SWL OF NTUCM CLSMB
Area: MOST Inno Zone
Booth No: S810a
Website: www.cellenvision.biz
Exhibitor Profile
Product name:Factor IX-TripleL (FIX-TripleL)
Hemophilia B is caused by a single-gene defect in factor IX (FIX) that results in bleeding diathesis. The incidence is 1/30000. The current standard of care involves intravenous infusions with FIX protein. Although patients have achieved a normal average life span, they still suffer from blood-borne diseases, chronic joint damage (arthropathy), and frequent infusions. Phase III Gene therapy clinical trials for hemophilia B gene therapy are ongoing, however, several issues remain, such as the concern over rAAV vectors and the transgene levels. The technology of the team is that we have developed a supra-active FIX called FIX-TripleL, that can minimize the high dose AAV-related gene therapy issues. We have completed POC and validated the therapeutic efficacy of FIX-TripleL in gene therapy in hemo B mouse model. The team is about to complete the GLP toxicology test and safety assessment. Taken together, FIX-TripleL shows powerful potential in the market for hemophilia B gene therapy.
Brands
Factor IX-TripleL
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