a reduction in painful episodes among patients, providing hope for improved quality of life.
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Despite the groundbreaking nature of these therapies, concerns and challenges persist. The FDA has added a warning to Lyfgenia’s label regarding the risk of blood cancer, following cases found in bluebird’s trial. The long-term effects of these gene therapies are yet to be fully understood, prompting the companies to conduct a 15-year follow-up study to assess potential safety risks.
Treatment with these gene therapies involves a complex process, including high-dose chemotherapy and potential risks such as infertility. While the therapies offer a promising alternative to conventional treatments, questions about accessibility, insurance coverage, and long-term effects underscore the need for ongoing research and monitoring.
As the medical community celebrates this groundbreaking achievement, attention turns to the potential transformative impact of gene therapies on other genetic disorders. The FDA’s approval sets the stage for a new era in medicine, offering hope to patients with genetic conditions and paving the way for further advancements in gene-editing technologies.
Our Reader’s Queries
What is the biggest downfall of gene therapy?
Although gene therapy has the potential to save lives, its development cost is a major deterrent for pharmaceutical companies. The process of creating a new therapy, which includes clinical trials for government approval, is a costly affair. As a result, the prospect of investing in gene therapy is not very appealing for these companies.
Who is father of gene therapy?
William French Anderson, also known as ‘the father of gene therapy’, made history in 1990 when he administered the first gene therapy to a 4-year-old girl who was struggling with ADA deficiency. Thanks to Anderson’s groundbreaking work, gene therapy has become a promising field of medicine that holds the potential to cure a wide range of genetic diseases.
Who first discovered gene therapy?
While French Anderson, MD, is often referred to as the “father of gene therapy,” the story behind his title is not quite accurate. In 1990, Dr. Anderson led a team that successfully treated a 4-year-old girl with a hereditary immune system disease using gene therapy. However, it was not a complete cure and the treatment had some complications. Nonetheless, Dr. Anderson’s work paved the way for further advancements in gene therapy and he remains a prominent figure in the field.
Is there any successful gene therapy?
Gene therapy clinical trials have demonstrated promising results in treating specific illnesses in humans, including severe combined immune deficiency, hemophilia, and retinitis pigmentosa-induced blindness. These breakthroughs offer hope for those suffering from these debilitating conditions and may pave the way for further advancements in gene therapy research.