Therapeutic Development Services
Treatment options for factor VII deficiency center around the administration of recombinant FVIIa for acute bleeding or prophylaxis. However, predicting thrombotic risk and standardizing replacement therapy across variants still poses a challenge. Protheragen, with its extensive knowledge and experience in factor VII deficiency therapy's development, is poised to provide tailored solutions alongside complete support to ease the transition from research to commercialization.
Factor VII deficiency is an uncommon form of autosomal recessive bleeding disorder which involves the impaired initiation of the extrinsic coagulation pathway. The clinical condition shows marked heterogeneity, with some patients being asymptomatic while others can have life threatening hemorrhages. There is a strikingly poor correlation between the activity of FVII in plasma and the severity of bleeding. The global prevalence of factor VII deficiency is estimated to be approximately 1 in 500,000 population.
Fig.1 Effects of multiple anticoagulant medications on the coagulation cascade. (Paulus E, et al., 2016)
The etiology of factor VII deficiency arises from mutations in the F7 gene (located at 13q34) which disrupts important functional domains in the coagulation protein. More than 200 mutations are known to lead to impaired secretion, ineffective tissue factor binding, or some form of catalysis malfunction. These molecular defects are accompanied by insufficient thrombin generation and delayed fibrin clot formation with a range of <1% to almost normal levels of FVII activity.
Fig.2 Schematic diagram of the F7 gene and factor VII protein expression. (Bernardi F, Mariani G., 2021)
| Drug Names | Mechanism of Action | Targets | NCT Number | Phase |
|---|---|---|---|---|
| Eptacog Alfa | Recombinant activated FVII that directly activates factor X in the absence of tissue factor, bypassing the intrinsic coagulation pathway. | Factor X | NCT03079063 | Approved |
| Marzeptacog Alfa | Engineered FVIIa variant with enhanced binding to activated platelets and increased proteolytic activity toward FX. | Factor X | NCT04548791 | Phase I/II |
| SS109 | Long-acting FVIIa variant with amino acid substitutions that increase binding to endogenous albumin, prolonging half-life. | Factor X/Albumin | NCT05651061 | Phase I |
Disclaimer: Protheragen focuses on providing preclinical research services. This table is for information exchange purposes only. This table is not a treatment plan recommendation. For guidance on treatment options, please visit a regular hospital.
By providing thorough diagnostic and therapeutic development services, Protheragen is devoted to progressing the treatment of rare bleeding disorders such as factor VII deficiency. We apply complete disease management methodologies starting from the development of in vitro diagnostic (IVD) kits all the way to the creation of new therapeutics. With the help of modern technologies and scientific know-how, we excel at creating accurate factor VII deficiency disease models which capture the intricacies of the condition.
Therapeutic Development Services
By deeply understanding the pathogenesis of reticular dysgenesis (RD), Protheragen is focused on developing innovative therapies across multiple molecule types to address the complex challenges in this field.
F7 Mutation Model: Protheragen employs gene editing techniques to introduce specific variants like Ala294Val and Arg304Gln into transgenic mice and dog models, creating tailored F7 mutation models. We offer validated models which allow quantification of FVII activity and phenotypic characterization as well as customized drug response evaluation to accelerate your therapeutic development.
At Protheragen, we commit ourselves to the advancement of novel therapies by providing complete preclinical research services. We offer unparalleled support in pharmacodynamics (PD), pharmacokinetic (PK) and toxicology studies, all of which are tailored to comprehensively assess your drug candidates. If you are interested in our services, please feel free to contact us for more details and quotation information of related services.
References
Myeloproliferative Disorders (MPDs)
Bone Marrow Failure Syndromes (BMFS)
