Treatment of skin diseases and disorders using inhibitors of kallikrein-related peptidases (klk)
AssigneesExicure Operating Company, SevenScore Pharmaceuticals, LLC
InventorsBart Anderson, Michael Mutolo
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Abstract
Provided herein are compositions and methods for inhibiting the expression and/or activity of kallikrein-related peptidase (KLK) (e.g., KLK5 and KLK7) genes and/or KLK (e.g., KLK5 and KLK7) gene products using oligonucleotide-based therapy. In some embodiments, the compositions are spherical nucleic acids (SNAs) targeting a region of a KLK5 gene and/or gene product and a second synthetic oligonucleotide targeting a region of a KLK7 gene and/or a KLK7 gene product. Such compositions and methods are useful, for instance, in the treatment, prevention, and/or amelioration of diseases or disorders associated with the dysregulation of KLK function.
Claims89
A synthetic oligonucleotide comprising a nucleic acid sequence complementary to or sufficiently complementary to a region of a kallikrein-related peptidase (KLK) gene and/or to a region of a KLK gene product, and at least one locked nucleic acid (LNA) modification or LNA modified nucleoside, or a pharmaceutically acceptable salt thereof.
A synthetic oligonucleotide comprising a nucleic acid sequence complementary to or sufficiently complementary to a region of a kallikrein-related peptidase (KLK) 7 gene and/or to a region of a KLK7 gene product, and at least one modification chosen from a base modification, a sugar modification or an internucleoside linkage modification, or a pharmaceutically acceptable salt thereof.
A spherical nucleic acid (SNA) comprising a core and an oligonucleotide shell, wherein the oligonucleotide shell comprises:
- a first synthetic oligonucleotide comprising a first nucleic acid sequence complementary to or sufficiently complementary to a region of a first kallikrein-related peptidase (KLK) gene that is a KLK5 gene and/or to a region of a first KLK gene product that is a KLK5 gene product;
- and
- a second synthetic oligonucleotide comprising a second nucleic acid sequence complementary to or sufficiently complementary to a region of a second KLK gene that is a KLK7 gene and/or to a region of a second KLK gene product that is a KLK7 gene product.
Dependencies are inferred from the claim text.