Expression Therapeutics, ABL Enter Collaboration For Hemophilia A Factor VIII Infusion Therapy Cell Line Development
By C. Rajan, contributing writer
Expression Therapeutics, a biotechnology company which is developing therapies for hemophilia A, has just selected the Maryland-based biomedical contract research and manufacturing organization, ABL to support the cell line development studies for its novel Factor VIII infusion therapy for Hemophilia A.
According to the terms of the agreement, ABL will perform cell line development studies to improve the manufacturing process and reduce the costs associated with large-scale production of recombinant factor VIII replacement therapy.
Hemophilia A is a genetically inherited blood clotting disorder in which an essential clotting component, factor VIII, is missing. The main treatment for the condition is a concentrated form of factor VIII administered intravenously to the patient three or more times a week. In over 75 percent of the cases, a recombinant factor VIII product is used rather than a plasma derived factor VIII product, according to the National Hemophilia Foundation.
Expression Therapeutics, a Georgia-based biotechnology company, has developed a recombinant factor VIII protein infusion product that could be manufactured at significantly lower costs and increase the availability of factor VIII supply, especially for patients in developing countries.
ABL will adapt the current cell line to grow in chemically defined, animal origin-free media, and conduct optimization studies of cell growth conditions to support large scale production in a bioreactor-based process.
“This is a great opportunity for ABL to apply our expertise in cell line development to help advance Expression Therapeutics’ factor VIII product towards clinical production. We are very excited about our collaboration with ABL. It will allow us to move our lead products into clinical trials faster and deliver our high expression recombinant factor VIII to patients,” explains Dr. Christopher Doering, Co-founder and Partner of Expression Therapeutics, LLC.
Expression Therapeutics is one of the few companies working on developing effective hemophilia A therapies. Just this year in June, the FDA approved Biogen's Eloctate, the first hemophilia A treatment with prolonged circulation in the body. Also in June, Bayer signed a deal with Dimension Therapeutics for the development and commercialization of a new gene replacement therapy for hemophilia A, which will also eliminate the need for multiple intravenous infusions of factor VIII. Expression Therapeutics has also developed a potentially curative hematopoietic stem cell gene therapy treatment using recombinant viral vectors.
According to Expression Therapeutics, hemophilia is estimated to occur in approximately 1 out of every 10,000 male births, and roughly one third of all cases occur without a family history of the disorder. Patients with severe hemophilia A experience spontaneous bleeding episodes, bruising, gastrointestinal bleeding, and sometimes life-threatening bleeding. Without treatment, hemophilia A is a lethal disorder resulting in death prior to adulthood. However, due to the high cost of intravenous factor VIII replacement therapy and the multiple weekly treatments required, fewer than 30 percent of hemophilia A patients receive treatment.