285: CRISPR history, biotech struggles, & a big week for deals
Dec 7, 2023
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Megan Molteni discusses the history and future of CRISPR-based medicine and the expected FDA approval of the first CRISPR-based medicine. Bruce Booth joins to discuss the challenges faced by the biotech industry and prospects for optimism in 2024.
The FDA approval of the first CRISPR-based medicine, Cass Jeavy, marks a significant milestone in genome editing, offering hope for future CRISPR-based therapies.
The complexity and high costs of the Cass Jeavy treatment raise concerns about equity in healthcare access for marginalized communities and the need for increased insurance coverage and policy changes to ensure comprehensive care.
Deep dives
The First CRISPR-Based Medicine Expected to Win FDA Approval
A groundbreaking week in genome editing is expected as the first CRISPR-based medicine, Cass Jeavy, developed by CRISPR Therapeutics and Vertex Pharmaceuticals, approaches FDA approval. Cass Jeavy aims to treat sickle cell disease by using CRISPR-Cas9 to increase the production of fetal hemoglobin, which helps prevent sickled red blood cells. The selection of sickle cell disease as the target for this groundbreaking development can be attributed to the suitability of CRISPR's capabilities for addressing this particular condition. This achievement marks a significant milestone in the field of genome editing and paves the way for future CRISPR-based therapies.
The Lengthy Process of Receiving Cass Jeavy and Recovering Post-Treatment
While Cass Jeavy offers a potential breakthrough treatment for sickle cell patients, the actual process of receiving the therapy is complex and time-consuming. Patients undergoing Cass Jeavy treatment will need to undergo bone marrow transplants, which entails collecting their own cells, editing them through CRISPR, and returning them to the body. This involves a four-to-six-month hospital stay and rigorous monitoring throughout the recovery process. The treatment also requires chemotherapy to clear out the existing affected cells and make way for the edited cells, leading to potential side effects such as hair loss and mucosal cell damage. Patients may also choose fertility preservation procedures before undergoing chemotherapy. However, the costs and accessibility of these accompanying services raise concerns, particularly for marginalized communities.
Concerns about Fertility Preservation and Equity in Access to Care
The issue of fertility preservation and its impact on patients undergoing Cass Jeavy treatment raises important questions about equity in healthcare access. While clinical trial participants received free fertility preservation services, the coverage and accessibility of these services for patients outside the trial may be limited. The costs of fertility preservation for patients with sickle cell disease can be significant, potentially creating a financial burden for individuals seeking to preserve their reproductive options. Marginalized populations, who are often disproportionately affected by sickle cell disease, may face additional challenges due to historical disparities in healthcare access and coverage. Advocates and healthcare professionals are calling for policy changes and increased insurance coverage to ensure equitable access to fertility preservation and comprehensive care for patients receiving Cass Jeavy and similar treatments.
Biotech Industry's Outlook and Prospects for 2024
While the biotech industry has faced challenges in recent years, there are signs of optimism for the future. Despite layoffs and financial difficulties faced by some biotech companies, there have been significant acquisitions and collaborations in the industry, indicating a positive trend. The areas of obesity medications and neuroscience, including Alzheimer's treatments, are expected to continue driving momentum and attracting investment. Additionally, there has been a noteworthy increase in mergers and acquisitions in both the public and private biotech sectors. The overall macroeconomic landscape, including factors such as inflation and interest rates, will play a significant role in shaping the industry's performance in 2024. However, the ongoing need for consolidation and addressing the oversaturation of the biotech market remains a topic of discussion and consideration for the future.
With the first CRISPR-based medicine expected to win FDA approval any minute now, STAT’s Megan Molteni joins us to discuss how we got here and what it means for the future. Then, Bruce Booth from Atlas Venture joins us to discuss what was yet another downbeat year for biotech and whether there’s reason for optimism heading into 2024.
