Krystal Biotech to Present at ATS 2024 Conference

May 15, 2024 – Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS), a biotechnology firm in the commercial stage, has announced its participation in the American Thoracic Society (ATS) 2024 International Conference. This event will take place from May 17-22, 2024, in San Diego, California. The company will present novel preclinical data for its respiratory genetic medicine candidates, KB407 and KB408.

KB407 and KB408 are designed as modified, replication-defective, non-integrating HSV-1 vectors. They are formulated for inhaled delivery to the lungs using nebulizers. KB407 is engineered to encode two full-length copies of the cystic fibrosis transmembrane conductance regulator (CFTR) and is being investigated as a treatment for cystic fibrosis, regardless of the specific genetic mutation present in the patient. On the other hand, KB408 encodes two full-length copies of alpha-1 antitrypsin and is being studied as a treatment for alpha-1 antitrypsin deficiency (AATD).

Both KB407 and KB408 are currently undergoing Phase 1 clinical trials. The data to be presented at the ATS conference will include new preclinical results concerning KB407’s ability to transduce fully differentiated airway epithelial cells derived from patients, specifically focusing on the production of full-length and fully glycosylated CFTR. Additionally, the presentation will provide an overview of the investigational new drug (IND)-enabling studies for KB408, which support the initiation of the ongoing Phase 1 SERPENTINE-1 study in AATD patients.

Details of the poster presentations are as follows:

1. Title: Murine Toxicology Study of Repeat-Dose Inhaled KB408, an HSV-1-Based Vector for the Treatment of Alpha-1 Antitrypsin Deficiency
Presenter: Sara Artusi, PhD
Date and Time: May 20, 2024, from 11:30 AM to 1:15 PM PT
Poster Session: B70: COPD in the Spotlight: Insights into Disease Pathogenesis
Poster Number: P659

2. Title: Evaluation of KB407, an HSV-1-Based Gene Therapy Vector for the Treatment of Cystic Fibrosis, in Healthy and Patient-Derived Airway Cells Including an Apical-out Diseased Airway Organoid Model
Presenter: Sara Artusi, PhD
Date and Time: May 21, 2024, from 11:30 AM to 1:15 PM PT
Poster Session: C70: From Buds to Breath: Mechanisms and Pathogenesis of Lung Development
Poster Number: P122

Krystal Biotech, Inc. is a commercial-stage biotechnology company dedicated to discovering, developing, and commercializing genetic medicines aimed at treating diseases with significant unmet medical needs. VYJUVEK® is the Company’s first commercial product, notable for being the first-ever redosable gene therapy and the first FDA-approved treatment for dystrophic epidermolysis bullosa. The Company is advancing a robust pipeline of investigational genetic medicines across various fields, including respiratory, oncology, dermatology, ophthalmology, and aesthetics. Krystal Biotech is headquartered in Pittsburgh, Pennsylvania.

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