DeepSummary
The podcast episode discusses a potential landmark treatment for sickle cell disease using gene editing technology. Politico healthcare reporter Lauren Gardner explains how the treatment called exacerbated works by removing a patient's stem cells, editing them using CRISPR to produce healthy red blood cells, and then transplanting the edited cells back into the patient after chemotherapy.
Gardner notes that the treatment has undergone extensive study and appears to have a robust safety profile based on the data submitted to the FDA. It is intended for patients with severe forms of sickle cell disease, which affects around 10-20% of the estimated 100,000 people with the condition in the U.S.
One of the major concerns regarding the treatment is its expected high cost, potentially over $1 million per treatment, and the challenges around insurance coverage and access. The FDA is expected to make a decision on approving the treatment by December 8th.
Key Episodes Takeaways
- A potential landmark gene editing treatment called exacerbated for severe sickle cell disease is nearing FDA approval decision
- The treatment involves removing stem cells, editing them with CRISPR to produce healthy cells, then transplanting them back after chemotherapy
- It has undergone extensive study and has a robust safety profile based on data submitted to the FDA
- The treatment would be intended for the 10-20% of sickle cell patients with the most severe form of the disease
- A major challenge will be the expected high cost of over $1 million per treatment and issues around insurance coverage and access
- The FDA is expected to make its decision on approval by December 8th
- The broad topics covered include gene editing, CRISPR technology, sickle cell disease treatments, drug costs and insurance
- Key developments around landmark medical treatments and concerns over patient access are highlighted
Top Episodes Quotes
- “So then the new disease free cells are transplanted back into the patient after that process is complete.“ by Lauren Gardner
- “As of right now, this therapy is intended for folks with the most severe forms of sickle cell disease. So one way that that's being defined is people who have had to go to the hospital, for example, for intense pain episodes due to the disease.“ by Lauren Gardner
- “Gene therapies typically fetch a million plus dollars per treatment. And there's a lot of unknowns with respect to how insurance plans intend to cover this.“ by Lauren Gardner
- “December 8 is when FDA is expected to make a decision.“ by Lauren Gardner
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Episode Information
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POLITICO
12/4/23