BMCC-2022-GT01

Hyperactive Factor IX Variant “FIX-TripleL” for Hemophilia B

BMCC

Superior activity! Safety! Efficacy!

未定中標題
BMCC Targeted indication

1. Replacement therapy of Hemophilia B
2. Gene therapy of Hemophilia B

BMCC Status

Pre-clinical studies

BMCC Key features
  • One injection lasting >>3 years.
  • Higher activity at lower dosage than competitors.
  • No thrombosis risk or genotoxicity in a long-term follow up animal model.
BMCC Market

The technology of the team is that we have developed a supra-active FIX molecule called FIX-TripleL. Combining our self-owned global patented FIX-TripleL technology with AAV-8 vector, we have completed POC and validated the therapeutic efficacy of FIX-TripleL in gene therapy using a hemophilia B mouse model. FIX-TripleL is therapeutically effective when used at a supra-low dose, as low as 50 times less than what has currently been used in hemophilia B gene therapy trials. Lowering viral doses is the emerging goal of gene therapy. FIX-TripleL, having extremely high activity, shows powerful potential in the market for hemophilia B gene therapy. In addition, the team is about to complete the GLP toxicology test and safety assessment under the grant supported by the Ministry of Science and Technology, Executive Yen, Taiwan. Taken together, the team's technology will become the first gene therapy clinical product that is entirely accomplished by Taiwan from development, mass production to GLP testing.

MODE OF ACTION