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公开(公告)号：US20190002890A1
公开(公告)日：2019-01-03
申请号：US16110309
申请日：2018-08-23
Applicant: ModernaTX, Inc.
Inventor： Paolo MARTINI , Stephen G. HOGE , Kerry BENENATO , Vladimir PRESNYAK , Iain MCFADYEN , Ellalahewage Sathyajith KUMARASINGHE , Xuling ZHU , Lin Tung GUEY , Staci SABNIS
Abstract: The invention relates to mRNA therapy for the treatment of Fabry disease. mRNAs for use in the invention, when administered in vivo, encode human the α-galactosidase A (GLA), isoforms thereof, functional fragments thereof, and fusion proteins comprising GLA. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of GLA expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient GLA activity in subjects, namely Gb3 and lyso-Gb3.
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公开(公告)号：US20190382774A1
公开(公告)日：2019-12-19
申请号：US16300223
申请日：2017-05-18
Applicant: ModernaTX, Inc.
Inventor： Stephen G. HOGE , Kerry BENENATO , Vladimir PRESNYAK , Iain MCFADYEN , Ellalahewage Sathyajith KUMARASINGHE , Staci SABNIS , William BUTCHER
IPC: C12N15/67 , A61K31/7115 , A61K48/00
Abstract: The invention related to polyribonucleotides comprising an open reading frame of linked nucleosides encoding a polypeptide of interest (e.g., a therapeutic polypeptide), isoforms thereof, functional fragments thereof, and fusion proteins comprising the polypeptide. In some embodiments, the open reading frame is sequence-optimized. In particular embodiments, the invention provides sequence-optimized polyribonucleotides comprising nucleotides encoding the sequence of the polypeptide of interest, or sequence having high sequence identity with those sequence optimized polyribonucleotides.
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公开(公告)号：US20190314291A1
公开(公告)日：2019-10-17
申请号：US16262758
申请日：2019-01-30
Applicant: ModernaTX, Inc.
Inventor： Gilles BESIN , Luis BRITO , Stephen G. HOGE , Edward HENNESSY , Mark CORNEBISE , Kerry BENENATO , Staci SABNIS , Michael W. DANNEMAN
IPC: A61K9/51 , C07K14/525 , A61P35/00 , C07K14/705 , A61K39/245
Abstract: The disclosure features immune cell delivery lipid nanoparticle (LNP) compositions that allow for enhanced delivery of agents, e.g., nucleic acids, such as therapeutic and/or prophylactic RNAs, to immune cells, in particular T cells, as well as B cells, dendritic cells and monocytes. The LNPs comprise an effective amount of an immune cell delivery potentiating lipid such that delivery of an agent by an immune cell delivery LNP is enhanced as compared to an LNP lacking the immune cell delivery potentiating agent. Methods of using the immune cell delivery LNPs for delivery of agents, e.g., nucleic acid delivery, for protein expression, for modulating immune cell activity and modulating immune responses are also disclosed.
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公开(公告)号：US20190298657A1
公开(公告)日：2019-10-03
申请号：US16302298
申请日：2017-05-18
Applicant: MODERNATX, INC.
Inventor： Paolo MARTINI , Stephen HOGE , Kerry BENENATO , Vladimir PRESNYAK , Iain McFADYEN , Ellalahewage Sathyajith KUMARASINGHE , Evan Lockwood RACHLIN , Staci SABNIS
Abstract: The invention relates to mRNA therapy for the treatment of VLCADD. mRNAs for use in the invention, when administered in vivo, encode human acyl-CoA dehydrogenase, very longchain (ACADVL), isoforms thereof, functional fragments thereof, and fusion proteins comprising ACADVL. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of ACADVL expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient ACADVL activity in subjects, namely acylcarnitine and acylcarnitine metabolites.
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5.发明申请公开(公告)号：US20230112986A1
公开(公告)日：2023-04-13
申请号：US17813984
申请日：2022-07-21
Inventor： Paolo MARTINI , Stephen HOGE , Kerry BENENATO , Vladimir PRESNVAK , Lei JIANG , Iain MCFADYEN , Ellalahewage Sathyajith KUMARASINGHE , Antonio FONTANELLAS ROMA , Pedro BERRAONDO LOPEZ , Matias Antonio AVILA ZARAGOZA , Lin Tung GUEY , Staci SABNIS
Abstract: The invention relates to mRNA therapy for the treatment of Acute Intermittent Porphyria (AIP). mRNAs for use in the invention, when administered in vivo, encode human porphobilinogen deaminase (PBGD), isoforms thereof, functional fragments thereof, and fusion proteins comprising PBGD. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to affect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of PBGD expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient PBGD activity in subjects, namely porphobilinogen and aminolevulinate (PBG and ALA).
