公开(公告)号：US20200277446A1

公开(公告)日：2020-09-03

申请号：US16621045

申请日：2018-06-11

Applicant: TRANSLATE BIO, INC.

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

IPC: C08G79/04 ,  C08F293/00 ,  A61K47/34 ,  A61K38/53

Abstract: Disclosed are polymers comprising the moiety A, which is a moiety of formula I: and pharmaceutically acceptable salts thereof, wherein R, R1, R2, L, n1 and n2 are as defined herein. These polymers are useful for delivering nucleic acids to subject. These polymers and pharmaceutically acceptable compositions comprising such polymers and nucleic acids can be useful for treating various diseases, disorders and conditions.

公开(公告)号：US20200172923A1

公开(公告)日：2020-06-04

申请号：US16540791

申请日：2019-08-14

Applicant: Translate Bio, Inc.

Inventor： Frank DeRosa ,  Michael Heartlein ,  Daniel Crawford ,  Shrirang Karve

IPC: C12N15/85 ,  C12P21/00 ,  C12N15/67 ,  C07H21/02 ,  A61K31/7125 ,  A61K31/7115 ,  A61K38/18 ,  A61K38/45 ,  A61K38/44 ,  A61K38/17 ,  A61K48/00 ,  C07K14/705 ,  C12N9/02 ,  C12N9/10 ,  C07K14/505

Abstract: The present invention provides, among other things, multimeric coding nucleic acids that exhibit superior stability for in vivo and in vitro use. In some embodiments, a multimeric coding nucleic acid (MCNA) comprises two or more encoding polynucleotides linked via 3′ ends such that the multimeric coding nucleic acid compound comprises two or more 5′ ends.

公开(公告)号：US20200085745A1

公开(公告)日：2020-03-19

申请号：US16553747

申请日：2019-08-28

Applicant: Translate Bio, Inc.

Inventor： Shrirang Karve ,  Zarna Patel ,  Yi Zhang ,  Ashish Sarode ,  Rebecca L. Ball ,  Frank DeRosa ,  Michael Heartlein

IPC: A61K9/127 ,  A61K9/14 ,  A61K9/51 ,  A61K31/7105

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 a solution of pre-formed lipid nanoparticles and mRNA at a low concentration.

公开(公告)号：US20200017440A1

公开(公告)日：2020-01-16

申请号：US16491475

申请日：2018-03-07

Applicant: Translate Bio, Inc.

Inventor： Michael Heartlein ,  Frank DeRosa ,  Shrirang Karve

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

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：US10471153B2

公开(公告)日：2019-11-12

申请号：US15809605

申请日：2017-11-10

Applicant: Translate Bio, Inc.

Inventor： Frank DeRosa ,  Michael Heartlein ,  Shrirang Karve

IPC: A61K47/54 ,  A61K38/17 ,  A61K47/60 ,  A61K47/69 ,  A61K48/00 ,  A61K9/00 ,  A61K9/127 ,  A61P11/12

Abstract: The present invention provides, among other things, compositions and methods of formulating nucleic acid-containing nanoparticles comprising no more than three distinct lipids components, one distinct lipid component being a sterol-based cationic lipid. In some embodiments, the present invention provides compositions and methods in which the lipid nanoparticles further comprise helper lipids and PEG-modified lipids. The resulting formulation comprises a high encapsulation percentage for nucleic acids.

公开(公告)号：US20190192688A1

公开(公告)日：2019-06-27

申请号：US16228596

申请日：2018-12-20

Applicant: Translate Bio, Inc.

