Planegg, Germany
Planegg, Germany

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Patent
ethris GmbH | Date: 2017-01-04

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.


Grant
Agency: European Commission | Branch: FP7 | Program: CP-TP | Phase: KBBE.2012.3.3-03 | Award Amount: 7.11M | Year: 2012

The European Life science and chemical industries increasingly depend on effi-cient, sustainable, and cost-effective bioprocessing platforms to remain competi-tive. A critical assessment of current bottlenecks during (bio) manufacturing clearly indicates that the recovery and purification of biologicals in large scale in responsible for many inefficiencies. INTENSO proposes an evaluation of the current situation of the downstream processing scenario with the aim of identifying inefficiencies and concomitantly introduce a debottlenecking overarching strategy. The later will be build up on the basis of a multidisciplinary approach, which considers opportunities to im-prove the process technology and underlying chemistry / biology and materials science at the same time. INTENSO will work alongside 4 technological axes, targeting promising and up-coming technologies and tailoring such technologies to the manufacturing of various classes of (bio) products. Intensification of individual unit operations and global process integration, as well as, dovetailing with fermentation / cell cultivation will be employed to the mentioned end. INTENSO will target new classes of (bio) products like Monoclonal Antibodies (Mabs), pDNA (e.g. for genetic vaccination), Virus Like Particles (VLP) or nano-plexes. All the mentioned new products are part of most industrial R&D pipelines and offer an excellent opportunity to introduce innovative bioprocessing. The results of the project are expected to contribute to the understanding of current industrial downstream processing practice, to the definition and alleviation of current inefficiencies, to the development and / or implementation of novel technologies, and to more efficient / sustainable and cost effective (bio) manufacturing. Various technologies will be studied utilizing a nano-to-process strategy so as to introduce integration / intensification during bioprocessing.


Patent
Shire Inc and Ethris GmbH | Date: 2015-10-06

Materials, formulations, production methods, and methods for delivery of CFTR mRNA for induction of CFTR expression, including in the mammalian lung are provided. The present invention is particularly useful for treating cystic fibrosis.


The invention relates to a polyribonucleotide with a sequence that codes a protein or protein fragment, wherein the polyribonucleotide comprises a combination of unmodified and modified nucleotides, wherein 5 to 50% of the uridine nucleotides and 5 to 50% of the cytidin nucleotides are modified uridine nucleotides or modified cytidin nucleotides.


The invention relates to a polyribonucleotide with a sequence that codes a protein or protein fragment, wherein the polyribonucleotide comprises a combination of unmodified and modified nucleotides, wherein 5 to 50% of the uridine nucleotides and 5 to 50% of the cytidin nucleotides are modified uridine nucleotides or modified cytidin nucleotides.


The invention relates to a polyribonucleotide with a sequence that codes a protein or protein fragment, wherein the polyribonucleotide comprises a combination of unmodified and modified nucleotides, wherein 5 to 50% of the uridine nucleotides and 5 to 50% of the cytidine nucleotides are modified uridine nucleotides or modified cytidine nucleotides.


Patent
Ethris Gmbh | Date: 2014-06-27

The present invention relates to oligomers, polymers and lipidoids comprising characteristic oligo(alkylene amine) moieties which are useful as vehicles for transfecting a cell with RNA. The present invention furthermore relates to a composition comprising at least a nucleic acid and an oligomer or polymer or a lipidoid comprising such oligo(alkylene amine) moieties and to a method of transfecting a cell using said composition. Furthermore, the present invention relates to pharmaceutical compositions and uses.


The invention relates to a polyribonucleotide with a sequence that codes a protein or protein fragment, wherein the polyribonucleotide comprises a combination of unmodified and modified nucleotides, wherein 5 to 50% of the uridine nucleotides and 5 to 50% of the cytidin nucleotides are modified uridine nucleotides or modified cytidin nucleotides.


Patent
Ethris GmbH | Date: 2016-06-22

The present invention relates to polymers comprising a characteristic combination of alkylene amine moieties which are useful as vehicles for transfecting a cell with a nucleic acid. The present invention furthermore relates to a composition comprising a nucleic acid and such a polymer, and to a method of transfecting a cell using said composition. Furthermore, the present invention relates to pharmaceutical compositions and uses.


Patent
Ethris Gmbh | Date: 2014-03-24

Described is a conjugate of agent complex and at least one target-finding ligand, where the agent complex comprises an agent encapsulated by an encapsulation material and where the target-finding ligand is a prostacyclin analog, and the use of the conjugate.

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