公开(公告)号：US20250049714A1

公开(公告)日：2025-02-13

申请号：US18717095

申请日：2022-12-08

Applicant: Massachusetts Institute of Technology

Inventor： Daniel Griffith Anderson ,  Luke Hyunsik Rhym ,  Bowen Li ,  Akiva Gordon ,  Manan Rajith Singh ,  Jacob Witten

IPC: A61K9/127 ,  A61K9/00 ,  A61K9/16 ,  A61K9/51 ,  A61K31/7105 ,  A61K31/711 ,  C07C229/12 ,  C07C229/16 ,  C07D207/06 ,  C07D213/38 ,  C07D255/02 ,  C07D295/13 ,  C12N15/88

Abstract: Provided herein are compounds, such as compounds of Formula (I), and pharmaceutically acceptable salts, hydrates, solvates, polymorphs, co-crystals, tautomers, stereoisomers, and isotopically labeled derivatives thereof, and compositions, methods, uses, and kits thereof. The compounds provided herein are lipids useful for delivery of polynucleotides, such as mRNA, for the treatment and/or prevention of various diseases and conditions (e.g., genetic disease, proliferative disease, hematological disease, neurological disease, liver disease, spleen disease, lung disease, painful condition, psychiatric disorder, musculoskeletal disease, a metabolic disorder, inflammatory disease, or autoimmune disease).