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1. 发明申请

WO2020061332A1 STEROL ANALOGS AND USES THEREOF 审中-公开
公开(公告)号：WO2020061332A1
公开(公告)日：2020-03-26
申请号：PCT/US2019/051959
申请日：2019-09-19
申请人： MODERNATX, INC.
发明人： BENENATO, Kerry , CORNEBISE, Mark , HENNESSY, Edward, J.
IPC分类号： A61K31/575 , C07J9/00
摘要： The invention relates to compositions and methods for the preparation, manufacture, and therapeutic use of compositions comprising mRNA and a lipid nanoparticle comprising a compound of the invention and an ionizable lipid.

2. 发明申请

WO2017201328A1 POLYNUCLEOTIDES ENCODING α-GALACTOSIDASE A FOR THE TREATMENT OF FABRY DISEASE 审中-公开
标题翻译：编码α-半乳糖苷酶A用于治疗败血症的多核苷酸
公开(公告)号：WO2017201328A1
公开(公告)日：2017-11-23
申请号：PCT/US2017/033398
申请日：2017-05-18
申请人： MODERNATX, INC.
发明人： MARTINI, Paolo , HOGE, Stephen , BENENATO, Kerry , PRESNYAK, Vladimir , MCFADYEN, Iain , KUMARASINGHE, Ellalahewage Sathyajith , ZHU, Xuling , GUEY, Lin Tung , SABNIS, Staci
IPC分类号： C12N15/67
摘要： 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.
摘要翻译：本发明涉及用于治疗法布里病的mRNA疗法。用于本发明的mRNA在体内施用时编码人α-半乳糖苷酶A（GLA），其同种型，其功能片段和包含GLA的融合蛋白。当向其施用时，本发明的mRNA优选包封在脂质纳米颗粒（LNP）中以实现向受试者中的细胞和/或组织的有效递送。本发明的mRNA疗法增加和/或恢复受试者中GLA表达和/或活性的缺陷水平。本发明的mRNA疗法进一步降低受试者中与缺乏的GLA活性有关的毒性代谢物的水平，即Gb3和溶血-Gb3。

3. 发明申请

WO2022204371A1 LIPID NANOPARTICLES CONTAINING POLYNUCLEOTIDES ENCODING GLUCOSE-6-PHOSPHATASE AND USES THEREOF 审中-公开
公开(公告)号：WO2022204371A1
公开(公告)日：2022-09-29
申请号：PCT/US2022/021692
申请日：2022-03-24
申请人： MODERNATX, INC.
发明人： BENENATO, Kerry , CORNEBISE, Mark , HENNESSY, Edward
IPC分类号： A61K9/00 , A61K9/51 , A61K31/7088 , A61K31/7105 , A61P3/00 , C12N9/16 , C12N15/87 , A61K31/713 , A61K38/465 , A61K48/0041 , A61K48/005 , A61K9/0019 , A61K9/5123 , C12N15/88 , C12Y301/03009
摘要： This disclosure relates to ionizable amino lipid-based lipid nanoparticles for delivery of mRNA encoding glucose-6-phosphatase. Lipid nanoparticle/mRNA therapies of the invention increase and/or restore deficient levels of glucose-6-phosphatase expression and/or activity in subjects and are useful for the treatment of glycogen storage disease type 1a (GSD-Ia). Lipid nanoparticle/mRNA therapies of the invention increase glucose production and reduce the abnormal accumulation of glycogen and glucose-6-phosphate associated with GSD-Ia.

4. 发明申请

WO2022204370A1 LIPID NANOPARTICLES AND POLYNUCLEOTIDES ENCODING ORNITHINE TRANSCARBAMYLASE FOR THE TREATMENT OF ORNITHINE TRANSCARBAMYLASE DEFICIENCY 审中-公开
公开(公告)号：WO2022204370A1
公开(公告)日：2022-09-29
申请号：PCT/US2022/021690
申请日：2022-03-24
申请人： MODERNATX, INC.
发明人： BENENATO, Kerry , BICKNELL, Alicia Anne , CORNEBISE, Mark , DOUSIS, Athanasios , GIESSEL, Andrew Jacob , HENNESSY, Edward , JAIN, Ruchi , KÖHRER, Caroline , LICHT, Stuart Spencer , RAVICHANDRAN, Kanchana , REID, David
IPC分类号： C12N9/10 , C12N15/67 , A01K2217/075 , A01K2227/105 , A01K2267/0306 , A61K48/0041 , A61K48/005 , A61K9/5123 , C12N9/1018 , C12Y201/03003
摘要： This disclosure relates to mRNA therapy for the treatment of ornithine transcarbamylase deficiency (OTCD). mRNAs for use in the invention, when administered in vivo, encode human ornithine transcarbamylase (OTC). mRNA therapies of the disclosure increase and/or restore deficient levels of OTC expression and/or activity in subjects. mRNA therapies of the disclosure further decrease levels of toxic ammonia associated with deficient OTC activity in subjects.

