Hemophilia is an inherited bleeding disorder in which the blood does not clot properly due to the lack of sufficient congenital coagulation factors.
Hemophilia is a rare disease that occurs about one in every 10,000 people and is classified as Type A (FVIII) and Type B (FIX) depending on the
deficiency of each factor, and Type A hemophilia accounts for 80% and Type B hemophilia accounts for 20% of the total. In addition, about 20% of
hemophilia patients with type A and type B cannot achieve blood
coagulation with conventional treatments due to the occurrence of
neutralizing antibodies to hemophilia drugs, so active FVIII is being used as a bypass factor to treat them. As hemophilia is a disease that requires
lifelong care after birth, and the main focus of product development is to improve the patient's convenience by increasing the half-life of the drug.
The transferrin fusion gene recombination technology is our platform technology and it increases half-life. We are currently developing bypass factor therapy (FVII) and type B hemophilia treatment (FIX) for patients with neutralizing antibody-bearing hemophilia patients using transferrin fusion gene recombination technology and developing a new type-A hemophilia treatment(FVII) with a new mechanism.
TU7710 is a bypass-factor treatment for patients with neutralizing antibodies, and TU7918 is a continuous B-type hemophilia treatment that increases half-life by combining transferrin with gene recombination. If TU7710 and TU7918 are released with a half-life three times greater than conventional treatments, it is expected to greatly increase patient convenience.
NBX001 Next-generation continuous hemophilia treatment provides solutions to the factors that limited the half-life increase of the existing hemophilia treatment, and is being developed as a treatment that has an even greater half-life than the existing one. We are developing NBX001, a next-generation continuous hemophilia treatment, as a treatment with a better half-life than the existing treatments by offering solutions to factors that limited the half-life of the existing A-type hemophilia treatment. Our core researchers continue to work on developing more innovative and convenient treatments for patients based on their expertise and experience in developing candidate materials for AFSTLA (Type A hemophilia treatment, NBP601), a hemophiliac treatment approved by the FDA and EMA during their tenure at SK Chemical.