We seek out unmet needs to discover unparalleled solutions
We study diseases in areas of high unmet need, where few, if any, therapies exist today. These include neutrophil-mediated inflammatory diseases and rare pulmonary disorders.
Nontuberculous Mycobacterial (NTM) Lung Disease
Nontuberculous mycobacterial (NTM) lung disease is a rare and serious disorder most commonly caused by a group of bacteria in the environment called Mycobacterium avium complex (MAC).1 Patients with NTM lung disease caused by MAC can experience a range of symptoms that often worsen over time, including chronic cough, dyspnea, fatigue, fever, weight loss, and chest pain. In some cases, the disease can cause severe, even permanent damage to the lungs, and can be fatal.
People who have underlying conditions such as bronchiectasis, COPD, and asthma are at greater risk of getting NTM lung disease.2,3,4
Find additional information and resources about NTM lung disease.
Bronchiectasis
Bronchiectasis is a chronic pulmonary disorder in which the airways, or bronchi, become permanently dilated due to a vicious cycle of inflammation and complications from prior infections.5,6 Bronchiectasis is marked by frequent pulmonary exacerbations requiring antibiotic therapy and/or hospitalizations. Symptoms include chronic cough, excessive sputum production, shortness of breath, and repeated respiratory infections, which can worsen the underlying condition.5,6
There is a high overlap in patients with NTM lung disease and patients with bronchiectasis.6 Today, no therapies are approved specifically for the treatment of bronchiectasis in the U.S., EU, or Japan.
Cystic Fibrosis
Cystic fibrosis (CF) is a rare, progressive, genetic disease caused by mutations in the cystic fibrosis transmembrane regulator (CFTR) gene.7 CF leads to persistent lung infections and limits the ability to breathe over time.8 Common symptoms include salty-tasting skin, chronic cough that may produce thick mucus or blood, wheezing or shortness of breath, nasal polyps, poor growth in childhood, and constipation.9
Today, most CF diagnoses are made at newborn screenings, allowing for earlier intervention. With progressive advances in care, life expectancy has increased from months to approximately 40 years.10 But despite available therapies, challenges in treating CF remain, and new treatment options are needed.
Pulmonary Hypertension associated with Interstitial Lung Disease
Interstitial lung diseases (ILD) are a large group of lung conditions where scar tissue or fibrosis develops diffusely throughout both lungs. Pulmonary hypertension (PH), or high blood pressure in the lungs, is a common and significant complication of ILD that further impairs breathing, exercise capacity, and quality of life.11,12 Common symptoms of PH-ILD include shortness of breath with physical activity that gradually worsens over time, chest pain, fainting episodes, difficulty breathing when lying flat, and swelling of the ankles.13
Survival in patients with PH-ILD is worse than those with ILD without PH, and additional therapies are urgently needed.
Pulmonary Arterial Hypertension
Pulmonary arterial hypertension (PAH) is a serious, progressive, rare disease involving high blood pressure in the pulmonary arteries, which carry blood from the heart to the lungs.14 The cause of PAH is often unknown, but it can be inherited, caused by certain drugs or toxins, or caused by conditions such as connective tissue disease, HIV infection, liver disease, congenital heart disease, sickle cell disease, schistosomiasis, or sequelae to thromboembolic events.15 The most common symptoms include shortness of breath, chest pain, dizziness or fainting, fatigue, and weakness.16 Untreated, PAH can be debilitating, and often fatal.17,18
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References
- Adjemian J, Prevots DR, Gallagher J, Heap K, Gupta R, Griffith D. Lack of adherence to evidence-based treatment guidelines for nontuberculous mycobacterial lung disease. Ann Am Thorac Soc. 2014;11(1):9-16.
- Mirsaeidi M, Hadid W, Ericsoussi B, Rodgers D, Sadikot RT. Non-tuberculous mycobacterial disease is common in patients with non-cystic fibrosis bronchiectasis. Int J Infect Dis.2013;17(11):e1000-e1004.
- Andréjak C, Nielsen R, Thomsen VØ, Duhaut P, Sørensen HT, Thomsen RW. Chronic respiratory disease, inhaled corticosteroids and risk of non-tuberculous mycobacteriosis. Thorax. 2013;68(3):256-262.
- Fritscher LG, Marras TK, Bradi AC, Fritscher CC, Balter MS, Chapman KR. Nontuberculous mycobacterial infection as a cause of difficult-to-control asthma: a case-control study. Chest. 2011;139(1):23-27.
- Prevalence and incidence of noncystic fibrosis bronchiectasis among US adults in 2013 (Weycker). https://www.ncbi.nlm.nih.gov/pubmed/28555504. Accessed December 2020.
- Advances in bronchiectasis: endotyping, genetics, microbiome, and disease heterogeneity (Flume). https://www.ncbi.nlm.nih.gov/pubmed/30215383. Accessed December 2020.
- National Institutes of Health. Genetic and Rare Disease Information Center. https://rarediseases.info.nih.gov/diseases/6233/cystic-fibrosis. Accessed October 2020.
- Cystic Fibrosis Foundation. About Cystic Fibrosis. https://www.cff.org/What-is-CF/About-Cystic-Fibrosis/. Accessed December 2020.
- Centers for Disease Control and Prevention. Cystic Fibrosis. https://www.cdc.gov/genomics/disease/cystic_fibrosis.htm. Accessed December 2022.
- Redondo M, Keyt H, Dhar R, Chalmers JD. Global impact of bronchiectasis and cystic fibrosis. Breathe (Sheff). 2016 Sep;12(3):222-235.
- American Lung Association. Interstitial Lung Disease. https://www.lung.org/lung-health-diseases/lung-disease-lookup/interstitial-lung-disease. Accessed January 2021.
- Caminati A. Pulmonary hypertension in chronic interstitial lung diseases. European Respiratory Review 2013 22: 292-301 https://err.ersjournals.com/content/22/129/292. Accessed January 2021
- University of California San Francisco Health. Pulmonary Hypertension and Interstitial Lung Disease. https://www.ucsfhealth.org/education/pulmonary-hypertension-and-interstitial-lung-disease. Accessed December 2022.
- Pulmonary Hypertension Association. About Pulmonary Hypertension (PH) and Pulmonary Arterial Hypertension (PAH). http://www.ourphlibrary.com/module1-about-pah/what-is-pah. Accessed January 2021.
- American Heart Association. Pulmonary Hypertension – High Blood Pressure in the Heart-to-Lung System. https://www.heart.org/en/health-topics/high-blood-pressure/the-facts-about-high-blood-pressure/pulmonary-hypertension-high-blood-pressure-in-the-heart-to-lung-system. Accessed February 2019.
- Rare Disease Report. Rare Disease Quick Facts – Pulmonary Arterial Hypertension (PAH). https://www.raredr.com/news/rare-disease-quick-facts-pah. Accessed February 2019.
- Klinger JR, Elliott CG, Levine DJ, Bossone E, Duvall L, Fagan K, Frantsve-Hawley J, Kawut SM, Ryan JJ, Rosenzweig EB, Sederstrom N, Steen, VD, Badesch DB. Therapy for Pulmonary Arterial Hypertension in Adults: Update of the CHEST Guideline and Expert Panel Report. Chest. 2019;155(3):565-586.
- Delcroix M, Howard L. Pulmonary arterial hypertension: the burden of disease and impact on quality of life. Eur Respir Rev. 2015;24:621-629.
