Compositions and methods for the treatment of autosomal recessive congenital ichthyosis
AssigneeKrystal Biotech, Inc.
InventorsSuma Krishnan, Pooja Agarwal, John C. Freedman, Mark E. O'Malley, Lauren K. Regula
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Abstract
The present disclosure provides recombinant nucleic acids comprising one or more polynucleotides encoding a transglutaminase (TGM) polypeptide (e.g., a Transglutaminase-1 (TGM1) polypeptide); viruses comprising the recombinant nucleic acids; compositions comprising the recombinant nucleic acids and/or viruses; methods of their use; and articles of manufacture or kits thereof.
Claims21
A pharmaceutical composition comprising:
- (a) a replication defective herpes simplex virus type 1 (HSV-1) comprising a recombinant HSV-1 genome, wherein the recombinant HSV-1 genome comprises one or more polynucleotides encoding a transglutaminase (TGM) polypeptide;
- and
- (b) a pharmaceutically acceptable excipient.
A method of delivering one or more polynucleotides encoding a transglutaminase polypeptide into one or more cells of a subject, the method comprising administering to the subject an effective amount of a pharmaceutical composition comprising:
- (a) a replication defective herpes simplex virus type 1 (HSV-1) comprising a recombinant HSV-1 genome, wherein the recombinant HSV-1 genome comprises the one or more polynucleotides encoding a human transglutaminase (TGM) polypeptide;
- and
- (b) a pharmaceutically acceptable excipient.
Dependencies are inferred from the claim text.