Abstract: The present invention provides new adeno-associated virus-derived vectors and pharmaceutical compositions containing the same for the treatment of lysosomal storage disorders and specially, for the treatment of mucopolysaccharidoses Type IIID.

Abstract: The present invention relates to lipid particles comprising at least one cationic lipid, at least one water-soluble therapeutically effective compound and RNA. Further, the present invention relates to a pharmaceutical composition comprising such particles. Said pharmaceutical composition is useful for inducing an immune response. It is also useful in a prophylactic and/or therapeutic treatment of a disease involving an antigen. Furthermore, the present invention relates to a method for producing the particles.

Abstract: The invention relates to a method for manufacturing an elastic and flexible fiber with a pre-de-termined non-circular cross-sectional geometry, the method comprising extrusion of an elasto-mer from a nozzle onto a substrate, wherein the pre-determined non-circular cross-sectional geometry of the fiber is determined by the height and velocity of the nozzle relative to the sub-strate. The invention relates to an elastic and flexible fiber produced using the method, wherein the fiber comprises an elongated indentation along a length of the fiber (groove). The invention relates to methods for producing preferably biocompatible microfluidic, electrically conducting or light-guiding fibers using the methods of the invention. The invention further re-lates to elastic and flexible fibers produced by the method.

Abstract: The invention relates to an electrolyte arrangement for a cell having at least one anode (1) and at least one cathode (3) comprising at least three superposed layers (2.1, 2.2, 2.3), wherein the middle layer (2.2) comprises a porous electrically nonconductive structure, and wherein a layer of a polymer-based electrolyte (2.1, 2.3) is arranged on both opposite sides of the porous electrically nonconductive structure, wherein at least one of the superposed layers (2.1, 2.2, 2.3) contains a ceramic material, wherein the ceramic material of the middle layer (2.2) is selected from metal ion-conductive ceramic material, a ceramic material which does not conduct metal ions, and/or mixtures thereof, and the ceramic material of the polymer-based electrolyte layer(s) (2.1, 2.3) is a metal ion-conductive ceramic material.

Abstract: The present invention relates to a novel method for producing fluorinated cycloaliphatic compounds from the analogous aromatic compounds by hydrogenation with an Rh-carbene catalyst system.

Abstract: Disclosed are compounds represented by the following general formula (I): Cyclo-(Arg-Gly-Asp-X1-X2-X3-X4-X5) (I) wherein the variables groups X1 to X5 have the following meanings X1: Leu, Ile, Nle, Val, Tyr, Phe; X2: D-amino acid such as D-Pro, N-Me-D-Phe; X3: Pro, N-Me-amino acid such as N-Me-Lys, N-Me-Lys(Ac); Pro-Rx3, N-Me-Lys-Rx4; X4: Gly, Ala, Ser, Thr; X5: Leu, Ala, Tyr, His, Ile, Nle, Val, Phe wherein Pro-Rx3 represents a proline residue that carries at the C-3, C-4 or C-5 carbon atom and preferably the C-4 carbon atom a functional group selected from —NH2, —OH, —NH—Ac, —NH-hexyne, and wherein Lys-Rx4 represents a lysine residue, wherein the ?-amino nitrogen atom carries a group of the formula -L4-R4, wherein L4 is selected from the group consisting of covalent bond, —C(O)—, and —C(O)—O—, . . .

Abstract: Provided is a method of preventing, treating or delaying progression of a disease involving aberrant fibroblast proliferation. The method involves using a compound that reduces the level of tyrosine phosphatase activity effected by the protein EYA1A in the parenchymal organ, a nucleic acid ligase IV inhibitor, or an antisense oligonucleotide against the p53-binding protein 1 (53BP1).

Abstract: The present invention relates to a nucleotide-modified messenger RNA for the permanent correction of a genetic alteration on a DNA. The invention further relates to a nucleotide-modified messenger RNA in combination with a repair template. It also relates to a pharmaceutical composition. It finally relates to methods for the correction of a genetic alteration on a DNA.

Abstract: An implantable fluid pump system is disclosed for supporting or initiating flow inside a hollow organ through which fluid circulates, in particular the heart. The fluid pump system comprises an intracardiac module, which includes two separate fluid channels, each of which possesses an intracardiac fluid channel opening and, located opposite the latter, an extracardiac fluid channel opening, a fastening module, which provides a joining contour for purposes of joining onto the intracardiac module in a fluid-tight manner, and a fastening structure for purposes of intracorporeal fastening onto the hollow organ, and a pump module, which can be mounted in a releasable manner directly or indirectly onto the intracardiac module, and can be attached in a fluid-tight manner to the extracardiac fluid channel openings in order to produce a fluid-tight connection of both fluid channels.

Abstract: A method of determining a modified shape of a computer-aided design model of an object or a partition thereof is described. With the method, at least two reference landmarks are placed at a reference position with respect to the shape of the object or to the partition. An adjustment landmark is placed at the reference position of the corresponding reference landmark. The adjustment landmarks are displaced from the reference position to a modified position, and a displacement between the modified position and the reference position is obtained. The modified shape of the object or the partition is generated by modifying the shape of the object or the partition by applying a homogeneous function of the displacement to the computer-aided design model of the shape of the object or the partition.

Abstract: A detector and a method for tracking an arrival time of single particles in an ion beam are disclosed, wherein the single particles are provided as a bunch of ions by a synchrotron. Herein, the detector comprises a detector segment comprising a scintillating material, the scintillating material being designated for generating radiation upon passing of a single particle comprised by the bunch of ions through the scintillating material, wherein the scintillating material comprises a plurality of scintillating fibers, the scintillating fibers being provided as a fiber layer, wherein the fiber layer is located perpendicularly with respect to a direction of the incident ion beam; at least one detector element, the detector element being designated for generating a detector signal from the radiation; and at least one evaluation device, the evaluation device being designated for determining information about the single particles from the detector signals provided by the at least one detector element.

