公开(公告)号：US12178855B2

公开(公告)日：2024-12-31

申请号：US16961187

申请日：2019-01-10

Applicant: Translate Bio MA, Inc.

Inventor： Balkrishen Bhat ,  Jia Tay ,  Saswata Karmakar ,  Nelson Chau

IPC: C07H21/04 ,  A61K38/45 ,  A61K47/54 ,  C12N15/113

Abstract: Provided herein are compositions and methods for facilitating or enhancing delivery of nucleic acids, such as synthetic mRNAs, into cells or tissues. Such compositions and methods may include use of a targeting moiety-conjugated, such as an N-acetylgalactosamine (GalNAc)-conjugated, oligonucleotide to facilitate or enhance delivery.

公开(公告)号：US20190224326A1

公开(公告)日：2019-07-25

申请号：US16317229

申请日：2017-07-11

Applicant: Translate Bio MA, Inc.

Inventor： Balkrishen Bhat ,  Saswata Karmakar ,  Debatosh Majumdar ,  Jia Tay ,  Nelson Chau

IPC: A61K47/54 ,  C12N15/113 ,  A61K31/7088

Abstract: Provided herein are conjugates comprising targeting moieties such as sugars, folates and cell-penetrating peptides, which can be used for the improved delivery of agents (e.g., nucleic acids, such as oligonucleotides or mRNAs, or other agents) to cells. The invention provides conjugates and compounds comprising targeting moieties, methods for preparing the same, and intermediates useful in their preparation. In another aspect, the present invention provides formulations (e.g., pharmaceutical compositions) comprising the targeting moiety-containing conjugates and compounds. The present invention also provides methods for delivering agents (e.g., nucleic acids such as oligonucleotides or mRNAs) to a cell, methods for treating and/or preventing a disease or condition in a subject, and methods for modulating gene expression in a cell or a subject. Further, provided herein are kits comprising the conjugates, or formulations thereof; and kits for the preparation of conjugates described herein.

公开(公告)号：US20190062743A1

公开(公告)日：2019-02-28

申请号：US16118297

申请日：2018-08-30

Applicant: Translate Bio MA, Inc.

Inventor： Eugen Uhlmann ,  Markus Weber ,  Romesh R. Subramanian ,  Thomas Dino Rockel ,  Arthur M. Krieg

IPC: C12N15/113

Abstract: The disclosure provides multimeric oligonucleotide compounds, comprising two or more target-specific oligonucleotides (e.g., antisense oligonucleotides (ASOs)), each being resistant to cleavage, and linked together by a cleavable linker. In particular, two or more linked target-specific oligonucleotides, each to a different target, allows concomitant inhibition of multiple genes' expression levels, while exhibiting favorable pharmacokinetic and pharmacodynamic properties. Methods of making and uses of the described compounds are also provided

公开(公告)号：US10059941B2

公开(公告)日：2018-08-28

申请号：US14401194

申请日：2013-05-16

Applicant: Translate Bio MA, Inc. ,  The General Hospital Corporation d/b/a Massachusetts General Hospital

Inventor： Arthur M. Krieg ,  Romesh Subramanian ,  James McSwiggen ,  Jeannie T. Lee

IPC: C12N15/113 ,  A61K31/7125 ,  A61K31/7115 ,  A61K31/712 ,  A61K31/7088 ,  C07H21/00

CPC classification number: C12N15/113 ,  A61K31/7088 ,  C07H21/00 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/3231 ,  C12N2310/343 ,  C12N2310/3517 ,  C12N2310/321 ,  C12N2310/3521 ,  C12N2310/322 ,  C12N2310/3533

Abstract: Aspects of the invention provide single stranded oligonucleotides for activating or enhancing expression of SMN1 or SMN2. Further aspects provide compositions and kits comprising single stranded oligonucleotides for activating or enhancing expression of SMN1 or SMN2 that comprises exon 7. Methods for modulating expression of SMN1 or SMN2 using the single stranded oligonucleotides are also provided. Further aspects of the invention provide methods for selecting a candidate oligonucleotide for activating or enhancing expression of SMN1 or SMN2.

公开(公告)号：US10041074B2

公开(公告)日：2018-08-07

申请号：US14700269

申请日：2015-04-30

Applicant: Translate Bio MA, Inc.

