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NCFB is a chronic pulmonary disorder in which the bronchi become permanently dilated due to a vicious cycle of inflammation and complications from prior infections.^1,2 The condition is marked by frequent pulmonary exacerbations requiring antibiotic therapy and/or hospitalizations.

Nontuberculous mycobacterial (NTM) lung disease is a rare and serious disorder most commonly caused by a group of bacteria called Mycobacterium avium complex (MAC).³ People who have bronchiectasis, COPD, and asthma are at greater risk of getting NTM lung disease.^4,5,6

Nontuberculous mycobacterial (NTM) lung disease is a rare and serious disorder most commonly caused by a group of bacteria called Mycobacterium avium complex (MAC).³ People who have bronchiectasis, COPD, and asthma are at greater risk of getting NTM lung disease.^4,5,6

Interstitial lung diseases (ILD) comprise a large group of diseases that cause fibrosis (scarring) of the lungs. Pulmonary hypertension (high blood pressure in the lungs) is a common and important complication of several ILDs that is associated with reduced exercise capacity and poor prognosis.^7,8 PH-ILD is also known as WHO Group 3 Pulmonary Hypertension.

PAH is a serious, progressive, rare disease involving narrowing and constriction of the pulmonary arteries that carry blood from the right side of the heart to the lungs.⁹ PAH is also known as WHO Group 1 Pulmonary Hypertension.

PPF is a type of interstitial lung disease associated with a known cause that is demonstrated to progress, causing gradual worsening of inflammation and tissue scarring, or fibrosis, in the walls of the air sacs in the lungs. As fibrosis progresses, breathlessness during exercise and daily activities becomes more common, and patients can eventually experience lung failure.¹⁰

IPF is a serious, chronic disease affecting the tissues surrounding the air sacs in the lungs. Over time, the lung tissue becomes thick and stiff, eventually causing fibrosis that makes it progressively more difficult to breathe. While the causes of the disease are unknown, it is more common in older patients and those with a family history of IPF or who smoke. Initial symptoms often include shortness of breath and cough, which can progress to pulmonary hypertension and respiratory failure.^11,12

ILD includes a heterogeneous group of approximately 200 disorders characterized by inflammation and fibrosis of the tissues in or around the air sacs and airways of the lungs. ILDs can be medication- or radiation-induced, genetic, environmental, autoimmune, occupational, or idiopathic (unknown). Symptoms may include shortness of breath, cough, chest discomfort, and fatigue, and certain ILDs may progress to life-threatening complications such as high blood pressure and heart or respiratory failure.^13,14

Asthma is a chronic lung condition involving inflammation and hyperactivity of the airways, which can cause recurrent cough, wheezing, shortness of breath, chest tightness, straining of the neck and chest muscles, rapid breathing, and changes in heart rate. Symptoms are often triggered by environmental irritants, and in severe cases, these can cause attacks requiring hospitalization. Patients with moderate to severe asthma experience symptoms daily or throughout the day, have somewhat or very limited activity due to exacerbations, experience regular nighttime symptoms, and have significantly decreased lung function, despite treatment with available therapies.^15,16

HS is a chronic relapsing inflammatory disorder, characterized by painful, inflamed, and swollen lesions affecting hair follicles, often in the armpits, groin, and skin folds.¹⁷

Rheumatoid arthritis is a chronic, inflammatory condition in which the body’s immune system attacks its own tissues, causing significant inflammation in joints and other organs. It most commonly affects small joints in the wrists, hands, and feet, causing joint pain, stiffness, swelling, and decreased flexibility, but can also affect larger joints and organs such as the skin, eyes, heart, lungs, and blood vessels. Despite medical advancements, rheumatoid arthritis can still cause long-term damage and increase the risk of heart disease.^18,19

IBD refers to a group of chronic inflammatory diseases, including ulcerative colitis and Crohn’s disease, that cause swelling of the tissues in the digestive tract.²⁰ It occurs when the body’s natural defense system mistakenly attacks healthy bowel cells.²¹ Symptoms typically flare up and then recede and often include diarrhea, stomach pain, rectal bleeding, fatigue, and weight loss. While IBD can be mild for some, for others, it can cause disability and lead to life-threatening complications.²⁰

Chronic refractory gout is a rare form of gout, which results from a buildup of uric acid in the body, leading to the deposition of uric acid crystals in joints and tissues. These deposits can cause swelling, redness, and intense pain in the joints; tophi, or lumps, that form around the hands, elbows, and other body parts; chronic arthritis; and permanent joint damage. In this refractory form of the disease, uric acid levels cannot be controlled with conventional gout medications or treatment.^22,23

DMD is caused by a genetic mutation that prevents the body from producing dystrophin, a protein that muscles need to work properly. Without it, muscle cells become damaged and weaken.²⁴ DMD is the most common childhood onset form of muscular dystrophy and affects males almost exclusively.²⁵

ALS is a progressive neurological disease that affects the nerve cells in the brain and spinal cord that control voluntary muscle movement and breathing. As these nerve cells degenerate, the body’s muscles begin to weaken and waste away. Eventually, the brain loses its ability to control voluntary movements like walking, talking, chewing, and breathing. Most people with ALS die of respiratory failure within three to five years from initial symptom onset.²⁶

Stargardt disease is a rare genetic eye disease caused by the buildup of fatty material on the macula, the small part of the retina needed for sharp, central vision.²⁷ The disease leads to the loss of central vision in both eyes, typically beginning in childhood. Today, there are no treatments available for the disease, and management options focus on optimizing patients’ remaining sight.

AT is an inherited, fatal childhood disorder driven by mutations in the ataxia-telangiectasia mutated (ATM) gene, which is involved in DNA damage repair. Symptoms vary between individuals but typically involve degeneration across a range of neurological and neuromuscular functions, impaired metabolic and immune system functions, and an increased risk of cancer. People with AT often require significant support and face a significantly reduced life expectancy.²⁸

AOA1 is an autosomal recessive disease caused by mutations in the APTX gene that encodes aprataxin, a nuclear protein involved in DNA repair. The disease typically presents in childhood with the onset of progressive problems with balance (cerebellar ataxia), difficulty moving the eyes (oculomotor apraxia), nerve damage affecting the peripheral nerves, and involuntary movements. Adult patients often experience high cholesterol, cognitive impairment, and low levels of albumin, a protein responsible for keeping fluid in the blood vessels.^29,30