Crispr/cas-related methods and compositions for knocking out c5
US-2024415980-A1 · Dec 19, 2024 · US
US2026091136A1 · US · A1
| Field | Value |
|---|---|
| Publication number | US-2026091136-A1 |
| Application number | US-202519407768-A |
| Country | US |
| Kind code | A1 |
| Filing date | Dec 3, 2025 |
| Priority date | Jun 23, 2023 |
| Publication date | Apr 2, 2026 |
| Grant date | — |
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Methods for preventing and/or treating a cardiometabolic disease, e.g., heart failure with preserved ejection fraction (HFpEF) using Adropin are provided herein. The presently disclosed subject matter further relates to gene therapy methods for providing endogenous production of Adropin.
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What is claimed is: 1 . A method for preventing and/or treating a subject having cardiometabolic disease, comprising administering a therapeutically effective amount of an Adropin-based therapy. 2 . The method of claim 1 , wherein the Adropin-based therapy is a composition comprising a virus, wherein the virus comprises a heterologous nucleic acid encoding an Energy Homeostasis-associated (ENHO) gene. 3 . The method of claim 2 , wherein the virus is administered into the liver of the subject. 4 . The method of claim 2 , wherein the virus is administered to hepatocytes of the subject. 5 . The method of claim 2 , wherein the heterologous nucleic acid is operably linked to a promoter selected from the group consisting of thyroxine binding globulin (TBG), albumin (ALB), hepatitis virus (HBV), alpha-1 antitrypsin (AAT), and human cytomegalovirus (CMV). 6 . The method of claim 2 , wherein the virus is an adenoviral associated virus (AAV). 7 . The method of claim 6 , wherein the AAV isotype is selected from the group consisting of AAV1, AAV2, AAV3, AAV6, AAV6.2, AAV7, AAV8, AAV9, AAVrh10, AAV/DJ, and AAV/DJ8. 8 . The method of claim 2 , wherein the ENHO gene comprises SEQ ID NO.: 3. 9 . The method of claim 1 , wherein the Adropin-based therapy is a recombinant Adropin peptide. 10 . The method of claim 9 , wherein the recombinant Adropin peptide comprises SEQ ID NO.: 1 or SEQ ID NO.: 2. 11 . The method of claim 1 , wherein the Adropin-based therapy is administered intravenously, intraperitoneally, or subcutaneously. 12 . The method of claim 9 , wherein the recombinant Adropin peptide is administered in an amount between about 1 ng/kg to about 200 mg/kg. 13 . The method of claim 1 , wherein the cardiometabolic disease is selected from the group consisting of heart failure with preserved ejection fraction (HFpEF), type-2 diabetes, hypertension, or non-alcoholic fatty liver disease (NAFLD). 14 . A pharmaceutical composition comprising a therapeutically effective amount of an Adropin-based therapy. 15 . The pharmaceutical composition of claim 14 , further comprising a pharmaceutically acceptable carrier. 16 . The pharmaceutical composition of claim 14 , wherein the Adropin-based therapy is a virus, wherein the virus comprises a heterologous nucleic acid encoding an ENHO gene. 17 . The method of claim 16 , wherein the virus is an AAV. 18 . The pharmaceutical composition of claim 16 , wherein the ENHO gene comprises SEQ ID NO.: 3. 19 . The pharmaceutical composition of claim 14 , wherein the Adropin-based therapy is a recombinant Adropin peptide. 20 . The pharmaceutical composition of claim 19 , wherein the recombinant Adropin peptide comprises SEQ ID NO.: 1 or SEQ ID NO.: 2.
viral genome or elements thereof as genetic vector · CPC title
Viral vectors · CPC title
characterised by an aspect of the delivery route, e.g. oral, subcutaneous · CPC title
Hormones (derived from pro-opiomelanocortin, pro-enkephalin or pro-dynorphin A61K38/33, e.g. corticotropin A61K38/35) · CPC title
Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner (non-active ingredients are additionally classified in A61K47/00) · CPC title
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