公开(公告)号：US20220323355A1

公开(公告)日：2022-10-13

申请号：US17054886

申请日：2019-05-15

Applicant: Translate Bio, Inc.

Inventor： Frank DeRosa ,  Shrirang Karve ,  Yi Zhang ,  Michael Heartlein

IPC: A61K9/127 ,  C07H13/06 ,  A61K9/00 ,  A61K38/18

Abstract: Disclosed are cationic lipids which are compounds of Formula (I′). Cationic lipids provided herein can be useful for delivery and expression of mRNA and encoded protein, e.g., as a component of liposomal delivery vehicle, and accordingly can be useful for treating various diseases, disorders and conditions, such as those associated with deficiency of one or more proteins.

公开(公告)号：US11427535B2

公开(公告)日：2022-08-30

申请号：US17172640

申请日：2021-02-10

Applicant: Translate Bio, Inc.

Inventor： Michael Heartlein ,  Frank DeRosa ,  Shrirang Karve

IPC: C07C305/06 ,  C07C317/18 ,  A61K9/127 ,  A61K47/20 ,  A61K47/54 ,  A61K47/58 ,  A61K31/7105

Abstract: Disclosed are dendritic anionic lipids which are compounds of Formula (I): wherein R and R1 are non-polar groups, L is a linking moiety, and Dm is a dendritic moiety of m generations, each as defined herein. These dendritic anionic lipids are useful for delivery and expression of m RNA and encoded protein, e.g., as a component of liposomal delivery vehicle, and accordingly can be useful for treating various diseases, disorders and conditions, such as those associated with deficiency of one or more proteins.

公开(公告)号：US20220257724A1

公开(公告)日：2022-08-18

申请号：US17608347

申请日：2020-05-01

Applicant: TRANSLATE BIO, INC.

Inventor： Shrirang Karve ,  Frank DeRosa

IPC: A61K38/45 ,  A61K9/127 ,  A61K38/17 ,  C07D295/15 ,  C07C327/30 ,  C07C329/16

Abstract: The present invention provides, in part, di-thioester cationic lipid compounds or a pharmaceutically acceptable salt thereof. The compounds provided herein can be useful for delivery and expression of mRNA and encoded protein, e.g., as a component of liposomal delivery vehicle, and accordingly can be useful for treating various diseases, disorders and conditions, such as those associated with deficiency of one or more proteins.

公开(公告)号：US20220218612A1

公开(公告)日：2022-07-14

申请号：US17611020

申请日：2020-05-14

Applicant: Translate Bio, Inc.

Inventor： Shrirang Karve ,  Frank DeRosa ,  Michael Hearlein ,  Ashish Sarode ,  Zarna Patel ,  Rebecca L. Ball ,  Natalia Vargas Montoya ,  Priyal Patel ,  Asad Khanmohammed

IPC: A61K9/127 ,  A61K9/00 ,  A61K31/7088 ,  A61K9/16

Abstract: The present invention provides an improved process for lipid nanoparticle formulation and mRNA encapsulation. In some embodiments, the present invention provides a process for enhanced encapsulation of messenger RNA (mRNA) in lipid nanoparticles comprising a step of heating the mRNA-encapsulated lipid nanoparticles in a drug product formulation solution.

公开(公告)号：US20220177423A1

公开(公告)日：2022-06-09

申请号：US17604244

申请日：2020-04-17

Applicant: Translate Bio, Inc.

Inventor： Shrirang Karve ,  Saswata Karmakar ,  Frank DeRosa

IPC: C07C323/58 ,  A61K9/127 ,  A61K38/17 ,  A61K38/45

Abstract: The present invention provides, in part cystine cationic lipid compounds of formula A, and sub-formulas thereof: or a pharmaceutically acceptable salt thereof. The compounds provided herein can be useful for delivery and expression of mRNA and encoded protein, e.g., as a component of liposomal delivery vehicle, and accordingly can be useful for treating various diseases, disorders and conditions, such as those associated with deficiency of one or more proteins.

公开(公告)号：US20220110884A1

公开(公告)日：2022-04-14

申请号：US17450629

申请日：2021-10-12

Applicant: Translate Bio, Inc.

