Commercialization of new gene-editing technology in beta-thalassemia –

Gene therapies and research into them have grown tremendously in recent years, providing more innovative tools in regenerative medicine to fight diseases, including rare diseases and genetic disorders. Over the past decade, there has also been rapid development and interest in CRISPR/Cas9 technology and other gene-editing tools that could offer transformative pathways to deliver gene therapies to patients and families. affected by devastating diseases such as sickle cell anemia and thalassemia.

Beta-thalassemia is a rare blood disease caused by a genetic defect in hemoglobin. Several manufacturers are developing new treatments for the disease, including Vertex, which has partnered with CRISPR Therapeutics to develop a gene-editing treatment for beta-thalassemia and sickle cell disease. In October 2022, companies announced their plan to file exagamglogene autotemcel, the CRISPR/Cas9-edited therapy, for continued review with the FDA. Novartis also recently signed an up to $1.5 billion contract with Precision Biosciences to support its development of point treatments for beta-thalassemia and sickle cell disease. This decision marks the continuation of its ongoing commitment to exploring gene editing technology.

Despite renewed interest from scientific and financial players, there remain many unique challenges at each step of this new frontier. From timely therapy development and funding, to market access challenges due to lack of maturity and broad evidence, and explaining complex science to physicians and patients, there are a variety of barriers. to overcome before bringing this type of technology to market. . And while regenerative and curative therapies are often highly valued, there are also challenges in treating affected patient populations, including cost, accessibility, side effects, and other associated risks.

Earlier this year, the U.S. Food and Drug Administration (FDA) approved the first potentially curative gene therapy to treat beta-thalassemia. While several companies have worked to bring transformative technologies such as CRISPR to market, approval will require further consideration of the many concerns and challenges of each stakeholder at every stage. From the research and development phase to patient access and treatment, it is essential to remain attentive to the populations who will ultimately be the end users and who will witness the success of the technology.

Bring the patient’s point of view

Diagnosed with beta-thalassemia at six months old, Amar received regular blood transfusions every few weeks for most of his life. As a child, he found himself falling behind others his age. “I always felt like I was catching up, through school, work, life — always catching up,” he says.

Now in his 40s, he reflects on the physical and mental impacts of the disease. He says: “As I get older, my body deteriorates and that raises concerns about my future and what my life expectancy will be like. The disease triggered other health issues including osteoarthritis, hypogonadism and diabetes. It’s a constant challenge, both physically and mentally,” he says.

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