4DMT's inhalable gene therapy for cystic fibrosis shows early positive signs in tiny group of patients
4D Molecular Therapeutics announced Wednesday that its inhalable gene therapy for cystic fibrosis showed signs of improved quality of life in an early look from its Phase I dose exploration study.
The drug, 4D-710, is delivered via nebulizer and is designed to achieve expression of a protein called CFTR in lung airway epithelial cells. CFTR helps keep the balance between salt and water on surfaces including the lung, but a malfunction of the protein can lead to the accumulation of a thick mucus that causes some of the symptoms behind cystic fibrosis.
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