Publications by authors named "N N Kulagina"

The analysis of Non-Liquid Matrices (NLMs) can provide key information on many aspects in drug discovery and development. These include but are not limited to drug uptake and distribution, engagement and modulation, and target exposure. A thorough understanding of these aspects is fundamental to the progression of drug development.

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Aberrant or dysfunctional cellular enzymes are responsible for a wide range of diseases including cancer, neurodegenerative conditions, and metabolic disorders. Deficiencies in enzyme level or biofunction may lead to intracellular accumulation of substrate to toxic levels and interfere with overall cellular function, ultimately leading to cell damage, disease, and death. Marketed therapeutic interventions for inherited monogenic enzyme deficiency disorders include enzyme replacement therapy and small molecule chaperones.

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Article Synopsis
  • Immunogenicity testing helps understand the immune response to protein therapies, and neutralizing antibody (NAb) assays are key to characterizing anti-drug antibody responses.
  • A group of NAb assay experts has created standardized reporting recommendations to streamline communication and facilitate regulatory submissions.
  • The document outlines essential components for NAb sample analysis reports, though it does not cover the interpretation of immunogenicity data related to safety and efficacy.
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Mucopolysaccharidosis type II, commonly called Hunter syndrome, is a rare X-linked recessive disease caused by the deficiency of the lysosomal enzyme iduronate-2-sulphatase (I2S). A deficiency of I2S causes an abnormal glycosaminoglycans accumulation in the body's cells. Although enzyme replacement therapy is the standard therapy, adeno-associated viruses (AAV)-based gene therapy could provide a single-dose solution to achieve a prolonged and constant enzyme level to improve patient's quality of life.

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