Cytos Biotechnology Ag

Other

Synthena Ag

Novartis Pharma Ag

Cytos Biotechnology

Wolfgang Andreas Renner

Martin F Bachmann

James E Bailey

Alain Tissot

Franziska Lechner

Peter Sebbel

Frank Hennecke

Lars Nieba

Branislav Dugovic

Christine Piossek

Georg H Orberger

Daniel Koller

Patrick Maurer

Patrik Maurer

Paul Pumpens

Matthias Staufenbiel

Indulis Cielens

Regina Renhofa

Martin Fussenegger

Vania Manolova

Rainer Ortmann

Sebastian Fuchs

Peter Frey

Conrad Coester

Rainer Lūönd

Klaus Dietmeier

Reto Bertolini

Marco Boorsma

Nicolas Dunant

Yu Zou

Hans M Eppenberger

Rainer Luond

The present invention relates to a composition comprising an oligomeric compound comprising one or more tricyclo-deoxyribonucleic acid (tc-DNA) nucleosides and one or more lipid moiety, wherein said one or more lipid moiety is covalently linked to said oligomeric compound either directly or via a spacer, and wherein preferably said oligomeric compound comprises from 5 to 40 monomer subunits, as well as pharmaceutical compositions thereof and their uses in the prevention or treatment of neuromuscular or musculoskeletal diseases such as Duchenne muscular dystrophy or Steinert disease.

Modified oligomeric compounds comprising tricyclo-DNA nucleosides and uses thereof

In some embodiments, disclosed herein are oligomeric compounds which include one or more tricyclo-deoxyribonucleic acid (tc-DNA) nucleosides and one or more 2'-modified ribonucleic acid (2&#x2032;-modified-RNA) nucleosides, and which optionally also include one or more non-nucleotides, each of which is joined by a plurality of internucleoside linkages, including pharmaceutical compositions and methods of using the pharmaceutical compositions for the treatment of diseases including Duchenne muscular dystrophy treatment of familial dysautonomia, spinal muscular atrophy, ataxia telangiectasia, congenital disorder of glycosylation, fronto-temporal dementia, Parkinsonism linked to chromosome 17, Niemann-Pick disease type C, neurofibromatosis type 1, neurofibromatosis type 2, megalencephalic leukoencephalopathy with subcortical cysts type 1, Pelizaeus-Merzbacher disease, Pompe disease, myotonic dystrophy type 2 (DM2 or proximal myotonic myopathy), and myotonic dystrophy type 1 (DM1 or Steinert disease).

Mixed tricyclo-DNA, 2'-modified RNA oligonucleotide compositions and uses thereof

The application is related to compositions and methods for the treatment of hypersensitivity, wherein the compositions comprise a particle packaged with immunostimulatory nucleic acids. The compositions of the invention are particularly useful in the treatment of atopic eczema, asthma and IgE-mediated allergy (type I allergy), especially pollen allergy and house dust allergy.

Immunostimulatory nucleic acid packaged particles for the treatment of hypersensitivity

The present invention is related to the fields of molecular biology, virology, immunology and medicine. The invention provides a composition comprising an ordered and repetitive antigen or antigenic determinant array. The invention also provides a process for producing an antigen or antigenic determinant in an ordered and repetitive array. The ordered and repetitive antigen or antigenic determinant is useful in the production of vaccines for the treatment of infectious diseases, the treatment of allergies and as a pharmaccine to prevent or cure cancer and to efficiently induce self-specific immune responses, in particular antibody responses.

Molecular antigen array

The invention provides compositions and processes for the production of ordered and repetitive antigen or antigenic determinant arrays. The compositions of the invention are useful for the production of vaccines for the prevention of infectious diseases, the treatment of allergies and the treatment of cancers. Various embodiments of the invention provide for a virus, virus-like particle, viral capsid particle, phage or recombinant form thereof coated with any desired antigen in a highly ordered and repetitive fashion as the result of specific interactions. In one specific embodiment, a versatile new technology based on a cassette-type system (AlphaVaccine Technology) allows production of antigen coated viral particles. Other specific embodiments allow the production of antigen coated hepatitis B virus-like particles or antigen coated Measles virus-like particles.

Ordered molecular presentation of antigens, method of preparation and use

The present invention provides novel expression vectors which permit tight regulation of gene expression in eucaryotic cells. More specifically, the invention provides DNA vectors comprising nucleotide sequences that are transcribed to form RNA molecules which are then replicated by a temperature-sensitive replicase to form additional RNA molecules. The RNA molecules produced by replication contain a nucleotide sequence which may be translated to produce a protein of interest or which encode one or more untranslated RNA molecules. Also provided are methods for producing heterologous proteins and untranslated RNA molecules. Further provided are methods for administering heterologous proteins and untranslated RNA molecules to individuals. In addition, pharmaceutical compositions are provided comprising the DNA and RNA molecules of the invention and a pharmaceutically acceptable carrier.

Inducible alphaviral gene expression system

The invention provides compositions and processes for the production of ordered and repetitive antigen or antigenic determinant arrays. The compositions of the invention are useful for the production of vaccines for the prevention of infectious diseases, the treatment of allergies and the treatment of cancers. Various embodiments of the invention provide for a core particle that is coated with any desired antigen in a highly ordered and repetitive fashion as the result of specific interactions.

The present invention relates to methods for the modification of genomes of eukaryotic cells to alter the expression of endogenous genes. The invention also relates to recombinant eukaryotic host cells and polypeptides produced by the practice of the disclosed methods. The invention further relates to vector systems useful for modifying the genomes of eukaryotic cells to alter the expression of endogenous genes.

Replicon based activation of endogenous genes

The present invention is directed to a method for detection, characterization and isolation of nucleic acids encoding proteins of a desired property, such as a particular cellular localization. The invention further provides for rapid expression of such proteins or glycoproteins in mammalian cells and for facilitated purification of the novel secreted proteins or glycoproteins. Further, the invention provides for radioactive labelling of the novel proteins or glycoproteins, for rapid identification of sites of binding including animals and for rapid production of infective viral vectors for use in gene transfer.

Expression cloning processes for the discovery, characterization and isolation of genes encoding polypeptides with a predetermined property

Provided by the invention are novel methods, vectors and cells for the recombinant production of desired gene products. In particular, the invention relates to increased production of desired gene products by inducibly arresting cell proliferation. The invention also provides novel multicistronic expression vectors that are useful not only for recombinant gene expression, but also for other applications such as gene therapy, tissue engineering and metabolic engineering.

Cytostatic process increases the productivity of cultured cells

Expression cloning processes for the discovery characterization, and isolation of genes encoding polypeptides with a predetermined property

Cell cultures are described which proliferate in a serum- and protein-free environment and which are characterized by the circumstance that the concentration of at least one cell-cycle-regulatory protein is increased during at least one period of the cell-cycle. Methods for the production of such cell cultures are presented. With these methods cell lines can be transferred to serum- and protein-free growth that are already in use in a production process which, prior to the use of these methods, requires serum or proteins added to the basal medium.

Methods for the activation of proliferation of animal cells

Growing community of inventors

Zurich, Switzerland

Wolfgang Andreas Renner