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1. 发明申请

US20210009995A1 POLYRIBONUCLEOTIDE CONTAINING DEUTERATED NUCLEOTIDES 有权
公开(公告)号：US20210009995A1
公开(公告)日：2021-01-14
申请号：US16969897
申请日：2019-02-13
申请人： ethris GmbH
发明人： Carsten Rudolph , Johannes Geiger , Sandra Hocke , Ludwig Weiss
IPC分类号： C12N15/11
摘要： The present disclosure provides polyribonucleotides, in particular polyribonucleotides, which comprise deuterated adenosine, cytidine, guanosine, and/or uridine residues and which show reduced immunogenicity and/or enhanced expression, and methods of using such polyribonucleotides for the therapy of diseases.

2. 发明申请

US20150126589A1 Pulmonary Delivery of Messenger RNA 审中-公开
标题翻译：信使RNA的肺部递送
公开(公告)号：US20150126589A1
公开(公告)日：2015-05-07
申请号：US14406112
申请日：2013-06-07
申请人： ETHRIS GMBH
发明人： Johannes Geiger , Manish Kumar Aneja , Carsten Rudolph
IPC分类号： A61K48/00
CPC分类号： A61K48/0041 , A61K31/7105 , A61K47/59 , A61K48/0075 , A61K2300/00
摘要： The invention discloses a method for expressing an mRNA in lung wherein —the mRNA to be expressed is combined with polyethyleneimine (PEI) to provide a combination comprising the mRNA and PEI; —the combination comprising the mRNA and PEI is administered to lung where it enters lung cells; and —the mRNA is expressed in the lung cells.
摘要翻译：本发明公开了一种在肺中表达mRNA的方法，其中将待表达的mRNA与聚乙烯亚胺（PEI）组合以提供包含mRNA和PEI的组合; - 将包含mRNA和PEI的组合施用于肺进入肺细胞的肺; 并且mRNA在肺细胞中表达。

3. 发明申请

US20200308556A1 ORNITHINE TRANSCARBAMYLASE CODING POLYRIBONUCLEOTIDES AND FORMULATIONS THEREOF 审中-公开
公开(公告)号：US20200308556A1
公开(公告)日：2020-10-01
申请号：US16475678
申请日：2017-12-15
申请人： ethris GmbH
发明人： Carsten Rudolph , Rebekka Kubisch-Dohem , Manish Kumar Aneja , Johannes Geiger , Marino Schuhmacher
IPC分类号： C12N9/10 , C07K14/47
摘要： In certain aspects, the disclosure relates to compositions comprising modified Ornithine transcarbamylase (OTC) polyribonucleotides and methods of use.

4. 发明授权

US11136585B2 UTRs increasing the translation efficiency of RNA molecules 有权
公开(公告)号：US11136585B2
公开(公告)日：2021-10-05
申请号：US15739147
申请日：2016-06-30
申请人： Ethris GmbH
发明人： Carsten Rudolph , Manish Kumar Aneja , Mehrije Ferizi , Johannes Geiger
IPC分类号： C07H21/04 , C12N15/67 , C07K14/47 , A61K38/00
摘要： Described is an RNA molecule comprising (a) a coding region coding for a polypeptide; and (b) upstream of said coding region one or more UTR(s) comprising the sequence as shown in SEQ ID NO:1 or a sequence which shows 1 to 4 substitutions in comparison to SEQ ID NO:1 and which results in an RNA molecule having the same or a higher translation efficiency as an RNA molecule comprising an UTR comprising SEQ ID NO:1; and/or (c) downstream of said coding region one or more UTR(s) comprising the sequence as shown in SEQ ID NO:2 or a sequence which shows 1 to 7 substitutions in comparison to SEQ ID NO:2 and which results in an RNA molecule having the same or a higher translation efficiency as an RNA molecule comprising an UTR comprising SEQ ID NO:2; wherein said polypeptide encoded by said coding region is not a cytochrome b-245 alpha polypeptide (CYBA). Moreover, described is a nucleic acid molecule encoding the RNA molecule according to the present invention. Further, described is a vector comprising the nucleic acid molecule according to the present invention and to a host cell comprising the vector according to the present invention. Further, described is a pharmaceutical composition comprising the RNA molecule according to the present invention and optionally a pharmaceutically acceptable carrier. Moreover, described is a kit comprising the RNA molecule according to the present invention. Finally, described is the use of one or more UTR(s) as defined in (b) and/or one or more UTR(s) as defined in (c) for increasing the efficiency of translating a coding region of an RNA molecule into a polypeptide or a protein encoded by said coding region.

5. 发明申请

US20220162586A1 MRNA PURIFICATION BY TANGENTIAL FLOW FILTRATION 有权
公开(公告)号：US20220162586A1
公开(公告)日：2022-05-26
申请号：US17430318
申请日：2020-02-11
申请人： ethris GmbH
发明人： Johannes Geiger , Martin Treml
IPC分类号： C12N15/10 , A61K31/7088
摘要： The present disclosure provides a method of purifying mRNA molecules comprising (Ia) purifying precipitated mRNA molecules from a suspension comprising precipitated mRNA molecules, (Ib) washing and dissolving the purified precipitated mRNA molecules, (IIa) purifying the mRNA molecules using a solution comprising a chelating agent, followed by (IIb) washing the purified mRNA molecules, wherein steps (Ia) to (IIb) are performed using tangential flow filtration.

