Krystal Biotech announced today that the Company will be presenting new preclinical data on respiratory genetic medicine candidates KB407 and KB408 at the American Thoracic Society 2024 International Conference being held from May 17-22, 2024 in San Diego, California. KB407 and KB408 are modified, replication-defective, non-integrating HSV-1 vectors formulated for inhaled delivery to the lung via nebulization. KB407 encodes two full-length copies of cystic fibrosis transmembrane conductance regulator and is under investigation for the treatment of cystic fibrosis irrespective of underlying patient mutation. KB408 encodes two full-length copies of alpha-1 antitrypsin and is under investigation for the treatment of alpha-1 antitrypsin deficiency. Both KB407 and KB408 are being evaluated in ongoing Phase 1 studies. Data presented at ATS will include new preclinical data related to KB407 transduction of fully differentiated, patient airway epithelial cell-derived apical out airway organoids and production of full-length and fully glycosylated CFTR, as well as an overview of KB408 IND-enabling studies conducted to support initiation of the ongoing Phase 1 SERPENTINE-1 study in patients with AATD.
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