Krystal Biotech, Inc.
COMPOSITIONS AND METHODS FOR THE TREATMENT OF SKIN DISEASES

Abstract:

The present disclosure provides recombinant nucleic acids comprising one or more polynucleotides encoding a laminin polypeptide (e.g., a human laminin polypeptide) and/or a filaggrin polypeptide (e g , a human filaggrin polypeptide); viruses comprising the recombinant nucleic acids; compositions and formulations comprising the recombinant nucleic acids and/or viruses; methods of their use (e.g., for the treatment of Junctional Epidermolysis Bullosa); and articles of manufacture or kits thereof.