The risk posed by Trump tariffs on rare disease, cell, and gene therapies is a concern for the industry. With over 10,000 rare diseases lacking FDA-approved therapies, preserving incentives is crucial for public health. BioMarin, a company with approved gene therapies like Roctavian for hemophilia A, emphasizes the need to sustain innovation in this ecosystem despite financial challenges.
The potential impact of tariffs on these therapies underscores the delicate balance between promoting innovation and ensuring accessibility. As companies struggle to achieve profitability while advancing treatments for neglected diseases, policy decisions can significantly influence the future of rare disease therapies. Addressing these challenges requires a comprehensive approach that considers both economic sustainability and patient access to life-changing treatments.
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