Abstract: The present invention relates to engineered extracellular vesicles (EVs) as a therapeutic modality for the treatment of various severe diseases. More specifically, the invention relates to a novel approach to manufacturing engineered EVs which extends their half-life and alters their biodistribution, resulting in a novel therapeutic modality able to carry various types of drugs suitable for application in not only genetic diseases but more broadly across essentially all therapeutic areas.

Abstract: The present invention pertains to extracellular vesicles (EVs), wherein the EVs comprise domains present on the surface of the EV, which modify the circulation time of such EVs. Such EVs display improved pharmacokinetics and are therefore useful as therapeutics. Such EVs may also be useful for affinity purification of the EV drug substance.

Abstract: The present invention pertains to a new nanoparticle-like delivery system for intracellular delivery of cargo molecules such as nucleic acids, ribonucleoproteins and extracellular vesicles.

Abstract: The present invention pertains to an inventive release mechanism for extracellular vesicle (EV)-mediated intracellular and intramembrane delivery of therapeutic polypeptides. More specifically, the invention relates to EVs comprising polypeptide constructs which comprise a therapeutic polypeptide releasably attached to an exosomal polypeptide. Furthermore, the present invention pertains to manufacturing methods, pharmaceutical compositions, medical uses and applications, and various other embodiments related to the inventive EVs.

Abstract: The present invention pertains to inter alia methods for purifying extracellular vesicles including exposing a sample comprising at least one extracellular vesicle to ultrafiltration; and exposing the sample following the ultrafiltration in step (i) to size exclusion liquid chromatography.

Abstract: The present invention pertains to inter alia methods for purifying extracellular vesicles including exposing a sample comprising at least one extracellular vesicle to ultrafiltration; and exposing the sample following the ultrafiltration in step (i) to size exclusion liquid chromatography.

Abstract: The present invention pertains to an inventive release mechanism for extracellular vesicle (EV)-mediated intracellular and intramembrane delivery of therapeutic polypeptides. More specifically, the invention relates to EVs comprising polypeptide constructs which comprise a therapeutic polypeptide releasably attached to an exosomal polypeptide. Furthermore, the present invention pertains to manufacturing methods, pharmaceutical compositions, medical uses and applications, and various other embodiments related to the inventive EVs.

Abstract: The present invention pertains to inter alia therapeutic delivery vesicles, for instance exosomes or microvesicles, comprising polypeptide constructs, methods for producing said therapeutic delivery vesicles, pharmaceutical compositions and medical uses thereof. The therapeutic polypeptide constructs comprised in the extracellular delivery vesicles enable sequestering target molecules of interest, to treat e.g. neuro-inflammatory diseases and cancer.

Abstract: The present invention relates to exosomes, loaded with biotherapeutic protein and/or peptide and methods of producing them and to the use of such exosomes for delivering protein and/or peptide in vivo, in particular the use of such exosomes in methods of therapy.

Abstract: the present invention relates to peptides, in particular cell-penetrating peptides, having a first hydrophobic domain positioned at the C-terminus of the peptide and a second hydrophobic domain positioned at the N-terminus of the peptide, and to conjugates of such cell-penetrating peptides with a therapeutic molecule. The present invention further relates to use of such peptides or conjugates in methods of treatment or as a medicament, especially in the treatment of genetic disorders and in particular muscular dystrophies such as Duchenne muscular dystrophy.

Abstract: The present invention relates to exosomes, loaded with biotherapeutic protein and/or peptide and methods of producing them and to the use of such exosomes for delivering protein and/or peptide in vivo, in particular the use of such exosomes in methods of therapy.

Abstract: The present invention relates to administration of gene therapy, in particular the development of effective combinatorial strategies for gene delivery in inter alia monogenetic diseases. More specifically, the present invention relates to a combination therapy, methods of producing the combination therapy, as well as various related embodiments.

Abstract: The present invention relates to compositions and buffers for storing engineered extracellular vesicles (EVs), such as exosomes, for extended time periods with maintained stability and function. The present invention allows for both optimized long-term storage and for clinical application of the stored EVs.

Abstract: The present invention pertains to inter alia therapeutic delivery vesicles, for instance exosomes or microvesicles, comprising polypeptide constructs, methods for producing said therapeutic delivery vesicles, pharmaceutical compositions and medical uses thereof. The therapeutic polypeptide constructs comprised in the extracellular delivery vesicles enable sequestering target molecules of interest, to treat e.g. neuro-inflammatory diseases and cancer.

Abstract: The present invention pertains to an inventive release mechanism for extracellular vesicle (EV)-mediated intracellular and intramembrane delivery of therapeutic polypeptides. More specifically, the invention relates to EVs comprising polypeptide constructs which comprise a therapeutic polypeptide releasably attached to an exosomal polypeptide. Furthermore, the present invention pertains to manufacturing methods, pharmaceutical compositions, medical uses and applications, and various other embodiments related to the inventive EVs.

Abstract: The present invention pertains to inter alia therapeutic delivery vesicles, for instance exosomes or microvesicles, comprising polypeptide constructs, methods for producing said therapeutic delivery vesicles, pharmaceutical compositions and medical uses thereof. The therapeutic polypeptide constructs comprised in the extracellular delivery vesicles enable sequestering target molecules of interest, to treat e.g. neuro-inflammatory diseases and cancer.

Abstract: The present invention relates to exosomes comprising a transferrin targeting moiety on their surface, methods of producing them and to the use of such exosomes for delivering genetic material and/or biotherapeutic proteins or peptides or chemotherapeutic agents in vivo, in particular the use of such exosomes in methods of gene therapy or gene silencing, or delivery of other therapeutic agents.

Abstract: The present invention relates to a system for intracellular delivery of a cargo comprising at least one component A chosen from aliphatic linear or branched moieties with at least 4 carbon atoms and/or cyclic ring systems comprising 2-4 rings which may contain several hetero atoms chosen from N, S, O and P, wherein component(s) A is (are) attached to a cell penetrating peptide B and/or a non-peptide analogue thereof. It also relates to methods of using the system in diagnosis of diseases, as research tool and as a targeting system, a composition comprising the system and especially a pharmaceutical composition a material covered with the system and a material having the delivery systems into the material. Finally it relates to novel peptides.

Abstract: Viral vectors are potential tools for eliminating the viability of eukaryotic cells in anti-cancer therapies since they can efficiently destroy the cancer cells and trigger an immune response against tumours. Typically viruses are not specific to cancer cells and all methods known in art aiming to the construction of cancer-specific viruses suffer from serious problems. The present invention presents a universal method to overcome these problems and is usable for any DNA virus replicating in nucleus or for any layered vector of RNA viruses. In this method the viral gene expression and/or replication will be blocked by the introduction of one or more aberrantly spliced introns into crucial gene expression units of the virus or vector. Lethal effect of these mutations is reverted in a controlled manner by the delivery of splice-switch oligonucleotide (s) correcting the introduced defects and restoring the biological functionality of the virus or vector, including cytolytic properties.

Abstract: The present invention relates to a system for intracellular delivery of a cargo comprising at least one component A chosen from aliphatic linear or branched moieties with at least 4 carbon atoms and/or cyclic ring systems comprising 2-4 rings which may contain several hetero atoms chosen from N, S, O and P, wherein component(s) A is (are) attached to a cell penetrating peptide B and/or a non-peptide analogue thereof. It also relates to methods of using the system in diagnosis of diseases, as research tool and as a targeting system, a composition comprising the system and especially a pharmaceutical composition a material covered with the system and a material having the delivery systems into the material. Finally it relates to novel peptides.