Type 1 Diabetes Treatment

Protection and regeneration of β cells by gene therapy


A completely new therapeutic mechanism for type 1 diabetes (T1D) that “protects and regenerates β cells,” this safe, single, low-dose regimen suppresses hyperglycemia in acute and chronic phases and improves glucose tolerance over the long term.

Background and Technology

T1D is caused by an autoimmune reaction that destroys the beta cells of the pancreas that synthesize and secrete insulin. Insulin therapy is the only treatment, but it is only a symptomatic hypoglycemic treatment and does not prevent the risk of hypoglycemia and progression of complications because physiological blood glucose control is difficult. Therefore, the development of an innovative treatment is of paramount importance, and islet transplantation therapy is being investigated, but its actual application is limited due to the need for permanent immunosuppressive drugs, the shortage of donors, and the high cost of medical care.

To address these issues, we have developed a new gene therapy technology for novel therapeutic factors (Factor 1 and 2) that protect and regenerate beta cells in vivo. A two-week intravenous administration of a vector carrying the Factor 1 + 2 genes to a streptozotocin (STZ)-induced T1D mouse model suppressed hyperglycemia from the time of administration until the end of observation (70 days) (left figure), and a glucose tolerance test 14 days and 60 days after administration showed significant suppression of hyperglycemia and significant reduction of insulin secretion induction was observed. (right figure).

Reference and Patent

Patent pending

Principal Investigator

Ken-ichiro Kosai (Kagoshima University Graduate School of Medical and Dental Science)

Current Stage and Next Step

  • Currently, the university laboratory is in the process of discovering and demonstrating in vivo therapeutic effects in T1D model animals.
  • In the future, by using vectors that can sustain expression for a long period of time and by further increasing the activity of Factor, the company aims to eventually develop a gene therapy that can demonstrate an almost permanent T1D therapeutic effect with a single administration.
  • We are looking for partner companies to jointly conduct practical R&D on this technology. Detailed information can be disclosed upon signing a nondisclosure agreement.

Product No.  BK-04162

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