6. 发明授权

US11104887B2 Ornithine transcarbamylase coding polyribonucleotides and formulations thereof 有权
公开(公告)号：US11104887B2
公开(公告)日：2021-08-31
申请号：US16475678
申请日：2017-12-15
申请人： ethris GmbH
发明人： Carsten Rudolph , Rebekka Kubisch-Dohmen , Manish Kumar Aneja , Johannes Geiger , Marino Schuhmacher
IPC分类号： C12N9/10 , C07K14/47
摘要： In certain aspects, the disclosure relates to compositions comprising modified Ornithine transcarbamylase (OTC) polyribonucleotides and methods of use.

7. 发明授权

US11479768B2 ATP-binding cassette family coding polyribonucleotides and formulations thereof 有权
公开(公告)号：US11479768B2
公开(公告)日：2022-10-25
申请号：US15739146
申请日：2016-06-30
申请人： Ethris GmbH
发明人： Christian Plank , Carsten Rudolph , Manish Kumar Aneja , Ludwig Weiss , Mehrije Ferizi , Johannes Geiger
IPC分类号： C12N15/113 , C12N15/63 , A61K48/00 , A61K38/16 , C07K14/47 , C12N15/62 , A61K9/107 , A61K9/127 , A61K9/51
摘要： Polynucleotides encoding peptides, proteins, enzymes, and functional fragments thereof are disclosed. The polynucleotides of the disclosure can be effectively delivered to an organ, such as the lung, and expressed within cells of the organ. The polyribonucleotides of the disclosure can be used to treat a disease or condition associated with a gene of the ATP-binding cassette (ABC) family, such as ABCA3.

8. 发明申请

US20220211807A1 TREATMENT OF CILIOPATHIES 有权
公开(公告)号：US20220211807A1
公开(公告)日：2022-07-07
申请号：US17431146
申请日：2020-02-13
申请人： ethris GmbH
发明人： Carsten Rudolph , Verena Kretzschmann , Rebekka Kubisch- Dohmen , Christian Dohmen , Johannes Geiger , Manish Aneja , Ludwig Weiss , Heymut Omran , Petra Pennekamp , Kai Wohlgemuth , Sandra Cindric , Niki Tomas Loges , Johanna Raidt , Adrian Ter Steege
IPC分类号： A61K38/17 , A61K31/198 , A61K31/7105 , A61P11/00
摘要： The present disclosure provides a pharmaceutical composition comprising a polyribonucleotide for use in treating a ciliopathy in a subject suffering of a ciliopathy, wherein the polyribonucleotide encodes a functional version of a protein a defect of which is associated with said ciliopathy, and wherein administration of said pharmaceutical composition to the respiratory system of said subject is effected when the subject shows an inflammation of the respiratory system. Further, the present disclosure relates to a method for analyzing the effect of a polyribonucleotide on ciliogenesis, wherein said polyribonucleotide encodes a protein involved in and/or required for ciliogenesis.

9. 发明申请

US20190194669A1 UTRs INCREASING THE TRANSLATION EFFICIENCY OF RNA MOLECULES 审中-公开
公开(公告)号：US20190194669A1
公开(公告)日：2019-06-27
申请号：US15739147
申请日：2016-06-30
申请人： Ethris GmbH
发明人： Carsten Rudolph , Manish Kumar Aneja , Mehrije Ferizi , Johannes Geiger
IPC分类号： C12N15/67 , C07K14/47
CPC分类号： C12N15/67 , A61K38/00 , C07K14/47
摘要： Described is an RNA molecule comprising (a) a coding region coding for a polypeptide; and (b) upstream of said coding region one or more UTR(s) comprising the sequence as shown in SEQ ID NO:1 or a sequence which shows 1 to 4 substitutions in comparison to SEQ ID NO:1 and which results in an RNA molecule having the same or a higher translation efficiency as an RNA molecule comprising an UTR comprising SEQ ID NO:1; and/or (c) downstream of said coding region one or more UTR(s) comprising the sequence as shown in SEQ ID NO:2 or a sequence which shows 1 to 7 substitutions in comparison to SEQ ID NO:2 and which results in an RNA molecule having the same or a higher translation efficiency as an RNA molecule comprising an UTR comprising SEQ ID NO:2; wherein said polypeptide encoded by said coding region is not a cytochrome b-245 alpha polypeptide (CYBA). Moreover, described is a nucleic acid molecule encoding the RNA molecule according to the present invention. Further, described is a vector comprising the nucleic acid molecule according to the present invention and to a host cell comprising the vector according to the present invention. Further, described is a pharmaceutical composition comprising the RNA molecule according to the present invention and optionally a pharmaceutically acceptable carrier. Moreover, described is a kit comprising the RNA molecule according to the present invention. Finally, described is the use of one or more UTR(s) as defined in (b) and/or one or more UTR(s) as defined in (c) for increasing the efficiency of translating a coding region of an RNA molecule into a polypeptide or a protein encoded by said coding region.

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