Abstract: The present invention relates to oligonucleotides which up-regulate or restore the expression of progranulin in cells, and their use in the treatment of neurological disorders, and disorders associated with progranulin haploinsufficiency.

Abstract: The present invention relates to splice modulating oligonucleotides (oligomers) that are complementary to the CARD9 pre-mRNA, for inhibiting the inclusion of a CARD9 exon 11 in CARD9 mRNA. The oligonucleotides of the invention are useful in the treatment of inflammatory diseases such as IBD.

Abstract: The present invention relates to antisense oligonucleotides that are complementary to mammalian CD73 (NT5E) pre-mRNA, wherein the antisense oligonucleotides are capable of modulating the splicing of mammalian CD73 pre-mRNA exon 7. Splice modulation of mammalian CD73 exon 7 is beneficial for a range of medical disorders, including disorders in the field of immune-oncology.

Abstract: The present invention relates to an oligonucleotide comprising at least one phosphorodithioate internucleoside linkage of formula (I) (I) as defined in the description and in the claim. The oligonucleotide of the invention can be used as a medicement.

Abstract: The present invention relates to oligonucleotides that upregulate or restore the expression of progranulin in cells by targeting the promoter region of the progranulin gene. The invention further relates to pharmaceutical compositions and methods for the treatment of diseases associated with progranulin, specifically progranulin haploinsufficiency and neurological disorders.

Abstract: The invention relates to the field of immunotherapy and vaccine treatment of diseased cells via enhancing the immune response to the diseased cells. In the context of the present invention this is done by engineering neo-antigens in cells via oligonucleotide mediated production of aberrant RNA transcripts which, when transcribed in the cell, result in the generation or increased expression of aberrant polypeptides. Extracellular display of these polypeptides, of peptide fragments derived provides antigen epitopes (neoantigen) for detection by the immune system.

Abstract: The present invention relates to antisense oligonucleotides which are complementary to conserved TDP-43 binding sites on pre-mRNA transcripts, which are capable of restoring RNA binding protein function in the processing of multiple independent mRNAs in TDP-43 depleted cells.

Abstract: The invention relates to the field of therapeutic oligonucleotide analytics, and provides methods for primer based parallel sequencing of therapeutic oligonucleotides which provide sequence based quality information which may be used in conjunction with or in place of present chromatography or mass spectroscopic methods, and may be used, for example, in oligonucleotide therapeutic discovery, manufacture, quality assurance, therapeutic development, and patient monitoring.

Abstract: The present invention relates to oligonucleotides which up-regulate or restore the expression of progranulin in cells, and their use in the treatment of neurological disorders, and disorders associated with progranulin haploinsufficiency.

Abstract: The invention relates to the field of therapeutic oligonucleotide analytics and discovery, and provides methods for primer based parallel sequencing of modified oligonucleotides which provide sequence based quality information which may be used in oligonucleotide therapeutic discovery, manufacture, quality assurance, therapeutic development, and patient monitoring.

Abstract: The invention relates to the field of therapeutic oligonucleotide analytics and discovery, and provides methods for primer based parallel sequencing of modified oligonucleotides which provide sequence based quality information which may be used in oligonucleotide therapeutic discovery, manufacture, quality assurance, therapeutic development, and patient monitoring.

Abstract: The present invention relates to an oligonucleotide comprising at least one phosphorodithioate internucleoside linkage of formula (I) (I) as defined in the description and in the claim. The oligonucleotide of the invention can be used as a medicament.

Abstract: The present invention relates to the detection of therapeutic modified oligonucleotides in biological samples and an adaptor oligonucleotide (capture probe) which enables a quantitative PCR based detection method and the sequencing of modified oligonucleotides. The invention provides novel adaptor probes for use in detecting therapeutic oligonucleotides and for in vivo discovery of preferred therapeutic oligonucleotide sequences.