Compositions and Methods for the Treatment of ATPase Disorders or Diseases of the Skin
AssigneeKrystal Biotech, Inc.
InventorsSuma Krishnan, Mary Jane Duermeyer, Bruce Chike Nmezi, Trevor Parry
Life of this patent
Estimating the term…
Abstract
The present disclosure provides recombinant nucleic acids comprising one or more polynucleotides encoding an ATPase polypeptide (e.g., a calcium-transporting ATPase type 2C member 1 polypeptide or a sarcoplasmic/endoplasmic reticulum calcium ATPase 2 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 treating an ATPase disorder or disease of the skin, for example, Hailey-Hailey disease or Darier disease); and articles of manufacture or kits thereof.
Claims27
-72. (canceled)
A pharmaceutical composition comprising:
- (a) a replication-defective herpes simplex virus comprising a recombinant herpes simplex virus genome, wherein the recombinant herpes simplex virus genome comprises a polynucleotide encoding a calcium-transporting ATPase type 2C member 1 (ATP2C1) polypeptide;
- and
- (b) a pharmaceutically acceptable excipient.
A pharmaceutical composition comprising:
- (a) a replication-defective herpes simplex virus comprising a recombinant herpes simplex virus genome, wherein the recombinant herpes simplex virus genome comprises a polynucleotide encoding a sarcoplasmic/endoplasmic reticulum calcium ATPase 2 (ATP2A2) polypeptide;
- and
- (b) a pharmaceutically acceptable excipient.
A method of providing prophylactic, palliative, or therapeutic relief of one or more signs or symptoms of Darier disease in a subject in need thereof, the method comprising administering to the subject an effective amount of a pharmaceutical composition comprising:
- (a) a replication-defective herpes simplex virus comprising a recombinant herpes simplex virus genome, wherein the recombinant herpes simplex virus genome comprises a polynucleotide encoding an ATP2A2 polypeptide;
- and
- (b) a pharmaceutically acceptable excipient.
Dependencies are inferred from the claim text.