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.
Claims30
A pharmaceutical composition comprising:
- (a) a herpes virus comprising a recombinant herpes virus genome, wherein the recombinant herpes virus genome comprises one or more polynucleotides encoding a transglutaminase 1 (TGM1) polypeptide;
- and
- (b) a pharmaceutically acceptable excipient.
A method of providing prophylactic, palliative, or therapeutic relief to one or more signs or symptoms of TGM1-deficient autosomal recessive congenital ichthyosis (ARCI) in a subject in need thereof, the method comprising administering to the subject an effective amount of a pharmaceutical composition comprising:
- (a) a herpes virus comprising a recombinant herpes virus genome, wherein the recombinant herpes virus genome comprises one or more polynucleotides encoding a transglutaminase 1 (TGM1) polypeptide;
- and
- (b) a pharmaceutically acceptable excipient;
- wherein the pharmaceutical composition is administered topically, transdermally, or intradermally to the subject;
- and
- wherein administration of the pharmaceutical composition affects a measurable improvement or prevention of the one or more signs or symptoms of TGM1-deficient ARCI.
Dependencies are inferred from the claim text.