US 2026/0139251 A1 · RNAi Agents for Inhibiting Expression of Mucin 5AC (MUC5AC), Compositions Thereof, and Methods of Use

Abstract

Described are RNAi agents, compositions that include RNAi agents, and methods for inhibition of a Mucin 5AC (MUC5AC) gene. The MUC5AC RNAi agents and RNAi agent conjugates disclosed herein inhibit the expression of an MUC5AC gene. Pharmaceutical compositions that include one or more MUC5AC RNAi agents, optionally with one or more additional therapeutics, are also described. Delivery of the described MUC5AC RNAi agents to pulmonary epithelial cells, in vivo, provides for inhibition of MUC5AC gene expression and a reduction in MUC5AC production, which can provide a therapeutic benefit to subjects, including human subjects, for the treatment of various diseases including mucoobstructive lung disease such as severe asthma and various cancers.

Claims34

Dependent claims

An RNAi agent for inhibiting expression of a Mucin 5AC gene, comprising:

an antisense strand comprising at least 17 contiguous nucleotides differing by 0 or 1 nucleotides from any one of the sequences provided in Table 2 or Table 3;
and
a sense strand comprising a nucleotide sequence that is at least partially complementary to the antisense strand.

Dependencies are inferred from the claim text.

RNAi Agents for Inhibiting Expression of Mucin 5AC (MUC5AC), Compositions Thereof, and Methods of Use

Life of this patent

RNAi Agents for Inhibiting Expression of Mucin 5AC (MUC5AC), Compositions Thereof, and Methods of Use

Life of this patent

Abstract

Claims34