公开(公告)号：US12151029B2

公开(公告)日：2024-11-26

申请号：US17277452

申请日：2019-09-19

Applicant: ModernaTX, Inc.

Inventor： Edward J. Hennessy ,  Kerry Benenato

IPC: A61K9/51 ,  A61K8/86 ,  A61K31/7105 ,  A61K47/14 ,  A61K47/18 ,  A61K47/22 ,  A61K47/24 ,  A61K47/28 ,  C07C69/708 ,  C07C223/02 ,  C07C321/14 ,  C07D317/32

Abstract: The present disclosure in part provides compounds (i.e., PEG lipids) which are useful in pharmaceutical compositions, cosmetic compositions, and drug delivery systems, e.g, for use in lipid nanoparticle (LNP) formulations. The present disclosure also provides LNP formulations comprising PEG lipids described herein, and methods of using the same. For example, the LNPs provided herein are useful for the delivery of an agent (e.g, therapeutic agent) to a subject. The PEG lipids and LNPs provided herein, in certain embodiments, exhibit increased PEG shedding compared to existing PEG lipids and LNP formulations.

公开(公告)号：US20250152518A1

公开(公告)日：2025-05-15

申请号：US18919839

申请日：2024-10-18

Applicant: ModernaTX, Inc.

Inventor： Edward J. Hennessy ,  Kerry Benenato

IPC: A61K9/51 ,  A61K8/86 ,  A61K31/7105 ,  A61K47/14 ,  A61K47/18 ,  A61K47/22 ,  A61K47/24 ,  A61K47/28 ,  C07C69/708 ,  C07C223/02 ,  C07C321/14 ,  C07D317/32

Abstract: The present disclosure in part provides compounds (i.e., PEG lipids) which are useful in pharmaceutical compositions, cosmetic compositions, and drug delivery systems, e.g. for use in lipid nanoparticle (LNP) formulations. The present disclosure also provides LNP formulations comprising PEG lipids described herein, and methods of using the same. For example, the LNPs provided herein are useful for the delivery of an agent (e.g., therapeutic agent) to a subject. The PEG lipids and LNPs provided herein, in certain embodiments, exhibit increased PEG shedding compared to existing PEG lipids and LNP formulations.

公开(公告)号：US20220370616A1

公开(公告)日：2022-11-24

申请号：US17726971

申请日：2022-04-22

Applicant: ModernaTX, Inc.

Inventor： Edward J. Hennessy

IPC: A61K47/22 ,  A61K31/711 ,  A61K31/7105 ,  A61K9/127 ,  A61K9/00 ,  A61J1/14

Abstract: Stabilized formulations of nucleic acids, including lipid nanoparticle formulations which encapsulate nucleic acids, are provided. Methods of making and of use of the formulations stabilized by chemical compounds are also provided.

公开(公告)号：US20230233475A1

公开(公告)日：2023-07-27

申请号：US17928684

申请日：2021-06-01

Applicant: ModernaTX, Inc.

Inventor： Kerry Benenato ,  Kristine Burke ,  Jingsong Cao ,  Paloma Hoban Giangrande ,  Edward J. Hennessy ,  Stephen Hoge ,  Jaclyn Milton ,  Staci Sabnis ,  Timothy Salerno ,  Matthew Theisen

IPC: A61K9/51 ,  A61K31/7088 ,  A61K9/00 ,  C12N9/16 ,  A61P35/00

CPC classification number: A61K9/5123 ,  A61K31/7088 ,  A61K9/0019 ,  C12Y301/03009 ,  C12N9/16 ,  A61P35/00

Abstract: This disclosure relates to ionizable lipid-based lipid nanoparticles for delivery of mRNA encoding glucose-6-phosphatase. Lipid nanoparticle/mRNA therapies of the invention increase and/or restore deficient levels of glucose-6-phosphatase expression and activity in subjects and are useful for the treatment of glycogen storage disease type 1a (GSD-Ia). Lipid nanoparticle/mRNA therapies of the invention increase glucose production and reduce the abnormal accumulation of glycogen and glucose-6-phosphate associated with GSD-Ia.