Patent classifications
C12Y207/01003
KETOHEXOKINASE (KHK) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
The present invention relates to RNAi agents, e.g., dsRNA agents, targeting the ketohexokinase (KHK) gene. The invention also relates to methods of using such RNAi agents to inhibit expression of a KHK gene and to methods of treating or preventing a KHK-associated disease in a subject.
KETOHEXOKINASE (KHK) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
The present invention relates to RNAi agents, e.g., dsRNA agents, targeting the ketohexokinase (KHK) gene. The invention also relates to methods of using such RNAi agents to inhibit expression of a KHK gene and to methods of treating or preventing a KHK-associated disease in a subject.
METHODS FOR THE TREATMENT OF NASH WITH ADVANCED FIBROSIS AND/OR CIRRHOSIS
The present invention provides methods for the treatment of non-alcoholic steatohepatitis (NASH) including advanced fibrotic and/or cirrhotic stages of NASH. The methods include administration of a compound comprising a double stranded RNAi (dsRNAi) oligonucleotide, or a pharmaceutically acceptable salt thereof, to the patient. The dsRNAi oligonucleotide optionally contains an antisense strand and a sense strand, wherein the antisense strand and the sense strand form a duplex region, and wherein the antisense strand comprises a region of complementarity to a KHK mRNA target sequence.
ENGINEERED IMMUNE CELL
An immune cell that is genetically engineered to express an exogenous alternative carbon source (ACS) metabolism gene, in which the ACS is not glucose and wherein the ability of the immune cell to metabolise the ACS is increased due to expression of the exogenous ACS metabolism gene. Also provided are polynucleotides, vectors, pharmaceutical compositions, methods of genetically engineering the immune cell and methods of use in therapy.
KETOHEXOKINASE (KHK) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
The present invention relates to RNAi agents, e.g., dsRNA agents, targeting the ketohexokinase (KHK) gene. The invention also relates to methods of using such RNAi agents to inhibit expression of a KHK gene and to methods of treating or preventing a KHK-associated disease in a subject.