Ethris Gmbh

Translate Bio, Inc.

Shire Human Genetic Therapies, Inc.

Rana Therapeutics, Inc.

Christian Plank

Carsten Rudolph

Michael W Heartlein

Frank DeRosa

Braydon Charles Guild

Lianne Smith

Manish Kumar Aneja

Ludwig Weiss

Christian Dohmen

Frank Derosa

Johannes Geiger

Rebekka Kubisch-Dohmen

Mehrije Ferizi

Christian Koch

Elizabeth Balmayor

Anna Macht

Thomas Langenickel

Zeljka Trepotec

Kristin Lohmer

Christian Bergemann

The present invention relates to a pharmaceutical composition comprising a polyribonucleotide (RNA) with a sequence which encodes a bone morphogenetic protein (BMP) for use in (i) treating or preventing a bone disease, bone disorder or bone injury; and/or (ii) inducing or enhancing osteogenic differentiation, osteogenesis, ossification, bone regeneration and/or bone morphogenesis in a patient. The present invention also relates to the respective BMP encoding RNAs (BMP RNAs), in particular in its chemically modified form. The present invention also relates to complexes which comprise or are complexed with the BMP RNA, in particular to the respective transfection complexes like lipofection, magnetofection and magnetolipofection complexes. The present invention further relates to a carrier and carrier body to which the RNA or complex has been loaded and to a pharmaceutical composition comprising said carrier or carrier body. The present invention further relates to a matrix or scaffold for sustained mRNA delivery and its application in bone regeneration in vivo, ex vivo and in vitro.

Induction of osteogenesis by delivering BMP encoding RNA

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.

Compositions for introducing RNA into cells

Described are DNA molecules which can be transcribed into an mRNA harbouring novel UTR sequences combining the advantages of being extremely short and at the same time allowing for high translation efficiencies of RNA molecules containing them. Further, described are vectors comprising such a DNA molecule and to host cells comprising such a vector. Moreover, described are corresponding RNA molecules containing such UTRs. Further, described is a pharmaceutical composition comprising the described RNA molecule and optionally a pharmaceutically acceptable carrier as well as to the use of the described UTRs for translating a coding region of an RNA molecule into a polypeptide or a protein encoded by said coding region.

Minimal UTR sequences

The present disclosure relates to materials, formulations, production methods, and methods for delivery of CFTR mRNA, including but not limited to chemically modified mRNA for induction of CFTR expression, including in the mammalian lung. The present invention is particularly useful for treating cystic fibrosis, but is also useful in the treatment of diseases related to CFTR gene.

CFTR MRNA compositions and related methods and uses

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.

ATP-binding cassette family coding polyribonucleotides and formulations thereof

Described are DNA molecules which can be transcribed into an mRNA harbouring novel UTR sequences combining the advantages of being extremely short and at the same time allowing for high translation efficiencies of RNA molecules containing them. Further, described are vectors comprising such a DNA molecule and to host cells comprising such a vector. Moreover, described are corresponding RNA molecules containing such UTRs. Further, described in a pharmaceutical composition comprising the described RNA molecule are optionally a pharmaceutically acceptable carrier as well as to the use of the described UTRs for translating a coding region of an RNA molecule into a polypeptide or a protein encoded by said coding region.

Compositions comprising mRNA formulated for pulmonary administration and related method 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.

Pulmonary delivery of mRNA to non-lung target cells

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.

Compositions for introducing nucelic acid into cells

In certain aspects, the disclosure relates to compositions comprising modified Cas9 polyribonucleotides and methods of use, as well as Cas9 polynucleotides and polyribonucleotides.

Cas9 mRNAs

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.

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.

Pharmaceutical compositions comprising an mRNA-loaded nanoparticle, wherein the mRNA is an in vitro transcribed mRNA and has a coding sequence at least 80% identical to SEQ ID NO: 3, and wherein the mRNA encodes a human cystic fibrosis transmembrane conductance regulator (CFTR) protein comprising the amino acid sequence of SEQ ID NO:1 are provided. The present invention is particularly useful for treating cystic fibrosis.

CFTR mRNA compositions and related methods and uses

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.

Growing community of inventors

Wessling, Germany

Christian Plank