Pharmaceutical composition for treating non-alcoholic fatty liver, non-alcoholic steatohepatitis, or hepatic fibrosis using Ssu72 protein or a polynucleotide encoding the same
Inventors
Lee, Chang Woo • LEE, Jin Kwan • Kim, Hyun Soo • CHOI, Ji Hyun • BYEON, Se Eun • Lee, Hae In • JO, HYEONJU
Assignees
Interested in licensing this patent?
MTEC can help explore whether this patent might be available for licensing for your application.
Publication Number
US-11793862-B2
Publication Date
2023-10-24
Expiration Date
Abstract
The present invention provides a method for preventing or treating a liver disease selected from the group consisting of non-alcoholic fatty liver, non-alcoholic steatohepatitis and hepatic fibrosis comprising administrating at least one selected from the group consisting of an Ssu72 peptide, a polynucleotide encoding the Ssu72 peptide, and an expression vector comprising the polynucleotide.
Core Innovation
The disclosed invention provides pharmaceutical compositions and methods for preventing or treating liver diseases selected from non-alcoholic fatty liver, non-alcoholic steatohepatitis and hepatic fibrosis. The approach administers to a patient in need thereof, via systemic administration or intra-hepatic injection, at least one selected from an Ssu72 peptide, a polynucleotide encoding the Ssu72 peptide, and an expression vector comprising the polynucleotide, with the polynucleotide operably linked to a promoter so that Ssu72 peptide expression can be achieved in the liver.
A central aspect is patient stratification based on reduced or inactive Ssu72 in the liver. The patient or subject shows lower expression or activity of the Ssu72 peptide in the liver than a normal subject, and this lower Ssu72 expression or activity is used as a defining condition for administration of the Ssu72 peptide, polynucleotide, or expression vector.
The invention further includes selection of the polynucleotide and promoter systems, including liver-specific promoters. The text describes promoters that may be constitutive, inducible, or liver-specific, and includes named liver-specific promoter options such as thyroxin-binding globulin promoter, PBGD promoter, α-1 anti-trypsin promoter, and Hepatic Control Region-based hybrid promoters, with viral vector embodiments including an AAV8 vector.
Claims Coverage
The document includes two independent methods that cover therapeutic and preventive uses. Both independent claims require systemic administration or intra-hepatic injection of an Ssu72 peptide, a polynucleotide encoding it, and/or an expression vector with a promoter, where the subject shows lower Ssu72 expression or activity in the liver than a normal subject.
Treating selected liver disease by Ssu72 delivery under lower Ssu72 expression/activity condition
A method for treating a patient suffering from a liver disease selected from non-alcoholic fatty liver, non-alcoholic steatohepatitis and hepatic fibrosis comprising administrating via systemic administration or intra-hepatic injection to the patient at least one selected from an Ssu72 peptide, a polynucleotide encoding the Ssu72 peptide, and an expression vector comprising the polynucleotide, wherein the polynucleotide is operably linked to a promoter, and wherein the patient shows lower expression or activity of Ssu72 peptide in the liver than a normal subject.
Preventing selected liver disease by Ssu72 delivery under lower Ssu72 expression/activity condition
A method for preventing a liver disease selected from non-alcoholic fatty liver, non-alcoholic steatohepatitis and hepatic fibrosis, comprising administering at least one selected from an Ssu72 peptide having an amino acid sequence represented by SEQ ID NO: 1, a polynucleotide encoding the Ssu72 peptide, and an expression vector containing the polynucleotide, wherein the polynucleotide is operably linked to a promoter, and wherein the subject shows lower expression or activity of Ssu72 peptide in the liver than a normal subject.
Overall, the independent claims define Ssu72 peptide or Ssu72-encoding polynucleotide or expression vector administration, via systemic administration or intra-hepatic injection, to subjects with lower Ssu72 expression or activity in the liver. Dependent claims further narrow implementation by specifying SEQ ID NO: 1 for the Ssu72 peptide, SEQ ID NO: 2 for the encoding polynucleotide, liver-specific promoter types and sequences, and viral vector embodiments including an AAV8 vector.
Stated Advantages
Prevents a liver disease selected from non-alcoholic fatty liver, non-alcoholic steatohepatitis and hepatic fibrosis.
Treats a liver disease selected from non-alcoholic fatty liver, non-alcoholic steatohepatitis and hepatic fibrosis.
Documented Applications
Prevention and treatment of non-alcoholic fatty liver, non-alcoholic steatohepatitis, and hepatic fibrosis by administering Ssu72 peptide, Ssu72-encoding polynucleotide, and/or an expression vector operably linked to a promoter to subjects or patients showing lower Ssu72 expression or activity in the liver [procedural detail omitted for safety].
Interested in licensing this patent?