Compositions and methods for treatment of netherton syndrome
AssigneeKrystal Biotech, Inc.
InventorsPooja AGARWAL, Suma KRISHNAN, John C. FREEDMAN
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Abstract
The present disclosure provides recombinant nucleic acids comprising one or more polynucleotides encoding a 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.
Claims31
1-6. (canceled)
A method of providing therapeutic relief to one or more signs or symptoms of Netherton Syndrome (NS) or atopic dermatitis (AD) 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 one or more polynucleotides encoding a Serine Protease Inhibitor Kazal-type 5 (SPINK5) polypeptide;
- and
- (b) a pharmaceutically acceptable excipient,
- wherein the one or more polynucleotides encoding the SPINK5 polypeptide are operably linked to a promoter suitable for transcription in a mammalian cell, and
- wherein the pharmaceutical composition is administered topically, transdermally, subcutaneously, intradermally, or transmucosally.
A method of enhancing, increasing, augmenting, and/or supplementing the levels of a Serine Protease Inhibitor Kazal (SPINK) polypeptide in 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 comprising a recombinant herpes simplex virus genome, wherein the recombinant herpes simplex virus genome comprises one or more polynucleotides encoding the SPINK 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 one or more polynucleotides encoding a SPINK polypeptide;
- and
- (b) a pharmaceutically acceptable excipient.
Dependencies are inferred from the claim text.