Silence Therapeutics Gmbh

Noxxon Pharma Ag

Humboldt-universität Zu Berlin

Tme Pharma Ag

Lucas Bethge

Adrien Weingärtner

Judith Hauptmann

Christian Frauendorf

Sibylle Dames

Stephan Tenbaum

Steffen Schubert

Dmitry Samarsky

David Anthony Rider

Alison Gallafent

Adrien Weingartner

Ute Schaeper

Oliver Seitz

Sven Hainke

Marie Wikström Lindholm

Verena Aumiller

Adrien Weingaertner

Ute Shaeper

Adrien WEINGÄERTNER

The present invention relates to products and compositions and their uses. In particular the invention relates to nucleic acid products that interfere with the LPA gene expression or inhibit its expression for use as treatment, prevention or reduction of risk of suffering cardiovascular disease such as coronary heart disease or aortic stenosis or stroke or any other disorder, pathology or syndrome linked to elevated of Lp(a)-containing particles.

Nucleic acids for inhibiting expression of LPA in a cell

The present invention relates to products and compositions and their uses. In particular the invention relates to nucleic acid products that interfere with target gene expression or inhibit target gene expression and therapeutic uses of such products.

Products and compositions

Nucleic acids for inhibiting expression of a target gene comprising phosphorodithioate linkages

The invention relates to nucleic acid products that interfere with complement component C3 gene expression or inhibit its expression. The nucleic acids are preferably for use as treatment, prevention or reduction of risk of suffering from complement component C3 associated diseases, disorders or syndromes, particularly C3 Glomerulopathy (C3G), Paroxysmal Nocturnal Hemoglobinuria (PNH), atypical Hemolytic Uremic Syndrome (aHUS), Lupus nephritis, IgA nephropathy (IgA N), Cold Agglutinin Disease (CAD), Myasthenia gravis (MG), and Primary Membranous Nephropathy.

Nucleic acids for inhibiting expression of C3 in a cell

The present invention relates to nucleic acids for inhibiting expression of a target gene in a cell, comprising at least one duplex region that comprises at least a portion of a first strand and at least a portion of a second strand that is at least partially complementary to the first strand, wherein said first strand is at least partially complementary to at least a portion of RNA transcribed from said target gene to be inhibited. The first strand of the nucleic acid has a terminal 5' (E)-vinylphosphonate nucleotide that is linked to the second nucleotide in the first strand by a phosphodiester linkage.

SiRNAs with vinylphosphonate at the 5' end of the antisense strand

The present invention relates to products and compositions and their uses. In particular the invention relates to nucleic acid products that interfere with the LPA gene expression or inhibit its expression, preferably for use as treatment, prevention or reduction of risk of suffering cardiovascular disease such as coronary heart disease or aortic stenosis or stroke or any other disorder, pathology or syndrome linked to elevated levels of Lp(a) particles.

The present invention is related to a method for the preparation of a modified nucleic acid molecule comprising a nucleic acid moiety and a non-nucleic acid moiety by reacting a first reactant and a second reactant, wherein the first reactant comprises the non-nucleic acid moiety and a carboxyl group, and wherein the second reactant is an amino-modified nucleic acid molecule comprising the nucleic acid moiety and an amino modification comprising an amino group which is attached to the nucleic acid moiety, wherein the method comprises the following steps: a) activating the first reactant, preferably the carboxyl group of the first reactant, by a condensation reagent in a water miscible organic solvent, and b) reacting the activated first reactant, preferably the activated carboxyl group of the first reactant, of step a) and the second reactant, preferably the amino group of the amino modification of the amino-modified nucleic acid molecule which has been dissolved in water or a mixture of a water miscible organic solvent and water, whereby the modified nucleic acid molecule is formed.

Method for polyalkoxylation of nucleic acids that enables recovery and reuse of excess polyalkoxylation reagent

The present invention relates to products and compositions and their uses. In particular the invention relates to nucleic acid products that interfere with the TMPRSS6 gene expression or inhibits its expression and therapeutic uses such as for the treatment of hemochromatosis, porphyria and blood disorders such as &#x3b2;-thalassemias, sickle cell disease and transfusional iron overload or myelodysplastic syndrome.

A method of obtaining a polyalkoxylated nucleic acid, which can be used to make a modified drug, from a liquid mixture of polyalkoxylated and non-polyalkoxylated nucleic acids is described. The method involves use of a solvent or a mixture of solvents that allow the polyalkoxylated nucleic acids to precipitate out of the solution.

Methods for the preparation of a polyalkoxylated nucleic acid molecule

The present invention relates to forced intercalation probes (FIT-probes) comprising at least one nucleoside analog which comprises at least a fluorescent artificial nucleobase directly bound to a carbon of a modified sugar moiety wherein said modified sugar moiety is a carba-sugar or an amino acid nucleic acid (AANA). Thereby the nucleoside analog is incorporated into DNA or RNA in the place of a single native base.

Growing community of inventors

Potsdam, Germany

Lucas Bethge