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Patent Agency Ranking
公开(公告)号：US20200149052A1
公开(公告)日：2020-05-14
申请号：US16570351
申请日：2019-09-13
Applicant: ModernaTX, Inc.
Inventor： Paolo MARTINI , Stephen G. HOGE , Kerry BENENATO , Vladimir PRESNYAK , Iain MCFADYEN , Ellalahewage Sathyajith KUMARASINGHE , Xuling ZHU , Lin Tung GUEY , Staci SABNIS
Abstract: The invention relates to mRNA therapy for the treatment of Fabry disease. mRNAs for use in the invention, when administered in vivo, encode human the α-galactosidase A (GLA), isoforms thereof, functional fragments thereof, and fusion proteins comprising GLA. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of GLA expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient GLA activity in subjects, namely Gb3 and lyso-Gb3.
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公开(公告)号：US20180214579A1
公开(公告)日：2018-08-02
申请号：US15936032
申请日：2018-03-26
Applicant: ModernaTX, Inc.
Inventor： Örn ALMARSSON , Noubar B. AFEYAN , Joseph Beene BOLEN , Staci SABNIS
IPC: A61K48/00 , A61K9/00 , A61K31/4172 , A61K31/405 , A61K31/401 , A61K31/198 , A61K38/00
CPC classification number: A61K48/0066 , A61K9/0019 , A61K31/198 , A61K31/401 , A61K31/405 , A61K31/4172 , A61K38/00
Abstract: The present invention relates to compositions and methods for the preparation, manufacture and therapeutic use of polynucleotides formulations containing amino acids.
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公开(公告)号：US20230323371A1
公开(公告)日：2023-10-12
申请号：US18296596
申请日：2023-04-06
Applicant: ModernaTX, Inc.
Inventor： Paolo MARTINI , Stephen G. HOGE , Kerry BENENATO , Vladimir PRESNYAK , Iain MCFADYEN , Ellalahewage Sathyajith KUMARASINGHE , Xuling ZHU , Lin Tung GUEY , Staci SABNIS
CPC classification number: C12N15/67 , A61K9/5123 , C12N9/2465 , C12Y302/01022 , A61P3/00 , A61K38/47 , A61K48/00
Abstract: The invention relates to mRNA therapy for the treatment of Fabry disease. mRNAs for use in the invention, when administered in vivo, encode human the α-galactosidase A (GLA), isoforms thereof, functional fragments thereof, and fusion proteins comprising GLA. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of GLA expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient GLA activity in subjects, namely Gb3 and lyso-Gb3.
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公开(公告)号：US20230027864A1
公开(公告)日：2023-01-26
申请号：US17747618
申请日：2022-05-18
Applicant: ModernaTX, Inc.
Inventor： Gilles BESIN , Luis BRITO , Stephen G. HOGE , Edward HENNESSY , Mark CORNEBISE , Kerry BENENATO , Staci SABNIS , Michael W. DANNEMAN
IPC: A61K9/51 , A61P35/00 , A61K39/245 , C07K14/525 , C07K14/705
Abstract: The disclosure features immune cell delivery lipid nanoparticle (LNP) compositions that allow for enhanced delivery of agents, e.g., nucleic acids, such as therapeutic and/or prophylactic RNAs, to immune cells, in particular T cells, as well as B cells, dendritic cells and monocytes. The LNPs comprise an effective amount of an immune cell delivery potentiating lipid such that delivery of an agent by an immune cell delivery LNP is enhanced as compared to an LNP lacking the immune cell delivery potentiating agent. Methods of using the immune cell delivery LNPs for delivery of agents, e.g., nucleic acid delivery, for protein expression, for modulating immune cell activity and modulating immune responses are also disclosed.
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公开(公告)号：US20190000932A1
公开(公告)日：2019-01-03
申请号：US16110788
申请日：2018-08-23
Applicant: ModernaTX, Inc.
Inventor： Paolo MARTINI , Stephen G. HOGE , Kerry BENENATO , Vladimir PRESNYAK , Iain MCFADYEN , Ellalahewage Sathyajith KUMARASINGHE , Xuling ZHU , Lin Tung GUEY , Staci SABNIS
Abstract: The invention relates to mRNA therapy for the treatment of Fabry disease. mRNAs for use in the invention, when administered in vivo, encode human the α-galactosidase A (GLA), isoforms thereof, functional fragments thereof, and fusion proteins comprising GLA. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of GLA expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient GLA activity in subjects, namely Gb3 and lyso-Gb3.
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