Krystal Biotech submitted an application in August seeking approval for the initiation of a Phase I clinical trial of KB408. After a 30-day review period, the company was informed that its Investigational New Drug (IND) application was cleared. Krystal Biotech’s clinical development senior vice president, Hubert Chen, expressed his excitement about advancing KB408, the company’s investigational gene therapy for patients with alpha-1 antitrypsin deficiency.
Chen stated that the IND acceptance was a major milestone in their efforts to address a serious lung disease that currently has limited treatment options. The approval also allows the company to demonstrate the potential of its platform to deliver genes repeatedly to the epithelial cells of the lung. The first participant in the trial is expected to be dosed in the first quarter of the coming year.
The single-dose escalation, open-label study aims to evaluate the pharmacodynamics and safety of KB408 in adults with AATD and a PI*ZZ genotype. The study will assess the efficacy, safety, and tolerability of three dosages of KB408 in each cohort of three patients. KB408 is an inhaled formulation of the company’s replication-defective, non-integrating herpes simplex virus type 1 (HSV-1) derived vector.
This vector delivers a functional full-length human serpin family A member 1 (SERPINA1) gene to the airways of the AATD patients. This formulation can be delivered to the respiratory cells of patients via nebulization. In addition to the IND clearance, the FDA granted orphan-drug designation to KB408 for the treatment of AATD this month.
This move by the FDA is significant considering the limited treatment options currently available for AATD, a serious lung disease. The clearance of the IND application for KB408 marks an important step in the development of new therapies for this condition. It also demonstrates the potential of Krystal Biotech’s platform to deliver genes to lung cells, which could have wider implications for the treatment of other diseases.
The single-dose escalation, open-label study will provide valuable insights into the pharmacodynamics and safety of KB408, which could inform the development of future treatment options. The orphan-drug designation awarded by the FDA further emphasizes the potential of KB408 in addressing the needs of AATD patients.
Krystal Biotech’s advancements in the field of gene therapy, as demonstrated by the FDA’s clearance of the IND application for KB408, suggest a promising future for the treatment of AATD and potentially other conditions. The company’s commitment to developing new therapies for serious lung diseases is evident in its ongoing research and development efforts.
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