A New Delivery System for Factor IX for the Treatment of Hemophilia B

Researchers have developed a new oral delivery system for factor IX for the treatment of hemophilia B, consisting of biodegradable hydrophilic carriers that release factor IX in response to environmental stimuli.

Sarena Horava, PhD, of Triton Systems, Inc. in Chelmsford, Massachusetts, and authors reported their findings in the International Journal of Pharmaceutics. This biodegradable carrier system is designed to capitalize on the body’s pH as well as changes in enzymes inside the gastrointestinal tract, which allows for site-specific degradation by trypsin in the small intestine. The carrier remains intact as it moves through the body, and once it reaches the small intestine. From there, the carrier is degraded by trypsin and slowly releases factor IX over time.

Both the microparticles and the degradation products enhanced the in vitro absorption of factor IX, the researchers reported. They are planning to conduct more preclinical experiments with the system before initiating any clinical trials.

Source: Horava SD, Moy KJ, Peppas NA. Biodegradable hydrophilic carriers for the oral delivery of hematological factor IX for hemophilia B treatment. Int J Pharmacoceut. 2016;514:220-8.