Inventor： Fatih Ozsolak

IPC: C07H21/04 ,  C12N15/113

Abstract: Provided herein are oligonucleotides complementary to euchromatic regions of target genes that are useful for increasing expression of the target genes; related compositions and methods are also provided.

公开(公告)号：US20180055869A1

公开(公告)日：2018-03-01

申请号：US15550112

申请日：2016-02-12

Applicant: Translate Bio MA, Inc.

Inventor： Fatih Ozsolak

IPC: A61K31/7088 ,  C12N15/113

CPC classification number: A61K31/7088 ,  A61K31/7105 ,  C12N15/113 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/317 ,  C12N2310/321 ,  C12N2310/3231 ,  C12N2310/3521

Abstract: Aspects of the invention relate to methods for increasing gene expression in a targeted manner. In some embodiments, methods are provided for increasing expression of a gene expressed in a liver cell. In some embodiments, methods and compositions are provided that are useful for posttranscriptionally altering protein and/or RNA levels in a targeted manner. Aspects of the invention disclosed herein provide methods and compositions that are useful for protecting RNAs from degradation (e.g., exonuclease mediated degradation).

公开(公告)号：US09732341B2

公开(公告)日：2017-08-15

申请号：US14698312

申请日：2015-04-28

Applicant: Translate Bio MA, Inc.

Inventor： Eugen Uhlmann ,  Markus Weber ,  Romesh Subramanian ,  Thomas Dino Rockel ,  Arthur M. Krieg

IPC: C12N15/113 ,  A61K48/00

CPC classification number: C12N15/113 ,  C12N2310/11 ,  C12N2310/113 ,  C12N2310/315 ,  C12N2310/3231 ,  C12N2310/341 ,  C12N2310/346 ,  C12N2310/51 ,  C12N2310/52

Abstract: The disclosure provides multimeric oligonucleotide compounds, comprising two or more target-specific oligonucleotides (e.g., antisense oligonucleotides (ASOs)), each being resistant to cleavage, and linked together by a cleavable linker. In particular, two or more linked target-specific oligonucleotides, each to a different target, allows concomitant inhibition of multiple genes' expression levels, while exhibiting favorable pharmacokinetic and pharmacodynamic properties. Methods of making and uses of the described compounds are also provided.

公开(公告)号：US20230062603A1

公开(公告)日：2023-03-02

申请号：US17791190

申请日：2021-01-06

Applicant: Translate Bio MA, Inc.

Inventor： Balkrishen Bhat ,  Brian Bettencourt ,  Saswata Karmakar ,  Caroline Woo ,  John Androsavich ,  Shrirang Karve

IPC: A61K31/7088 ,  A61K9/00 ,  A61K9/127 ,  C12N15/113 ,  C12N15/88

Abstract: The present disclosure provides a method of pulmonarily administering an oligonucleotide to lung cells. In some embodiments, the disclosure provides formulations that comprise an oligonucleotide that is complementary to a target gene in lung cells and a lipid nanoparticle. Methods of the present disclosure are useful in modifying expression of target genes associated with chronic obstructive pulmonary disease, asthma, and pulmonary fibrosis.

公开(公告)号：US20210052706A1

公开(公告)日：2021-02-25

申请号：US16961187

申请日：2019-01-10

Applicant: Translate Bio MA, Inc.

Inventor： Balkrishen Bhat ,  Jia Tay ,  Saswata Karmakar ,  Nelson Chau

IPC: A61K38/45 ,  A61K47/54 ,  C12N15/113

Abstract: Provided herein are compositions and methods for facilitating or enhancing delivery of nucleic acids, such as synthetic mRNAs, into cells or tissues. Such compositions and methods may include use of a targeting moiety-conjugated, such as an N-acetylgalactosamine (GalNAc)-conjugated, oligonucleotide to facilitate or enhance delivery.

公开(公告)号：US20190055553A1

公开(公告)日：2019-02-21

申请号：US15768579

申请日：2016-10-14

Applicant: TRANSLATE BIO MA, INC.

Inventor： James BARSOUM ,  Caroline WOO

IPC: C12N15/113

Abstract: Aspects of the disclosure provide steric-blocking oligonucleotide-based methods of modulating expression of target genes, e.g., by targeting non-coding RNA scaffolds.