A genetically modified rodent expressing human CD22

Simple SummaryContent extracted from patent full text and abstract with AI.

This patent describes a genetically modified rodent, particularly mice, engineered to express human CD22 protein (or fragments of it) instead of their own CD22 on B lymphocytes. These transgenic rodents serve as in vivo models for testing drugs and therapies—such as antibodies or small molecules—targeting human CD22, which is a key molecule involved in the regulation of B cell activity and is implicated in autoimmune diseases and B cell cancers. This model enables the identification and safety assessment of therapeutic agents directed against human CD22 in the context of a living organism.

Use CasesContent extracted from patent full text and abstract with AI.

• Preclinical in vivo testing of anti-CD22 therapies for diseases like systemic lupus erythematosus (SLE), rheumatoid arthritis, and multiple sclerosis.
• Evaluation of antibody drugs (including monoclonal antibodies and immunotoxins) targeting human CD22 for the treatment of B-cell lymphomas and leukemias.
• Safety studies to predict and screen potential adverse effects of anti-human CD22 agents before clinical trials.
• Screening and validation of small molecules, aptamers, siRNAs, and other therapeutic candidates targeting human CD22.
• Study of B cell biology and function, especially the role of CD22 in immune regulation and autoimmunity.
• Use as a platform to generate further disease models by crossing with other genetically modified mice, enabling more complex disease and therapy studies.

BenefitsContent extracted from patent full text and abstract with AI.

• Provides a robust and physiologically relevant in vivo model for testing human-specific therapies targeting CD22, overcoming the species differences between rodents and humans.
• Enables more accurate prediction of efficacy and safety for anti-CD22 therapeutics before moving to human clinical trials, thereby reducing risk and improving translational success.
• Facilitates the development and optimization of drugs for B cell-mediated autoimmune diseases and B cell malignancies.
• Allows for systemic investigation of the mechanisms of action, therapeutic windows, and long-term effects of anti-CD22 therapies in a controlled, repeatable setting.
• May reduce development time and costs by serving as an optimal preclinical model, minimizing false positives/negatives that result from using non-human CD22 models.

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Inventors & Applicants

Patent Abstract

The present invention relates to a genetically modified rodent comprising a nucleic acid sequence encoding human CD22, or (a) fragment(s) thereof, in its genome. The present invention further relates to methods of identifying an agent capable of treating diseases associated with dysregulated B lymphocytes as well as a method of testing the safety of an agent for anti-human CD22 therapy, based on the genetically modified rodent of the invention.