Patents Assigned to Translate Bio, Inc.
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Patent number: 10912844Abstract: The present invention provides, in part, a biodegradable compound of formula I, and sub-formulas thereof:Formula (I) or a pharmaceutically acceptable salt thereof, where each X independently is O or S, each Y independently is O or S, and each R1 independently is defined herein; and a liposome composition comprising the cationic lipid of formula I or a sub-formula thereof, and methods of delivering agents, such as nucleic acids including mRNA, in vivo, by administering to a subject the liposome comprising the cationic lipid of formula I or a sub-formula thereof, where the agent is encapsulated within the liposome.Type: GrantFiled: November 22, 2019Date of Patent: February 9, 2021Assignee: Translate Bio, Inc.Inventors: Frank DeRosa, Michael Heartlein
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Patent number: 10899830Abstract: The present invention provides, among other things, methods and compositions for delivering an antibody in vivo by administering to a subject in need thereof one or more mRNAs encoding a heavy chain and a light chain of an antibody, and wherein the antibody is expressed systemically in the subject. In some embodiments, the one or more mRNAs comprise a first mRNA encoding the heavy chain and a second mRNA encoding the light chain of the antibody.Type: GrantFiled: January 24, 2020Date of Patent: January 26, 2021Assignee: Translate Bio, Inc.Inventors: Michael Heartlein, Frank DeRosa, Anusha Dias, Braydon Charles Guild
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Patent number: 10888626Abstract: Disclosed herein are compositions and methods for modulating the production of a protein in a target cell. The compositions and methods disclosed herein are capable of ameliorating diseases associated with protein or enzyme deficiencies.Type: GrantFiled: July 16, 2020Date of Patent: January 12, 2021Assignee: Translate Bio, Inc.Inventors: Braydon Charles Guild, Frank DeRosa, Michael Heartlein
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Patent number: 10876104Abstract: The present invention provides, among other things, methods of purifying messenger RNA (mRNA) including the steps of subjecting an impure preparation comprising in vitro synthesized mRNA to a denaturing condition, and purifying the mRNA from the impure preparation from step (a) by tangential flow filtration, wherein the mRNA purified from step (b) substantially free of prematurely aborted RNA sequences and/or enzyme reagents used in in vitro synthesis.Type: GrantFiled: March 26, 2018Date of Patent: December 29, 2020Assignee: Translate Bio, Inc.Inventors: Michael Heartlein, Frank DeRosa, Anusha Dias, Shrirang Karve
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Patent number: 10864267Abstract: The present invention provides, among other things, a method of intra-articular delivery of messenger RNA (mRNA), comprising administering into a joint of a subject in need of delivery a composition comprising an mRNA encoding a protein, such that the administering of the composition results in expression of the protein encoded by the mRNA in the joint.Type: GrantFiled: March 6, 2018Date of Patent: December 15, 2020Assignee: TRANSLATE BIO, INC.Inventors: Frank DeRosa, Michael Heartlein
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Patent number: 10835583Abstract: The present invention provides, among other things, methods of treating ornithine transcarbamylase (OTC) deficiency, including administering to a subject in need of treatment a composition comprising an mRNA encoding an ornithine transcarbamylase (OTC) protein at an effective dose and an administration interval such that at least one symptom or feature of the OTC deficiency is reduced in intensity, severity, or frequency or has delayed 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.Type: GrantFiled: June 13, 2017Date of Patent: November 17, 2020Assignee: Translate Bio, Inc.Inventors: Frank DeRosa, Michael Heartlein, Lianne Smith, Shrirang Karve
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Patent number: 10822368Abstract: Disclosed are messenger RNA molecules and related compositions incorporating a 4?-thio modification in the furanose ring of at least one nucleotide residue, and methods of using these mRNAs to produce an encoded therapeutic protein in vivo and to treat or prevent diseases or disorders. In certain embodiments, the 4?-thio modified mRNA provides for enhanced stability and/or reduced immunogenicity in in vivo therapies.Type: GrantFiled: February 21, 2019Date of Patent: November 3, 2020Assignee: Translate Bio, Inc.Inventors: Frank DeRosa, Michael Heartlein
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Patent number: 10808241Abstract: 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.Type: GrantFiled: February 27, 2018Date of Patent: October 20, 2020Assignee: Translate Bio, Inc.Inventors: Jonathan Abysalh, Daniel Crawford, Frank DeRosa, Shrirang Karve, Anusha Dias, Michael Heartlein
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Patent number: 10786455Abstract: Disclosed herein are novel lipids and liposomal compositions prepared using such compounds and related methods of neutralizing or otherwise modifying such liposomal compositions. The lipids described herein are useful for example, as liposomal vehicles to facilitate the delivery of encapsulated polynucleotides to target cells and the subsequent transfection of such target cells. In certain embodiments, one or more of the compounds that comprise the liposomal delivery vehicle may be neutralized or further modified such that the properties of the liposomal delivery vehicle are modified.Type: GrantFiled: November 6, 2018Date of Patent: September 29, 2020Assignee: Translate Bio, Inc.Inventors: Frank DeRosa, Braydon Charles Guild, Michael Heartlein
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Patent number: 10780183Abstract: The present invention provides methods and compositions of treating Friedreich's ataxia (FRDA) based on administering an mRNA encoding a frataxin protein.Type: GrantFiled: June 19, 2018Date of Patent: September 22, 2020Assignee: TRANSLATE BIO, INC.Inventors: Michael Heartlein, Frank DeRosa, Jonathan Cherry, Paula Lewis, Shrirang Karve, Zarna Patel, Darshan Parekh, Susan Wood, Chi-Sung Chiu, Caroline J. Woo
