公开(公告)号：US20250064938A1

公开(公告)日：2025-02-27

申请号：US18725289

申请日：2023-01-03

Applicant: Massachusetts Institute of Technology

Inventor： Daniel Griffith Anderson ,  Bowen Li ,  Akiva Gordon ,  Idris Raji

IPC: A61K47/54 ,  A61K9/127 ,  A61K31/7105 ,  A61K48/00 ,  C07C233/47 ,  C07D211/14 ,  C07D231/12 ,  C07D233/61 ,  C07D243/08 ,  C07D295/13 ,  C12N15/113

Abstract: Provided herein are lipid compounds of Formulae (I) and (II), and pharmaceutically acceptable salts, co-crystals, tautomers, stereoisomers, solvates, hydrates, polymorphs, isotopically labeled derivatives, prodrugs, and compositions thereof. Also provided are methods and kits involving the inventive lipid compounds, compositions, or formulations for treating and/or preventing diseases (e.g., genetic disease, proliferative disease, hematological disease, neurological disease, painful condition, psychiatric disorder, metabolic disorder, long-term medical condition, inflammatory disease, autoinflammatory disease, liver disease, lung disease, spleen disease, familial amyloid neuropathy, cardiovascular disease, viral infection, infectious disease, fibrotic condition, or autoimmune disease) in a subject, methods for synthesizing the compounds described herein, and compounds described herein synthesized by the synthetic methods described herein. The compounds are effective carriers for the delivery of an agent such as a polynucleotide (e.g., RNA) to a tissue or cell in a subject (e.g., a liver, lung, or spleen tissue/cell).

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).