Selective Removal of Insulin-Specific B-cells to Prevent Type I Diabetes in NOD Mice

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 DESCRIPTION (provided by applicant): Akston Biosciences Corp. is developing a subcutaneously administered therapeutic for preventing or delaying the onset of diabetes in pre-diabetic patients who are at high risk (those who display autoantibodies for insulin, GAD65, IA-2, and ZnT8) for developing Type 1 diabetes (T1D). The therapeutic is designed to delete a subset of B-cells that likely play a role in disease development using a patient's own macrophages. The work covered in this submission builds on preliminary results obtained from both in vitro cell culture and in vivo rodent models. Before entering into preclinical development, however, Akston seeks to identify the best therapeutic regimen in a one month study in NOD Tg mice, followed by a more extensive eight month study in wild-type NOD mice to demonstrate statistically significant prevention of diabetes in treated animals. Doing so will provide critical "proof-of-concept" data and will lead to detailed preclinical, IND-enabling safety and efficacy work and, eventually, clinical testing of the therapeutic in man. The impacts to public health as a result of this project are potentially significant. Healthcare costs directly attributable to T1D patients currently account for nearly $20 billion annually. In addition to the 3 million Americans who currently live with T1D, 30,000 new T1D patients are diagnosed each year, with the rate of newly diagnosed patients < 20 years of age increasing by 23% in just the past decade. If successful, Akston's therapeutic could lead to a significant reduction in health care costs and possibly free pre-T1D children and teenagers from a lifetime of glucose monitoring and insulin injections.