Partnership to Aid Access to Man-made FVIII for Patients in China

GenScript ProBio and Neoletix Biotechnology have entered into an agreement for clinical and commercial production of a lab-made version of coagulation factor VIII, a product that the companies hope will make the preventive treatment for hemophilia A more accessible to patients in China.

Coagulation factor VIII, or FVIII, is a protein that is needed for blood to clot properly. In people with hemophilia A, this protein is either missing or unable to function properly, resulting in excessive and prolonged bleeding. A standard treatment strategy for hemophilia A involves administering FVIII protein prophylactically, or as preventive measure, to lower the risk of bleeds.

FVIII protein used for this purpose can be derived from human blood or artificially produced in a laboratory. Man-made, or recombinant, FVIII production is generally more feasible for industry-scale applications. However, because it is not a very stable protein, it can be difficult to reliably produce enough FVIII in an industrial setting.

Through this partnership, GenScript will be providing aid to Neoletix in developing the manufacturing capacities for its recombinant FVIII product. Neoletix is planning to start clinical trials of the product toward the end of this year, pending appropriate regulatory approvals.

“We appreciate GenScript ProBio’s efforts in providing sustained support to Neoletix’s recombinant FVIII clinical and commercial production,” Chester Li, MD, PhD, CEO of Neoletix, which is based in Beijing, said in a press release.

In part due to difficulties in manufacturing, recombinant FVIII therapies are often expensive and their supply is limited. Consequently, many people with hemophilia A who live in China are unable to access prophylactic therapy.

According to Genscript, the two companies have created a novel production process that allows for the production of recombinant FVIII at “levels unachievable by other commercial vendors, while maintaining the highest quality of product,” which is expected to make the medication more affordable and more easily available to people in the country.

“High FVIII production coupled with reduced low manufacturing costs, will make our [human recombinant FVIII] product much more accessible, which in turn will fundamentally reshape the treatment and prevention landscape for patients with hemophilia A and greatly improve their quality of life. We are looking forward to further cooperation with GenScript in bringing benefits for patients as early as possible,” Li said.

GenScript ProBio, founded in New Jersey in 2002, includes as its specialties the development and manufacturing of biologics.