公开(公告)号：US20220160633A1

公开(公告)日：2022-05-26

申请号：US17522754

申请日：2021-11-09

Applicant: Translate Bio, Inc.

Inventor： John Androsavich ,  Lianne Boeglin ,  Shraddha Sharma ,  Gang Sun ,  Neha Kaushal ,  Shrirang Karve

IPC: A61K9/127 ,  A61K31/7105 ,  A61K9/00

Abstract: The present invention provides, among other things, improved pharmaceutical compositions comprising codon-optimized mRNA encoding a peptide or polypeptide encapsulated in a lipid nanoparticle comprising one or more of the cationic lipids that are particularly effective for pulmonary delivery.

公开(公告)号：US20240197825A1

公开(公告)日：2024-06-20

申请号：US17761893

申请日：2020-09-17

Applicant: Translate Bio, Inc.

Inventor： Richard Wooster ,  Frank DeRosa ,  Lianne Boeglin ,  Priyaanka Nanduri ,  Anusha Dias ,  Khang Anh Tran

IPC: A61K38/17 ,  A61K48/00

CPC classification number: A61K38/177 ,  A61K48/0033 ,  A61K48/005

Abstract: The present invention provides, among other things, improved methods and pharmaceutical compositions for treating cystic fibrosis based on codon optimized mRNA encoding an engineered or mutant Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein.

公开(公告)号：US20230145188A1

公开(公告)日：2023-05-11

申请号：US17275803

申请日：2019-09-13

Applicant: Translate Bio, Inc.

Inventor： Lianne Boeglin ,  Christian Cobaugh ,  Frank DeRosa ,  Michael Heartlein ,  Kim Askew

IPC: A61K48/00 ,  A61K9/127 ,  C12N15/88 ,  C12N15/52 ,  A61P3/00

CPC classification number: A61K48/005 ,  A61K9/1271 ,  C12N15/88 ,  C12N15/52 ,  A61P3/00 ,  C12Y504/99002

Abstract: The present invention provides, among other things, methods and compositions for treating methylmalonic academia (MMA) based on mRNA therapy. The compositions used in treatment of MMA comprise an mRNA comprising a methymalonyl-CoA mutase (MUT) coding sequence and are administered at an effective dose and an administration interval such that at least one symptom or feature of MMA is reduced in intensity, severity, or frequency or has a delayed onset. mRNAs with optimized MUT coding sequences are provided that can be administered without the need for modifying the nucleotides of the mRNA to achieve sustained in vivo function.

公开(公告)号：US20220218803A1

公开(公告)日：2022-07-14

申请号：US17543543

申请日：2021-12-06

Applicant: Translate Bio, Inc.

Inventor： Michael Heartlein ,  Frank DeRosa ,  Lianne Boeglin

IPC: A61K38/53 ,  C12N9/00 ,  A61K9/127 ,  A61K9/51 ,  A61K31/7105

Abstract: The present invention provides, among other things, methods of treating Argininosuccinate Synthetase Deficiency (ASD), including administering to a subject in need of treatment a composition comprising an mRNA encoding argininosuccinate synthetase (ASS1) at an effective dose and an administration interval such that at least one symptom or feature of ASD is reduced in intensity, severity, or frequency or has delayed in onset. In some embodiments, the mRNA is encapsulated in a liposome comprising one or more cationic lipids, one or more non-cationic lipids, one or more cholesterol-based lipids and one or more PEG-modified lipids