Abstract: The present invention provides a solid oral pharmaceutical composition comprising a pharmaceutically effective amount of living microorganisms and one or more pharmaceutically acceptable water absorbing excipient(s), wherein the composition has a water content, determined according to European Pharmacopoeia 9.4, section 2.5.12., from 0.5 to 30% with respect the total weight of the composition. The invention also provides processes for its preparation as well as it use in therapy. The live-cell based composition of the invention is stable at mild conditions.

Abstract: The Sleeping Beauty (SB) transposon is an efficient non-viral tool for inserting transgenes into cells. Its broad utilization in gene therapy has been hampered by uncontrolled transposase gene activity and the inability to use transposase protein directly. The present invention concerns the finding that SB transposase spontaneously penetrates mammalian cells and can be delivered with transposon DNA to gene-modify various cell lines, embryonic, hematopoietic and induced pluripotent stem cells. The invention provides methods and compounds to apply the cell penetrating function of transposase in methods of genetically engineering cells as well as using the transposase as a shuttle for delivering cargo into a target cell or even into a target cell organelle. Genomic integration frequency can be titrated using the technology of the invention, which adds an additional layer of safety, opening opportunities for advanced applications in genetic engineering and gene therapy.

Abstract: The present invention provides methods for the treatment of a subject having a Hepatitis B virus (HBV) infection, e.g., a chronic HBV infection, using a combination of an RNAi agent that targets HBV and an HBV vaccine. It is disclosed a RNAi agent and an HBV vaccine for use in treatment of HBV infection, comprising sequentially administering to the subject having an HBV infection: a) an RNAi agent that inhibits expression of at least three HBV transcripts, wherein the RNAi agent forms a double stranded region; b) a protein-based vaccine comprising a first HBV core antigen (HBcAg) polypeptide, and a first HBV surface antigen (HBsAg) polypeptide; and c) a nucleic acid-based vaccine comprising an expression vector construct encoding a second HBcAg polypeptide, and/or a second HBsAg polypeptide, wherein the second HBcAg polypeptide, and/or the second HBsAg polypeptide, shares at least one epitope with at least one of the first HBcAg polypeptide, and/or the first HBsAg polypeptide.

Abstract: A needle assembly (10) for relieving a pneumothorax and/or a hemothorax, the needle assembly (10) comprising a canula (12), the canula (12) comprising: a distal end portion (20) comprising a cutting end (22) for puncturing a thoracic cavity wall (24), a proximal end portion (14) for removing a fluid or gas from a thoracic cavity (28), a first curved portion (18) between the distal end portion (20) and the proximal end portion (14), an intermediate portion (16) between the first curved portion (18) and the proximal end portion (14), and a first fixing bulge (42) arranged between the first curved portion (18) and the intermediate portion (16), wherein an outer diameter of the canula (12) at the first fixing bulge (42) is increased with respect to the diameter (D1) of the intermediate portion (16), said first fixing bulge (42) being arranged such as to be placed at or close to the inner surface (24) of the thoracic cavity wall (24i), when the canula (12) is inserted for relieving the pneumothorax, such as to pre

Abstract: Described is a parvovirus formulation which comprises (a) at least 1×109 pfu/ml of parvovirus H1 (H-1PV) or a related rodent parvovirus such as LuIII, Mouse minute virus (MMV), Mouse parvovirus (MPV), Rat minute virus (RMV), Rat parvovirus or Rat virus (RV) and (b) a pharmaceutically acceptable carrier containing 40-50% Iodixanol (w/v), 0.7-0.9 mmol CaCl2×2 H2O, 50-60 mmol NaCl, 0.9-1.2 mmol KCl, 0.7-0.95 mg/ml Tromethamine and 0.05-0.15 mg/ml Edetate calcium disodium. A preferred use is the therapy of a brain tumour by intratumoral injection.

Abstract: The invention relates to a bispecific HER2-targeting agent that includes (a) a first polypeptide ligand that binds to HER2 extracellular domain 1, (b) a second polypeptide ligand that binds to HER2 extracellular domain 4, and (c) a linker covalently attaching said first polypeptide ligand to said second polypeptide ligand.

Abstract: The invention relates to nucleic acid products that interfere with or inhibit PROS1 gene expression. It further relates to therapeutic uses of PROS1 inhibition for the treatment of bleeding disorders.

Abstract: Tryptophan degradation is a key metabolic pathway controlling immune reactions and evidence suggests that during cancer progression generation of tryptophan metabolites may be fundamental for immune escape promoting the malignant phenotype of cancer cells in an autocrine fashion. The present invention relates to methods of measuring mass tag labelled tryptophan and metabolites thereof and methods using the labelled molecules for monitoring in a subject the effectiveness of a treatment and of disease recurrence after treatment, for stratifying patients and for diagnosing suppression of an immune response in a subject.

Abstract: The present invention relates to compositions comprising polyplex formulations for delivery of RNA to a target organ or a target cell after parenteral administration, in particular after intramuscular administration. More precisely, the present invention relates to formulations for administration of RNA such as self-replicating RNA, in particular by intramuscular injection. In more detail, the formulations comprise polyplex particles from single stranded RNA and a polyalkyleneimine. The RNA may encode a protein of interest, such as a pharmaceutically active protein. Furthermore, the present invention relates to pharmaceutical products, comprising said RNA polyplex formulations for parenteral application to humans or to animals. The present invention relates as well to manufacturing of such pharmaceutical products, comprising, optionally, steps of sterile filtration, freezing and dehydration.