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Patent number: 10780052Abstract: The present invention provides, among other things, methods and compositions for effective delivery of messenger RNA (mRNA) to the central nervous 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.Type: GrantFiled: August 14, 2017Date of Patent: September 22, 2020Assignee: TRANSLATE BIO, INC.Inventors: Frank DeRosa, Michael Heartlein, Shrirang Karve
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Patent number: 10766852Abstract: Disclosed herein are novel compounds, pharmaceutical compositions comprising such compounds and related methods of their use. The compounds described herein are useful, e.g., as liposomal delivery vehicles to facilitate the delivery of encapsulated polynucleotides to target cells and subsequent transfection of said target cells, and in certain embodiments are characterized as having one or more properties that afford such compounds advantages relative to other similarly classified lipids.Type: GrantFiled: August 10, 2018Date of Patent: September 8, 2020Assignee: Translate Bio, Inc.Inventors: Frank DeRosa, Braydon Charles Guild, Michael Heartlein
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Patent number: 10702478Abstract: Disclosed herein are novel compounds, pharmaceutical compositions comprising such compounds and related methods of their use. The compounds described herein are useful, e.g. as liposomal delivery vehicles to facilitate the delivery of encapsulated polynucleotides to target cells and subsequent transfection of said target cells, and in certain embodiments are characterized as having one or more properties that afford such compounds advantages relative to other similarly classified lipids.Type: GrantFiled: June 25, 2019Date of Patent: July 7, 2020Assignee: Translate Bio, Inc.Inventors: Braydon Charles Guild, Michael Heartlein, Frank DeRosa, Jerry Chi Zhang
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Patent number: 10646504Abstract: Disclosed herein are pharmaceutical compositions that comprise “blends” of lipid nanoparticles and related methods of using such blended compositions to deliver polynucleotides to one or more target cells, tissues or organs. The blended compositions are generally characterized as being able to efficiently deliver polynucleotides to target cells and by their ability to enhance the expression of such polynucleotides and the production of functional proteins by target cells.Type: GrantFiled: October 10, 2018Date of Patent: May 12, 2020Assignee: Translate Bio, Inc.Inventors: Frank DeRosa, Lianne Smith, Michael Heartlein, Braydon Charles Guild
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Patent number: 10626439Abstract: The present invention provides, among other things, methods of quantitating mRNA capping efficiency, particularly mRNA synthesized in vitro. In some embodiments, methods according to the present invention comprise providing an mRNA sample containing capped and uncapped mRNA, providing a cap specific binding substance under conditions that permit the formation of a complex between the cap specific binding substance and the capped mRNA, and quantitatively determining the amount of the complex as compared to a control, thereby quantifying mRNA capping efficiency.Type: GrantFiled: March 14, 2014Date of Patent: April 21, 2020Assignee: Translate Bio, Inc.Inventors: Michael Heartlein, Frank DeRosa, Anusha Dias
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Patent number: 10584165Abstract: The present invention provides, among other things, methods and compositions for delivering an antibody in vivo by administering to a subject in need thereof one or more mRNAs encoding a heavy chain and a light chain of an antibody, and wherein the antibody is expressed systemically in the subject. In some embodiments, the one or more mRNAs comprise a first mRNA encoding the heavy chain and a second mRNA encoding the light chain of the antibody.Type: GrantFiled: September 12, 2018Date of Patent: March 10, 2020Assignee: Translate Bio, Inc.Inventors: Michael Heartlein, Frank DeRosa, Anusha Dias, Braydon Charles Guild
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Patent number: 10576166Abstract: Disclosed herein are compositions and methods of modulating the expression of gene or the production of a protein by transfecting target cells with nucleic acids. The compositions disclosed herein demonstrate a high transfection efficacy and are capable of ameliorating diseases associated with protein or enzyme deficiencies.Type: GrantFiled: April 6, 2016Date of Patent: March 3, 2020Assignee: Translate Bio, Inc.Inventors: Frank DeRosa, Michael Heartlein
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Patent number: 10507183Abstract: Disclosed herein are novel compounds, pharmaceutical compositions comprising such compounds and related methods of their use. The compounds described herein are useful, e.g., as liposomal delivery vehicles to facilitate the delivery of encapsulated polynucleotides to target cells and subsequent transfection of said target cells, and in certain embodiments are characterized as having one or more properties that afford such compounds advantages relative to other similarly classified lipids.Type: GrantFiled: February 2, 2018Date of Patent: December 17, 2019Assignee: Translate Bio, Inc.Inventors: Braydon Charles Guild, Michael Heartlein, Frank DeRosa, Jerry Chi Zhang
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Patent number: 10507249Abstract: Disclosed herein are compositions and methods for modulating the production of a protein in a target cell. The compositions and methods disclosed herein are capable of ameliorating diseases associated with protein or enzyme deficiencies.Type: GrantFiled: July 3, 2019Date of Patent: December 17, 2019Assignee: Translate Bio, Inc.Inventors: Braydon Charles Guild, Frank DeRosa, Michael Heartlein
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Patent number: 10493031Abstract: The present invention provides, among other things, methods of delivering mRNA in vivo, including administering 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 expression of the protein encoded by the mRNA in vivo, wherein the liposome comprises a cationic lipid of formula I-c: or a pharmaceutically acceptable salt thereof.Type: GrantFiled: July 3, 2018Date of Patent: December 3, 2019Assignees: Translate Bio, Inc., Massachusetts Institute of TechnologyInventors: Michael Heartlein, Daniel Anderson, Yizhou Dong, Frank DeRosa