Characterization of a HIR-Fab-IDS, Novel Iduronate 2-Sulfatase Fusion Protein for the Treatment of Neuropathic Mucopolysaccharidosis Type II (Hunter Syndrome).

Background: Mucopolysaccharidosis type II is a severe lysosomal storage disease caused by deficient activity of the enzyme iduronate-2-sulfatase. The only medicinal product approved by the US Food and Drug Administration for enzyme replacement therapy, recombinant iduronate-2-sulfatase (idursulfase, Elaprase), is a large molecule that is not able to cross the blood-brain barrier and neutralize progressive damage of the central nervous system caused by the accumulation of glycosaminoglycans. Novel chimeric protein HIR-Fab-IDS is an anti-human insulin receptor Fab fragment fused to recombinant modified iduronate-2-sulfatase.

Analytical and functional similarity of biosimilar Elizaria® with eculizumab reference product.

A recombinant humanized monoclonal antibody (mAb) Eculizumab, C5-complement cascade inhibitor, is an important treatment of complement-based diseases recommended by international guidelines. Elizaria® Drug Product (DP), developed by IBC Generium, Russia, is the world's first registered biosimilar of eculizumab (Soliris®, Alexion Pharmaceuticals). Using sensitive state-of-the-art analytical techniques extensive similarity assessment has been conducted to demonstrate the structural and functional similarity of original Soliris® (Eculizumab Reference Product, RP) and the biosimilar Elizaria®, focusing on the physicochemical and biological quality attributes, including those known to affect the mechanisms of action.

Physicochemical and Biological Characterization of rhC1INH Expressed in CHO Cells.

The disfunction or deficiency of the C1 esterase inhibitor (C1INH) is associated with hereditary or acquired angioedema (HAE/AAE), a rare life-threatening condition characterized by swelling in the skin, respiratory and gastrointestinal tracts. The current treatment options may carry the risks of either viral infection (plasma-derived Berinert) or immune reaction (human recombinant C1INH from rabbit milk, Ruconest). This study describes the physicochemical and biological characterization of a novel recombinant human C1 esterase inhibitor (rhC1INH) from Chinese hamster ovary (CHO) cells for the treatment of hereditary angioedema compared to the marketed products Berinert and Ruconest.

A development of high-throughput HPLC method for the polysorbate 80 quantitation in protein therapeutic products.

A quantitative analysis of polysorbate 80 is an essential part of a characterization of therapeutic protein products. There are many methods to perform such type of analysis, including spectrophotometry and HPLC. However, there is no high-throughput method with high sensitivity and accuracy.

Design of a stable cell line producing a recombinant monoclonal anti-TNFα antibody based on a CHO cell line.

Recombinant monoclonal antibodies (mAbs) against tumor necrosis factor alpha are widely used in the biopharmaceutical therapy of autoimmune diseases. Currently, a large number of drugs based on these antibodies are available. Accordingly, the development of these products for the Russian market is an important goal.