BMD003

BMD003 is an **inhaled mRNA therapy** that delivers messenger RNA encoding the cystic fibrosis transmembrane conductance regulator (**CFTR**) protein. It is designed as a gene therapy for patients with **cystic fibrosis**. The delivered mRNA is expected to enable recipient airway cells to transiently express functional CFTR protein, addressing the underlying defect in cystic fibrosis patients who have mutations in the CFTR gene. The drug is developed and being investigated by Peking Union Medical College Hospital. The primary indication is cystic fibrosis, with early clinical trials focusing on safety and efficacy in adult patients. The therapy is administered via nebulized inhalation, and clinical evaluation is at the early phase 1 stage in China[1][3][5].

Other names

BMD003BMD-003BMD 003