Inventor： Kim Askew ,  Jou-Ku Chung ,  Frank DeRosa ,  Michael Heartlein ,  Shrirang Karve ,  Thomas McCauley ,  Lianne Smith ,  Ann J. Barbier

IPC: A61K48/00 ,  C12N15/85 ,  C12N15/52 ,  A61K47/69 ,  A61K9/127 ,  A61K9/00

CPC classification number: A61K48/005 ,  A61K9/0019 ,  A61K9/1272 ,  A61K38/45 ,  A61K47/6929 ,  A61K48/0025 ,  A61K48/0075 ,  A61K48/0083 ,  A61P3/00 ,  C12N15/52 ,  C12N15/85 ,  C12Y201/03003

Abstract: The present invention provides, among other things, methods of treating ornithine transcarbamylase deficiency, including administering to a subject in need of treatment a composition comprising an mRNA encoding an ornithine transcarbamylase protein at a low dose and at an administration interval such that at least one symptom or feature of the OTC deficiency is reduced.

公开(公告)号：US20190185435A1

公开(公告)日：2019-06-20

申请号：US16178142

申请日：2018-11-01

Applicant: TRANSLATE BIO, INC.

Inventor： Frank DeRosa ,  Shrirang Karve ,  Michael Heartlein

IPC: C07D241/08 ,  A61K31/7105 ,  A61K31/495 ,  A61K9/127 ,  A61K47/22 ,  A61K31/7088

CPC classification number: C07D241/08 ,  A61K9/1271 ,  A61K9/1272 ,  A61K31/495 ,  A61K31/7088 ,  A61K31/7105 ,  A61K47/22 ,  A61K2300/00

Abstract: Provided, in part, is a composition comprising one or more chemical entities of formula I, each of which is a compound of formula I: a pharmaceutically acceptable salt thereof, a solvate thereof, or a solvate of a pharmaceutically acceptable salt thereof, the composition characterized in that greater than a first threshold amount of the total amount of chemical entities of formula I in the composition: are chemical entities of formula I.a, wherein the first threshold amount is 50%; or are chemical entities of formula I.b.1, wherein the first threshold amount is 25%; or are chemical entities of formula I.b.2, wherein the first threshold amount is 25%, wherein the chemical entities of formula I.a, I.b.1, and I.b.2, are described herein, and methods of using such compositions, for example, for the delivery of a polynucleotide in vivo.

公开(公告)号：US10155785B2

公开(公告)日：2018-12-18

申请号：US15831252

申请日：2017-12-04

Applicant: Translate Bio, Inc.

Inventor： Frank DeRosa ,  Anusha Dias ,  Shrirang Karve ,  Michael Heartlein

IPC: G01N1/34 ,  C07H1/06 ,  C07H21/00 ,  C12N15/10 ,  C07H21/02

Abstract: The present invention provides, among other things, methods of purifying messenger RNA (mRNA) including the steps of (a) precipitating mRNA from an impure preparation; (b) subjecting the impure preparation comprising precipitated mRNA to a purification process involving membrane filtration such that the precipitated mRNA is captured by a membrane; and (c) eluting the captured precipitated mRNA from the membrane by re-solubilizing the mRNA, thereby resulting in a purified mRNA solution. In some embodiments, a purification process involving membrane filtration suitable for the present invention is tangential flow filtration.

公开(公告)号：US20180127449A1

公开(公告)日：2018-05-10

申请号：US15831252

申请日：2017-12-04

Applicant: Translate Bio, Inc.

Inventor： Frank DeRosa ,  Anusha Dias ,  Shrirang Karve ,  Michael Heartlein

IPC: C07H1/06 ,  C12N15/10 ,  C07H21/02 ,  C07H21/00

CPC classification number: C07H1/06 ,  C07H21/00 ,  C07H21/02 ,  C12N15/1017

Abstract: The present invention provides, among other things, methods of purifying messenger RNA (mRNA) including the steps of (a) precipitating mRNA from an impure preparation; (b) subjecting the impure preparation comprising precipitated mRNA to a purification process involving membrane filtration such that the precipitated mRNA is captured by a membrane; and (c) eluting the captured precipitated mRNA from the membrane by re-solubilizing the mRNA, thereby resulting in a purified mRNA solution. In some embodiments, a purification process involving membrane filtration suitable for the present invention is tangential flow filtration.