The Readout Loud 334: Pharma silence on RFK Jr. & parents trying to develop gene therapies for their children
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Dec 12, 2024 Jason Mast, a colleague from STAT, dives into the intricate world of gene therapy, spotlighting a father's inspiring quest for solutions to help his child. They discuss the significant hurdles in this field, from financial challenges to unique fundraising efforts by families. Meanwhile, the conversation touches on why pharmaceutical companies remain silent on Robert F. Kennedy Jr.'s criticisms, exploring the broader implications for health policy. The emotional journey of families navigating rare conditions sheds light on the urgent need for effective therapies.
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ASH Meeting Focus: CAR-T Therapies
- Adam Feuerstein attended the ASH meeting in San Diego and noted the key takeaway was the "battle" between CAR-T therapies.
- Arcelix's anitocell emerged as superior to Legend Bio and J&J's Carvicti due to its lack of delayed neurotoxicity.
CAR-T Market and Myeloma
- The CAR-T field has seen ups and downs, with multiple myeloma being a significant market for BCMA CAR-Ts.
- Carvicti is projected to generate $1.6 billion in sales next year, highlighting the market's size and interest in Arcelix's drug.
GLP-1 for Addiction
- GLP-1 drugs, typically used for obesity, are being explored for treating addiction.
- They work in the brain to suppress cravings, potentially affecting broader desire circuits beyond food.