BioCryst is a commercial-stage biotech committed to rare diseases.
At BioCryst, our structure-guided drug design propels forward the discovery and development of novel, small-molecule, oral drugs for people living with rare diseases. With expertise in drug discovery, clinical development, and regulatory affairs, we are advancing clinical programmes and generating new compounds from our own discovery engine across locations in the United States, United Kingdom and European Union.
Our Approach to Drug Discovery
BioCryst’s structure-guided approach to drug design uses an iterative process to increase selectivity and enable efficient, rapid drug development.
In structure-guided drug design, scientists use detailed knowledge of the active sites of protein targets associated with particular diseases to design synthetic compounds that fight the disease. The active site of an enzyme is the area into which a chemical or biological molecule fits to initiate a biochemical reaction. Structure-guided drug design aims to create a molecule that will bind to the active site of a targeted enzyme, thereby preventing the normal chemical reaction and ultimately halting the progression of the disease.
BioCryst’s structure-guided drug design involves the integrated application of traditional biology and medicinal chemistry along with an array of advanced technologies, including x-ray crystallography, computer modeling of molecular structures, virtual screening, and protein biophysical chemistry to focus on the three-dimensional structure of the active site of the target enzyme.
By identifying the target protein in advance, and by determining molecular structure of the protein, our scientists aim to design innovative medicines to interact with the protein. BioCryst’s scientists design and synthesize drug candidates atom by atom, to fit the active site on the protein, thereby suppressing its biological activity. The initial targets for structure-guided drug design are selected based on their involvement in the biological pathways integral to the course of a disease.
Rare Disease Therapeutics
Our structure-guided drug design process leads us to the discovery and development of novel small-molecule oral drugs that target rare diseases.
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 plays an important role in limiting the formation of bradykinin and preventing the bradykinin-forming system from becoming hyperactive and mediating swelling in HAE. HAE is a rare condition, affecting between approximately 1 in 10,000 to 1 in 50,000 people.1 Left untreated, patients with HAE often have multiple attacks every month, and the swelling from each attack can last for 2 to 4 days.2
HAE is a rare condition, affecting between approximately 1 in 10,000 to 1 in 50,000 people.1 Left untreated, patients with HAE often have multiple attacks every month, and the swelling from each attack can last for 2 to 4 days.2
Fibrodysplasia ossificans progressiva
Fibrodysplasia ossificans progressiva (FOP) is a rare, progressive genetic disorder that has an estimated prevalence of 1 per 735,000 to 1 per 2 million births.3 Often diagnosed at birth based on malformation of the big toes, this is a devastating condition that causes bone formation in muscles, tendons, ligaments, and connective tissue. Over time it creates a second skeleton that makes movement in affected areas difficult or impossible.
Paroxysmal nocturnal hemoglobinuria
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, chronic, disabling, and life-threatening disease affecting 1 to 1.5 persons per million worldwide.4 In PNH, uncontrolled complement activity and consequent red blood cell destruction (hemolysis) leads to chronic hemolytic anemia, thrombophilia, and bone marrow failure.5 Individuals with PNH are at increased risk of thrombotic events, which occurs in up to 30% of patients (in western countries) during the disease course and is the main cause of mortality.4 The impact of PNH is both physical and psychological, reducing quality of life and activities of daily living.
1 Bernstein JA. HAE update: epidemiology and burden of disease. Allergy Asthma Proc. 2013;34(1):3-6.
2 Bernstein JA. Severity of hereditary angioedema, prevalence, and diagnostic considerations. Am J Manag Care. 2018;24:S292-S298.
3 Baujat et al. Orphanet Journal of Rare Diseases (2017) 12:123.
4 Hill A, DeZern AE, Kinoshita T, Brodsky RA. Paroxysmal nocturnal haemoglobinuria. Nat Rev Dis Primers. 2017;3:17028. doi:10.1038/nrdp.2017.28.
5 Parker CJ. Update on the diagnosis and management of paroxysmal nocturnal hemoglobinuria. Hematology Am Soc Hematol Educ Program. 2016; (1): 208–216.
Commitment to Clinical Trial Data Transparency
As a science-led biotech company, our clinical research is critical for the development of medicines for rare disease. We recognise the importance of sharing clinical trial data to advance science in order to serve the patients who use our medicines and the providers who prescribe them. We provide clinical trial data in a way and is consistent with patient privacy and data integrity and is consistent with applicable laws and regulations.
BioCryst registers clinical trial information and publishes summary results, regardless of outcome, in accordance with applicable laws and regulations on public registries including Clinicaltrials.gov and European Clinical Trials Database (EudraCT).
BioCryst encourages and seeks publication of registered clinical trial results in peer reviewed publications in a manner that ensures scientific accuracy and reflects applicable industry standards.
If you have any specific questions regarding BioCryst’s Clinical Trial Disclosure and Data Transparency commitment or practice, please contact us at email@example.com.
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