5. 发明申请

WO2022204380A1 LIPID NANOPARTICLES CONTAINING POLYNUCLEOTIDES ENCODING PROPIONYL-COA CARBOXYLASE ALPHA AND BETA SUBUNITS AND USES THEREOF 审中-公开
公开(公告)号：WO2022204380A1
公开(公告)日：2022-09-29
申请号：PCT/US2022/021713
申请日：2022-03-24
申请人： MODERNATX, INC.
发明人： BENENATO, Kerry , CORNEBISE, Mark , HENNESSY, Edward
IPC分类号： C12N15/52
摘要： This disclosure relates to ionizable amino lipid-based lipid nanoparticles for delivery of mRNA encoding propionyl-CoA carboxylase alpha (PCCA) and propionyl-CoA carboxylase beta (PCCB). Lipid nanoparticle/mRNA therapies of the invention increase and/or restore deficient levels of PCCA and/or PCCB expression and/or activity in subjects and are useful for the treatment of propionic academia.

6. 发明申请

WO2022204369A1 POLYNUCLEOTIDES ENCODING METHYLMALONYL-COA MUTASE FOR THE TREATMENT OF METHYLMALONIC ACIDEMIA 审中-公开
公开(公告)号：WO2022204369A1
公开(公告)日：2022-09-29
申请号：PCT/US2022/021689
申请日：2022-03-24
申请人： MODERNATX, INC.
发明人： BENENATO, Kerry , CORNEBISE, Mark , HENNESSY, Edward , JAIN, Ruchi , PRESNYAK, Vladimir
IPC分类号： C12N15/52 , A61K9/127 , A61K38/52 , A61P3/00 , A61P43/00 , C12N9/90 , A61K9/0019 , A61K9/5123 , C12Y504/99002
摘要： This disclosure relates to messenger RNA (mRNA) therapy for the treatment of methylmalonic acidemia. mRNAs for use in the invention, when administered in vivo, encode methylmalonyl-CoA mutase. mRNA therapies of the disclosure increase and/or restore deficient levels of MUT expression and/or activity in subjects.

7. 发明申请

WO2019152557A1 COMPOSITIONS AND METHODS FOR DELIVERY OF AGENTS TO IMMUNE CELLS 审中-公开
公开(公告)号：WO2019152557A1
公开(公告)日：2019-08-08
申请号：PCT/US2019/015913
申请日：2019-01-30
申请人： MODERNATX, INC.
发明人： BESIN, Gilles , BRITO, Luis , HOGE, Stephen G. , HENNESSY, Edward , CORNEBISE, Mark , BENENATO, Kerry , SABNIS, Staci , DANNEMAN, Michael W.
IPC分类号： A61K9/127 , C12N15/88 , A61K47/24 , A61K47/28
摘要： 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.

8. 发明申请

WO2017201352A1 MRNA COMBINATION THERAPY FOR THE TREATMENT OF CANCER 审中-公开
标题翻译： MRNA联合治疗癌症的研究
公开(公告)号：WO2017201352A1
公开(公告)日：2017-11-23
申请号：PCT/US2017/033425
申请日：2017-05-18
申请人： MODERNATX, INC.
发明人： FREDERICK, Joshua , HEWITT, Susannah , BAI, Ailin , HOGE, Stephen , PRESNYAK, Vladimir , MCFADYEN, Iain , BENENATO, Kerry , KUMARASINGHE, Ellalahewage Sathyajith
IPC分类号： A61K39/395 , C12N15/11 , C07K14/54 , C07K14/705
摘要： The present disclosure relates to the use of nucleic acid (e.g., mRNA) combination therapies for the treatment of cancer. The disclosure provides compositions, and methods for their preparation, manufacture, and therapeutic use, wherein those compositions comprise at least two polynucleotides (e.g., mRNAs) in combination wherein the at least two polynucleotides are selected from the group consisting of (i) a polynucleotide encoding an immune response primer (e.g., IL23), (ii) a polynucleotide encoding an immune response co-stimulatory signal (e.g., OX40L), (iii) a polynucleotide encoding a checkpoint inhibitor (e.g., an anti CTLA-4 antibody), and, (iv) a combination thereof. The therapeutic methods disclosed herein comprise, e.g., the administration of a combination therapy disclosed herein for the treatment of cancer, e.g., by reducing the size of a tumor or inhibiting the growth of a tumor, in a subject in need thereof. In some aspects, the combination therapies disclosed herein disclosed are administered intratumorally.
摘要翻译：本公开涉及核酸（例如，mRNA）组合疗法在治疗癌症中的用途。本公开内容提供了用于其制备，制造和治疗用途的组合物和方法，其中所述组合物包含至少两种多核苷酸（例如mRNA），其中所述至少两种多核苷酸选自（i）多核苷酸编码免疫应答引物（例如IL23），（ii）编码免疫应答共刺激信号（例如OX40L）的多核苷酸，（iii）编码检查点抑制剂（例如抗CTLA-4抗体）的多核苷酸，和（iv）其组合。本文公开的治疗方法包括例如在有此需要的受试者中施用本文公开的用于治疗癌症的组合疗法，例如通过减小肿瘤的大小或抑制肿瘤的生长。在一些方面，本文公开的组合疗法在肿瘤内施用。

