FDA assigns orphan drug and rare pediatric disease designations to SPIRO-2101 for treatment of cystic fibrosis

SPIRO-2101, an inhaled adeno-associated virus gene therapy, is designed to replace a defective cystic fibrosis transmembrane conductance regulator (CFTR) gene in patients with class 1 mutations or in those who are unable to tolerate an existing CFTR modulator.

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Biospace Inc.