Roche

The invention relates to a novel process for the removal of a monomethoxytrityl protecting group from a monomethoxytrityl protected amino linker moiety attached to the 5' or 3'-terminus of an oligonucleotide.

The present invention provides a pharmaceutical composition comprising an effective dosage of at least one anti miR-134 oligonucleotide for administration to a mammal suffering from a disease wherein lowering of the activity of miR-134 is beneficial, wherein single administration of the pharmaceutical composition time interval between each administration is at least 50 days and methods using such composition.

A method of testing tightness between a stopper (3) and a plunger (4) in a syringe (1), wherein the stopper (3) has a distal face (31), a proximal face (32) and an internal cavity (33) opening at the distal face (31), and the syringe (1) has a barrel (2) with a hollow interior, an orifice (21) and an opening (22) opposite to the orifice (21), the method comprising: opening the cavity (33) of the stopper (3) to the proximal face (32), setting the stopper (3) through the opening (22) of the barrel (2) into a hollow interior of the barrel (2) such that the distal face (31) of the stopper (3) is directed towards the plunger (4), coupling a gas detector to the opening (22) of the syringe barrel (2), and providing a gas through the orifice (21; 210; 219) of the barrel (2; 20; 29).

The present invention relates to 7-(4,7-diazaspiro[2.5]octan-7-yl)-2-(2,8-dimethylimidazo[1,2-b]pyridazin-6-yl)pyrido[1,2-a]pyrimidin-4-one for use in the treatment of spinal muscular atrophy (SMA), its pharmaceutical composition to be used in the treatment of SMA, its methods of treatment thereof.

The present invention relates to oligonucleotides that are capable of inducing expression of ubiquitin-protein ligase E3A (UBE3A) from the paternal allele in animal or human neurons. The oligonucleotides target the suppressor of the UBE3A paternal allele by hybridization to SNHG14 long non-coding RNA downstream of SNORD109B. The present invention further relates to pharmaceutical compositions and methods for treatment of Angelman syndrome.

The invention provides methods and compositions for treating locally advanced or metastatic breast cancer and for enhancing immune function in an individual having locally advanced or metastatic breast cancer. The methods comprise administering a PD-1 axis binding antagonist and a taxane.

Herein is reported a method for separating full and empty recombinant adeno-associated virus particles using an anion exchange chromatography step, wherein the method comprises a sequence of a applying a solution comprising empty and/or full rAAV particles to an anion exchange chromatography material inside a chromatography column, a first isocratic step, a first linear gradient, a second isocratic step and a second linear gradient, wherein the empty recombinant adeno-associated virus particles are eluted during the first linear gradient and the full recombinant adeno-associated virus particles are eluted during the second linear gradient. In certain embodiments, the solution applied in the first isocratic step comprises about 65 mM of the buffer substance, about 10 mM of the elution salt, about 2 mM of the salt and has a pH value of about 9.4. In certain embodiments, the solution applied in the second isocratic step comprises about 65 mM of the buffer substance, about 90 mM of the elution salt, about 2 mM of the salt and has a pH value of about 9.4. In certain embodiments, the buffer substance is N-(1,1-Dimethyl-2-hydroxyethyl)-3-amino-2-hydroxypropane sulfonic acid, the elution salt is tetramethyl ammonium chloride, and the salt is magnesium chloride.

The invention relates to novel compounds having the general formula I <img class="EMIRef" id="4a88fbbf-79dd-4ab1-bc05-7669f8350250-ia01" /> wherein R<1>, R<2>, R<3>, R<4>, X<1> and X<2> are as described herein, composition including the compounds and methods of using the compounds.

The invention relates to a method of manufacturing an in vivo analyte sensing device, which is adapted for detecting at least one analyte in a body fluid or tissue and an in vivo analyte sensing device obtainable by said manufacturing method. Further, the present invention relates to a method of manufacturing a sensor base plate and a sensor base plate obtainable by said manufacturing method. The sensor base plate may be used for the manufacture of an in vivo analyte sensing device.

The present invention relates to an anti-CD20 antibody, which is ocrelizumab, for use in methods of treating multiple sclerosis.