US20250387490
2025-12-25
Human necessities
A61K47/543
The patent describes novel lipid compounds of Formulae (I) and (II), along with their pharmaceutically acceptable variants. These lipids are designed to serve as carriers for the delivery of therapeutic agents such as polynucleotides, including RNA, to specific tissues or cells within a subject. The compounds can be utilized in treating a wide range of diseases, including genetic, proliferative, and infectious diseases, among others. The invention also includes methods for synthesizing these compounds and using them in pharmaceutical compositions.
Nucleic acid-based therapeutics, particularly mRNA, hold significant potential for disease treatment and vaccine development. However, their clinical application is limited by challenges in delivering them to specific organs and tissues due to their instability and susceptibility to degradation. Lipid nanoparticles (LNPs) have emerged as effective delivery systems, with ionizable lipids playing a crucial role in enabling organ-selective delivery. Despite existing LNPs used in approved drugs and vaccines, there is a need for more efficient and biodegradable ionizable lipids to reduce toxicity and improve therapeutic outcomes.
The patent highlights the development of a high-throughput synthesis method for over 600 ionizable lipids, which were screened for their ability to deliver mRNA to specific organs in mice. The identified biodegradable lipids effectively target the liver, lungs, and spleen, minimizing toxicity risks. The invention addresses the limitations of traditional lipid design by incorporating machine learning algorithms to streamline synthesis and screening processes, overcoming the challenges of extensive lipid library development.
The lipid compounds can be formulated into pharmaceutical compositions, potentially as nanoparticles or microparticles, to deliver therapeutic agents. These formulations are applicable in treating various diseases, including liver, lung, and spleen diseases, as well as autoimmune and inflammatory conditions. The compositions may also serve as adjuvants in mRNA vaccine systems, enhancing immune responses and delivering a range of antigens, including those for cancer and infectious diseases.
The patent also describes kits containing the lipid compounds or compositions, which can include single or multiple doses, along with instructions for use. These kits are intended for treating or preventing diseases by delivering therapeutic agents to target cells, tissues, or organs. The methods outlined in the patent involve administering therapeutically or prophylactically effective amounts of the compositions to subjects in need, offering a versatile approach to disease management and prevention.