公开(公告)号：US12064515B2

公开(公告)日：2024-08-20

申请号：US17737712

申请日：2022-05-05

Applicant: Translate Bio, Inc.

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

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

CPC classification number: A61K9/1277 ,  A61K9/141 ,  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：US12060381B2

公开(公告)日：2024-08-13

申请号：US17811029

申请日：2022-07-06

Applicant: Translate Bio, Inc.

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

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

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.

公开(公告)号：US12053551B2

公开(公告)日：2024-08-06

申请号：US17129666

申请日：2020-12-21

Applicant: Translate Bio, Inc.

Inventor： Shrirang Karve ,  Frank DeRosa ,  Ashish Sarode ,  Natalia Vargas Montoya ,  Priyal Patel

IPC: A61K9/51 ,  A61K31/7105 ,  B82Y5/00 ,  B82Y40/00

CPC classification number: A61K9/5192 ,  A61K31/7105 ,  B82Y5/00 ,  B82Y40/00

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 combining a solution of preformed lipid nanoparticles with lipid nanoparticles encapsulating mRNA, resulting in a composition of increased potency of mRNA encoded protein expression in vivo.

公开(公告)号：US12016954B2

公开(公告)日：2024-06-25

申请号：US17002460

申请日：2020-08-25

Applicant: Translate Bio, Inc.

Inventor： Frank DeRosa ,  Michael Heartlein ,  Shrirang Karve

IPC: C07H21/02 ,  A61K9/127 ,  A61K38/17 ,  A61K48/00 ,  C12N15/88 ,  A61P25/00

CPC classification number: A61K9/1272 ,  A61K38/1709 ,  A61K48/0033 ,  C12N15/88 ,  A61K48/005 ,  A61P25/00 ,  C07H21/02

Abstract: The present invention provides, among other things, methods and compositions for effective delivery of messenger RNA (mRNA) to the central system (CNS). In particular, the present invention provides methods and compositions for administering intrathecally to a subject in need of delivery a composition comprising an mRNA encoding a protein, encapsulated within a liposome, such that the administering of the composition results in the intracellular delivery of mRNA in neurons in the brain and/or spinal cord. The present invention is particularly useful for the treatment of CNS diseases, disorders or conditions, such as spinal muscular atrophy.

公开(公告)号：US11976272B2

公开(公告)日：2024-05-07

申请号：US17070877

申请日：2020-10-14

Applicant: Translate Bio, Inc.

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

IPC: C12N15/10 ,  B01L3/00 ,  C12Q1/6806

CPC classification number: C12N15/1017 ,  B01L3/502753 ,  C12Q1/6806 ,  B01L2200/028 ,  B01L2200/16 ,  B01L2300/0681 ,  B01L2400/0409 ,  C12Q2523/113 ,  C12Q2523/308 ,  C12Y201/03003

Abstract: The present invention relates, in part, to methods for large-scale purification of mRNA. The method includes, at least, steps of forming an mRNA slurry, stirring the slurry, and vacuum or pressure filtering the slurry.

公开(公告)号：US20230181483A1

公开(公告)日：2023-06-15

申请号：US17625507

申请日：2020-07-08

Applicant: Translate Bio, Inc.

Inventor： Shrirang Karve ,  Ashish Sarode ,  Frank DeRosa

IPC: A61K9/51 ,  A61K47/54 ,  A61K47/69 ,  A61K9/00 ,  A61K9/127

CPC classification number: A61K9/5192 ,  A61K47/544 ,  A61K47/6929 ,  A61K9/0019 ,  A61K9/1272 ,  A61K9/5123 ,  A61K38/00

Abstract: The present invention provides an improved lipid nanoparticle formulation encapsulating mRNA comprising DEPE as a helper lipid.

公开(公告)号：US20230071228A1

公开(公告)日：2023-03-09

申请号：US17291937

申请日：2019-11-07

Applicant: Translate Bio, Inc.

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

IPC: C07D241/04 ,  A61K47/24 ,  A61K31/7105 ,  A61K9/127

Abstract: The present invention provides, in part, cyclic amino acid 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：US20230051811A1

公开(公告)日：2023-02-16

申请号：US17786971

申请日：2020-12-18

Applicant: Translate Bio, Inc

Inventor： Shrirang Karve ,  Frank DeRosa ,  Ashish Sarode

IPC: A61K9/00 ,  A61K48/00 ,  A61K9/127 ,  A61K9/02 ,  A61K9/51

Abstract: The present invention provides, among other things, effective methods and compositions for delivering messenger RNA (mRNA) via rectal delivery. The present invention is, in part, based on unexpected observation that mRNA may be effectively delivered to the circulation, liver, kidney, colon and/or rectum via rectal delivery despite the barriers such as RNase and mucus layer.

公开(公告)号：US20220402959A1

公开(公告)日：2022-12-22

申请号：US17811029

申请日：2022-07-06

Applicant: Translate Bio, Inc.

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

IPC: 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：US11530298B2

公开(公告)日：2022-12-20

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 ,  A61K38/53 ,  A61K47/34 ,  C08F293/00 ,  A61K31/7088

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.