The biotech industry is sounding the alarm as Trump’s tariffs put rare disease cell and gene therapy at risk. With over 10,000 rare diseases still lacking FDA-approved therapies, companies like BioMarin are urging the preservation of incentives to sustain innovation. BioMarin, known for gene therapies like Roctavian for hemophilia A, is facing financial challenges despite breakthrough medicines for neglected diseases. As the debate rages on, the future of cell therapy in regenerative medicine hangs in the balance.
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