Inventor： Shrirang Karve ,  Travis Jeanotte ,  Monic Shah ,  Yi Zhang ,  Kimberly Gillis ,  Frank DeRosa

IPC: A61K9/51 ,  A61K9/127

Abstract: The present invention provides an improved process for lipid nanoparticle formulation and mRNA encapsulation. In some embodiments, the present invention provides a process of encapsulating messenger RNA (mRNA) in lipid nanoparticles comprising a step of mixing an mRNA solution containing low citrate concentration and a lipid solution at an ambient temperature. Thus, the present invention provides an effective, reliable, energy-saving and cost-effective method of encapsulating mRNA into lipid nanoparticles.

公开(公告)号：US20220087935A1

公开(公告)日：2022-03-24

申请号：US17420346

申请日：2020-01-07

Applicant: Translate Bio, Inc.

Inventor： Anusha Dias ,  Darshan Parekh ,  Jeffrey S. Dubins ,  Christian Cobaugh ,  Shrirang Karve ,  Zarna Patel ,  Sara J. Dunaj ,  Frank DeRosa ,  Michael Heartlein

IPC: A61K9/127 ,  A61K31/711 ,  A61P11/00

Abstract: The present invention provides, among other things, methods and compositions for treating primary ciliary dyskinesia (PCD) based on mRNA therapy. The compositions used in treatment of PCD comprise an mRNA comprising a dynein axonemal heavy chain 5 (DNAH5) coding sequence and are administered at an effective dose and an administration interval such that at least one symptom or feature of PCD is reduced in intensity, severity, or frequency or has a delayed onset. mRNAs with optimized DNAH5 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：US20210206739A1

公开(公告)日：2021-07-08

申请号：US17058574

申请日：2019-05-29

Applicant: Translate Bio, Inc.

Inventor： Yi Zhang ,  Shrirang Karve ,  Saswata Karmakar ,  Frank DeRosa ,  Michael Heartlein

IPC: C07D311/20 ,  C07C225/14 ,  C07C229/12 ,  C07C323/25 ,  C07C323/27 ,  C07C403/12

Abstract: Disclosed are cationic lipids comprising a vitamin substructure and an ionizable nitrogen-containing group. Cationic lipids provided herein can be useful for delivery and expression of mRNA and encoded protein, e.g., as a component of liposomal delivery vehicle, and accordingly can be useful for treating various diseases, disorders and conditions, such as those associated with deficiency of one or more proteins.

公开(公告)号：US10975369B2

公开(公告)日：2021-04-13

申请号：US15907086

申请日：2018-02-27

Applicant: Translate Bio, Inc.

Inventor： Frank DeRosa ,  Michael Heartlein ,  Jonathan Abysalh ,  Daniel Crawford ,  Anusha Dias ,  Shrirang Karve

IPC: C12Q1/68 ,  C07H21/00 ,  C12N15/10 ,  C07H21/04

Abstract: The present invention relates, in part, to methods for large-scale purification of mRNA. The method includes, at least, a step of centrifuging an mRNA suspension in a centrifuge comprising a porous substrate at a speed sufficient to remove process contaminants and to precipitate purified mRNA composition onto the porous substrate.

公开(公告)号：US10961184B2

公开(公告)日：2021-03-30

申请号：US16491475

申请日：2018-03-07

Applicant: Translate Bio, Inc.

Inventor： Michael Heartlein ,  Frank DeRosa ,  Shrirang Karve

IPC: C07C305/06 ,  C07C317/18 ,  A61K9/127 ,  A61K47/20 ,  A61K47/54 ,  A61K47/58

Abstract: Disclosed are dendritic anionic lipids which are compounds of Formula (I): wherein R and R1 are non-polar groups, L is a linking moiety, and Dm is a dendritic moiety of m generations, each as defined herein. These dendritic anionic lipids are useful for delivery and expression of m RNA and encoded protein, e.g., as a component of liposomal delivery vehicle, and accordingly can be useful for treating various diseases, disorders and conditions, such as those associated with deficiency of one or more proteins.