9. 发明申请

WO2017201350A1 POLYNUCLEOTIDES ENCODING INTERLEUKIN-12 (IL12) AND USES THEREOF 审中-公开
标题翻译：编码白细胞介素-12（IL12）的多核苷酸及其用途
公开(公告)号：WO2017201350A1
公开(公告)日：2017-11-23
申请号：PCT/US2017/033422
申请日：2017-05-18
申请人： MODERNATX, INC.
发明人： FREDERICK, Joshua , HEWITT, Susannah , BAI, Ailin , HOGE, Stephen , PRESNYAK, Vladimir , MCFADYEN, Iain , BENENATO, Kerry , KUMARASINGHE, Ellalahewage Sathyajith
IPC分类号： A61K38/20 , C07K14/54 , C12N15/67
摘要： The present disclosure relates to polynucleotides comprising an open reading frame of linked nucleosides encoding human interleukin-12 (IL12), functional fragments thereof, and fusion proteins comprising IL12. In some embodiments, the open reading frame is sequence-optimized. In particular embodiments, the disclosure provides sequence-optimized polynucleotides comprising nucleotides encoding the polypeptide sequence of human IL12, or sequences having high sequence identity with those sequence optimized polynucleotides.
摘要翻译：本公开涉及包含编码人白细胞介素-12（IL12）的连接核苷的开放阅读框，其功能片段和包含IL12的融合蛋白的多核苷酸。在一些实施方案中，开放阅读框是序列优化的。在具体的实施方案中，本公开提供了包含编码人IL12的多肽序列的核苷酸或与那些序列优化的多核苷酸具有高度序列同一性的序列的序列优化的多核苷酸。

10. 发明申请

WO2017201333A1 POLYNUCLEOTIDES ENCODING LIPOPROTEIN LIPASE FOR THE TREATMENT OF HYPERLIPIDEMIA 审中-公开
标题翻译：多核苷酸编码脂蛋白脂酶治疗高脂血症
公开(公告)号：WO2017201333A1
公开(公告)日：2017-11-23
申请号：PCT/US2017/033403
申请日：2017-05-18
申请人： MODERNATX, INC.
发明人： BENENATO, Kerry , HOGE, Stephen , MARTINI, Paolo , MCFADYEN, Iain , PRESNYAK, Vladimir , KUMARASINGHE, Ellalahewage, Sathyajith
IPC分类号： A61K48/00 , A61P3/06 , C12N15/88 , C12N15/55 , A61K9/51 , A61K47/16 , A61K47/22 , A61K47/14
摘要： The invention relates to mRNA therapy for the treatment of hyperlipidemia. mRNAs for use in the invention, when administered in vivo, encode human lipoprotein lipase (LPL), isoforms thereof, functional fragments thereof, and fusion proteins comprising LPL. 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 therapeaies of the invention increase and/or restore deficient levels of LPL expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of triglycerides associated with deficient LPL activity in subjects.
摘要翻译：本发明涉及用于治疗高脂血症的mRNA疗法。当体内施用时，用于本发明的mRNA编码人脂蛋白脂肪酶（LPL），其同种型，其功能片段和包含LPL的融合蛋白。优选将本发明的mRNA封装在脂质纳米颗粒（LNP）中以实现向受试者中的细胞和/或组织有效递送，当向其施用时，本发明的mRNA治疗增加和/或恢复LPL表达水平和/或科目。本发明的mRNA治疗进一步降低与受试者中LPL活性缺陷相关的甘油三酯水平。

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