Rare Disease Therapeutics
Our structure-guided drug design process leads us to the discovery and development of optimized novel small-molecule drugs that target rare diseases.
We focus on therapeutic areas with either no available treatments or large unmet patient needs that BioCryst can fill. BioCryst has announced 2 rare disease programs that are currently in development.
Hereditary angioedema (HAE) is a potentially life-threatening disease caused by a genetic deficiency of a protein called C1 esterase inhibitor (C1-INH). C1-INH regulates levels of a hormone in the blood plasma called kallikrein, which in turn plays an activating role in inflammation (swelling). The lack of normal regulation of kallikrein puts patients with HAE at risk of uncontrolled swelling attacks around the body that can be painful, disfiguring, and even life threatening in the case of throat swelling.
HAE is a rare condition, affecting between 1 in 10,000 to 1 in 50,000 people. Left untreated, patients with HAE often have multiple attacks every month, and the swelling from each attack can last for 3 days or longer. Current standard of care for patients with HAE involves frequent infusions or injections of plasma-derived C1-INH to prevent swelling attacks, as well as injectable therapies to treat attacks when they happen.
Fibrodysplasia ossificans progressiva (FOP)
FOP is a rare, severely disabling condition characterized by the irregular formation of bone outside the normal skeleton, also known as heterotopic ossification (HO). HO can occur in muscles, tendons, and soft tissue. FOP patients progressively become bound by this irregular bone formation, with restricted movement and fused joints, resulting in deformities and premature mortality. There are currently no approved treatments for FOP.