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Krystal Biotech Announces Virtual Presentation of Pre-clinical Data on KB408 for the Treatment of Alpha-1 Antitrypsin Deficiency at ESGCT
PITTSBURGH, Oct. 14, 2021 (GLOBE NEWSWIRE) -- Krystal Biotech Inc., (“Krystal”) (NASDAQ: KRYS), the leader in redosable gene therapies for rare diseases,
About this update from Krystal Biotech, Inc.
[{"type":"text","content":"PITTSBURGH, Oct. 14, 2021 (GLOBE NEWSWIRE) -- Krystal Biotech Inc., (“Krystal”) (NASDAQ: KRYS), the leader in redosable gene therapies for rare diseases, today announced that it will present pre-clinical pharmacology data on its second genetic pulmonary disease candidate KB408, for the treatment of alpha-1 antitrypsin deficiency (AATD), at the upcoming European Society of Gene & Cell Therapy (ESGCT) Virtual Congress held October 19 – 22, 2021. AATD is a rare genetic disorder that predisposes affected individuals to develop several complications, including severe and progressive lung disease in adults, and in a minority of patients (~10%) severe liver disease. AATD lung disease is caused by low levels of functional AAT protein in the blood and ultimately the lungs, its primary site of action, where it is needed to prevent tissue from enzymatic destruction. KB408 is an inhaled, redosable gene therapy designed to increase production of normal, fully functional AAT protein directly in the lung tissue to correct the deficiency. Abstract Information:e-Poster Title: Preclinical Pharmacology of KB408, an HSV-1-based Gene Therapy Vector, for the Treatment of Alpha-1 Antitrypsin DeficiencyPoster Number: 40 Full text of accepted abstracts will be made available to registrants on October 19, 2021, at 8:00am CEST. To register for the congress, please visit https://esgctcongress.ada.wats-on.co.uk/Registration.aspx. About AATD and KB408AATD is caused by mutations in the SERPINA1 gene that lead to decreased levels and/or decreased functionality of AAT. The protein is primarily produced in the liver and secreted into the bloodstream, where it acts as a circulating serine protease inhibitor whose principal substrate is neutrophil elastase in the lungs. Over time, the deficiency can lead to progressive enzymatic destruction of the lung tissue, ultimately causing life-threatening pulmonary impairment and severe respiratory insufficiency. Current standard of care is augmentation therapy with purified AAT from pooled human plasma to normalize serum AAT levels with the goal of maintaining protein levels in the lungs. While augmentation therapy has been shown to be effective in reducing the rate of lung destruction, it requires weekly IV infusions and is associated with the risks and limitations of being a human plasma-derived product. As such, there e...