Patents Assigned to Translate Bio, Inc.
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Patent number: 10493166Abstract: 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: June 7, 2018Date of Patent: December 3, 2019Assignee: Translate Bio, Inc.Inventors: Frank DeRosa, Michael Heartlein
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Patent number: 10471153Abstract: 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.Type: GrantFiled: November 10, 2017Date of Patent: November 12, 2019Assignee: Translate Bio, Inc.Inventors: Frank DeRosa, Michael Heartlein, Shrirang Karve
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Patent number: 10428349Abstract: 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.Type: GrantFiled: February 20, 2019Date of Patent: October 1, 2019Assignee: Translate Bio, Inc.Inventors: Frank DeRosa, Michael Heartlein, Daniel Crawford, Shrirang Karve
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Patent number: 10420791Abstract: The present invention is particularly useful for treating cystic fibrosis. The disclosure concerns method and compositions for treating cystic fibrosis. The method comprises inducing CFTR expression in epithelial cells in a lung of a mammal by contacting the epithelial cells in the lung of the mammal with a composition, where the composition is a pharmaceutical composition comprising in vitro transcribed mRNA.Type: GrantFiled: June 16, 2017Date of Patent: September 24, 2019Assignee: Translate Bio, Inc.Inventors: Michael Heartlein, Braydon Charles Guild, Frank DeRosa, Carsten Rudolph, Christian Plank, Lianne Smith
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Patent number: 10413618Abstract: 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: December 26, 2018Date of Patent: September 17, 2019Assignee: Translate Bio, Inc.Inventors: Braydon Charles Guild, Frank DeRosa, Michael Heartlein
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Patent number: 10350303Abstract: 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: February 26, 2019Date of Patent: July 16, 2019Assignee: Translate Bio, Inc.Inventors: Braydon Charles Guild, Frank DeRosa, Michael Heartlein
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Patent number: 10293057Abstract: 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: June 7, 2018Date of Patent: May 21, 2019Assignee: Translate Bio, Inc.Inventors: Frank DeRosa, Michael Heartlein
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Patent number: 10286082Abstract: 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: June 7, 2018Date of Patent: May 14, 2019Assignee: Translate Bio, Inc.Inventors: Frank DeRosa, Michael Heartlein
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Patent number: 10286083Abstract: 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: June 7, 2018Date of Patent: May 14, 2019Assignee: Translate Bio, Inc.Inventors: Frank DeRosa, Michael Heartlein
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Patent number: 10266559Abstract: 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: March 14, 2014Date of Patent: April 23, 2019Assignee: Translate Bio, Inc.Inventors: Frank DeRosa, Michael Heartlein
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Patent number: 10266843Abstract: 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.Type: GrantFiled: April 7, 2017Date of Patent: April 23, 2019Assignee: Translate Bio, Inc.Inventors: Frank DeRosa, Michael Heartlein, Daniel Crawford, Shrirang Karve
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Patent number: 10245229Abstract: Compositions comprising mRNA formulated for pulmonary administration and related methods for delivery of the mRNA and/or encoded protein to a non-lung cell or tissue. The compositions and methods may be used to prevent or ameliorate the symptoms of diseases associated with the mRNA encoded protein.Type: GrantFiled: June 7, 2013Date of Patent: April 2, 2019Assignees: Translate Bio, Inc., Ethris GmbHInventors: Michael Heartlein, Braydon Charles Guild, Frank DeRosa, Carsten Rudolph, Christian Plank
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Patent number: 10238754Abstract: 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: April 7, 2017Date of Patent: March 26, 2019Assignee: Translate Bio, Inc.Inventors: Braydon Charles Guild, Frank DeRosa, Michael Heartlein
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Patent number: 10208295Abstract: The present invention provides, among other things, methods of treating phenylketonuria (PKU), including administering to a subject in need of treatment a composition comprising an mRNA encoding phenylalanine hydroxylase (PAH) at an effective dose and an administration interval such that at least one symptom or feature of PKU is reduced in intensity, severity, or frequency or has delayed in 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: November 11, 2016Date of Patent: February 19, 2019Assignee: Translate Bio, Inc.Inventors: Frank DeRosa, Michael Heartlein, Anusha Dias
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Patent number: 10172924Abstract: The present invention provides, among other things, methods of treating Pompe disease, including administering to a subject in need of treatment a composition comprising an mRNA encoding acid alpha-glucosidase (GAA) at an effective dose and an administration interval such that at least one symptom or feature of Pompe disease is reduced in intensity, severity, or frequency or has delayed in 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: March 17, 2016Date of Patent: January 8, 2019Assignee: Translate Bio, Inc.Inventors: Frank DeRosa, Michael Heartlein
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Patent number: 10155785Abstract: 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.Type: GrantFiled: December 4, 2017Date of Patent: December 18, 2018Assignee: Translate Bio, Inc.Inventors: Frank DeRosa, Anusha Dias, Shrirang Karve, Michael Heartlein
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Patent number: 10143758Abstract: 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: March 13, 2013Date of Patent: December 4, 2018Assignee: Translate Bio, Inc.Inventors: Braydon Charles Guild, Frank DeRosa, Michael Heartlein
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Patent number: 10144942Abstract: The present invention provides, among other things, methods and compositions for large-scale production of capped mRNA using SUMO-Guanylyl Transferase fusion protein.Type: GrantFiled: October 14, 2016Date of Patent: December 4, 2018Assignee: Translate Bio, Inc.Inventors: Bettina Strack-Logue, Frank DeRosa, Michael Heartlein
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Patent number: 10137086Abstract: 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: January 23, 2018Date of Patent: November 27, 2018Assignee: Translate Bio, Inc.Inventors: Frank DeRosa, Braydon Charles Guild, Michael Heartlein
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Patent number: 10137087Abstract: 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: January 23, 2018Date of Patent: November 27, 2018Assignee: Translate Bio, Inc.Inventors: Frank DeRosa, Braydon Charles Guild, Michael Heartlein