340: Crisis in CRISPR world, RFK Jr.'s promises, and a novel non-opioid pain drug
Feb 6, 2025
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Jason Mast, a reporter for STAT, delves into the troubling downturn in the once-promising CRISPR gene editing field. The discussion highlights the challenges companies face amid market skepticism and compares CRISPR innovations with RNA therapies. The conversation also touches on RFK Jr.'s controversial views as a nominee for HHS secretary, alongside insights into recent pharma earnings that spotlight successes in obesity treatments and the approval of a groundbreaking non-opioid pain drug, revealing the complexities of today’s biotech landscape.
The CRISPR gene editing industry faces significant challenges with layoffs and skepticism over the viability of its commercial therapies.
Vertex Pharmaceuticals has launched a novel non-opioid pain medication, raising questions about affordability and insurance in a competitive market.
Deep dives
The Challenges Facing CRISPR Companies
The CRISPR gene editing industry is currently undergoing significant challenges, marked by widespread layoffs and declining stock prices among most companies in the field. Despite having made initial advancements, the expectation for rapid commercialization of CRISPR-derived therapies has not materialized, with significant market skepticism stemming from the limited number of patients treated with the first approved CRISPR medicine. Many firms, once riding a wave of optimism, are now struggling with tough questions surrounding their business models and the ongoing viability of their treatments. This downturn has led analysts to speculate whether these companies can find sustainable paths forward or if the industry's promise has diminished altogether.
Political Dynamics in Biotech Regulation
The political landscape surrounding biotech regulation continues to evolve, particularly with the recent Senate committee advancing RFK Jr.'s nomination as HHS secretary amidst concerns about his views on vaccines. Despite skepticism from other members regarding his commitment to public health initiatives, including vaccine safety, his confirmation seems well supported. This nomination reflects a broader trend of politicization within health sectors, that could potentially influence the direction of biotech regulation and funding. As the industry navigates these political currents, there are concerns about how policy changes could reshape research priorities and the commercial viability of emerging therapies.
The Emergence of Innovative Pain Management Solutions
Vertex has recently gained approval for Jernivax, a groundbreaking non-opioid pain medication targeting acute pain, which is significant in an era plagued by opioid addiction issues. The novel treatment, however, is priced significantly higher than traditional opioid medications, raising questions about its accessibility and insurance coverage. Vertex's development has been positively received by major insurers, which may pave the way for broader acceptance in the market. The situation underscores the challenge of introducing new therapies in competitive landscapes while addressing rising concerns over opioid dependency.
We bring on our colleague Jason Mast to discuss the dramatic downturn in the once-swaggering field of CRISPR gene editing. We also talk about the advancement of RFK Jr.'s nomination to be HHS secretary, the latest pharma earnings, and the approval of a non-opioid pain drug from Vertex Pharmaceuticals.
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