Biotech
Thursday, December 7th, 2023 2:51 pm EDT
Key Points
- Breakthrough in Sickle Cell Disease Treatment: The article highlights a groundbreaking gene therapy, exa-cel, developed by Vertex Pharmaceuticals and CRISPR Therapeutics, as a potential treatment for sickle cell disease. Sickle cell disease, an inherited blood disorder, causes excruciating pain and has limited treatment options, with a bone marrow transplant being the only cure. The therapy utilizes CRISPR technology to edit DNA and alleviate symptoms, with U.S. regulators expected to approve it soon after the U.K. approved it under the name Casgevy last month.
- Challenges and Milestones in Gene Editing: The article discusses the challenges and milestones associated with gene-editing technology, specifically CRISPR-Cas9, which researchers Jennifer Doudna and Emmanuelle Charpentier introduced in 2012. Sickle cell disease, considered the first molecular disease by scientist Linus Pauling in 1949, has become a prime target for gene-editing treatments. The therapy involves editing genes to activate fetal hemoglobin, facilitating red blood cells’ healthy shape. Vertex, in collaboration with CRISPR, aims to lead in launching exa-cel, anticipating a multibillion-dollar opportunity.
- Financial and Accessibility Concerns: The potential approval of exa-cel raises financial and accessibility concerns for the American health-care system. Wall Street anticipates a price tag of around $2 million per patient, making it a test case for the system. Analysts are skeptical about exa-cel’s profitability, projecting $1.2 billion in sales for Vertex in 2028. While the treatment shows promise in reducing pain crises, the lengthy timeline, risk of chemotherapy-induced infertility, limited availability at specialized facilities, and high cost pose challenges for some patients. Despite these challenges, patients like Joe Tsogbe, who participated in the clinical trial, see the potential cure as invaluable, emphasizing the life-changing impact of the treatment.
At 19, Joe Tsogbe underwent his first hip replacement due to sickle cell disease, an inherited blood disorder causing excruciating pain. By his 30s, he averaged over a dozen hospitalizations annually. Sickle cell disease results from a genetic mutation causing red blood cells to form half moons and obstruct blood vessels. It primarily affects about 100,000 people in the U.S., predominantly Black individuals, with limited available treatments.
Vertex Pharmaceuticals and CRISPR Therapeutics developed a gene therapy, exa-cel, utilizing CRISPR technology to edit DNA and alleviate sickle cell symptoms. U.S. approval is anticipated soon, following the UK’s approval as Casgevy. Another gene therapy, lovo-cel from Bluebird Bio, is under U.S. review, aiming to be approved later this month. These therapies signify a scientific milestone, offering potential relief and eliminating the need for bone marrow donors.
However, exa-cel’s potential approval poses a major test for the U.S. health-care system, with a projected cost of $2 million per patient, raising concerns about accessibility for tens of thousands of eligible individuals. The treatment involves extracting, genetically modifying, and infusing blood stem cells, requiring months and chemotherapy. Vertex sees exa-cel as a multibillion-dollar opportunity, focusing on severe cases and seeking approval for beta thalassemia.
Despite the groundbreaking potential, Wall Street remains skeptical about exa-cel’s profitability, projecting $1.2 billion in sales for Vertex in 2028. Challenges include the lengthy treatment timeline, chemotherapy-induced infertility risk, limited availability at specialized facilities, and the high cost. Wall Street expects insurers to be reluctant to widely cover exa-cel, despite it being less expensive than some gene therapies exceeding $3 million per person.
For patients like Tsogbe, who participated in the exa-cel trial, the potential cure outweighs the challenges. Despite not erasing prior damage, the treatment has kept him out of the hospital, allowing him to pursue his passions and fulfill a promise he made to find a cure for sickle cell.
For the full original article on CNBC, please click here: https://www.cnbc.com/2023/12/07/crispr-gene-editing-treatment-us-approval.html
