Our Pipeline

Actor portrayal

Your Window into Discovery

See research that's been realized and what's to come

At Insmed, our commitment to developing treatments for serious diseases remains strong. We believe in progress that serves patients everywhere, and the research we perform today has potential to make an impact tomorrow.

For more information on our clinical trials and who can participate, visit the Trials in Progress page.

Program Pipeline

Our areas of research fall under three therapeutic areas. Select a category or search by disease to take a closer look.

Showing 20 results

Respiratory Therapeutic Area

Pre-ClinicalPhase 1Phase 2Phase 3

Treprostinil Palmitil Inhalation Powder (TPIP)

Pulmonary Hypertension associated with Interstitial Lung Disease (PH-ILD)

Phase 3

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. PH-ILD is also known as WHO Group 3 Pulmonary Hypertension.

TPIP

Pulmonary Arterial Hypertension (PAH)

Phase 3

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. PAH is also known as WHO Group 1 Pulmonary Hypertension.

TPIP

Progressive Pulmonary Fibrosis (PPF)

Phase 2

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.

TPIP

Idiopathic Pulmonary Fibrosis (IPF)

Phase 2

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.

INS1148, SCF248 Monoclonal Antibody

Progressive Pulmonary Fibrosis (PPF)

Phase 1

INS1148, SCF248 Monoclonal Antibody

Idiopathic Pulmonary Fibrosis (IPF)

Phase 1

INS1033, DPP1 Inhibitor

Chronic Obstructive Pulmonary Disease (COPD)

Pre-clinical

Chronic obstructive pulmonary disease (COPD) is a progressive group of lung diseases, most commonly including emphysema and chronic bronchitis, that obstruct airflow and make breathing increasingly difficult. Typical symptoms include persistent cough, excess mucus production, wheezing, chest tightness, and shortness of breath. While presently available medicines do not offer a cure, early diagnosis, smoking cessation, and pulmonary rehabilitation can aid in slowing disease progression and reducing symptoms.

Next-Gen DPP1 Inhibitors

Other

Pre-clinical

Immunology & Inflammation Therapeutic Area

Pre-ClinicalPhase 1Phase 2Phase 3

INS1033, DPP1 Inhibitor

Rheumatoid Arthritis (RA)

Pre-clinical

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.

INS1033, DPP1 Inhibitor

Ulcerative Colitis (UC)

Pre-clinical

Ulcerative colitis (UC) is a chronic inflammatory bowel disease that causes inflammation and ulcers in the inner lining of the colon and rectum. Common symptoms include persistent diarrhea, rectal bleeding, abdominal pain, urgency to have a bowel movement, and fatigue, with symptoms often occurring in periods of flare-ups and remission. If not properly managed, UC can lead to life-threatening complications, including colorectal cancer.

Next-Gen Uricase

Chronic Refractory Gout

Pre-clinical

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.

IgG Protease

Other

Pre-clinical

INS1148, SCF248 Monoclonal Antibody

Other

Pre-clinical

Novel MOA

Other

Pre-clinical

Neuro & Other Rare Therapeutic Area

Pre-ClinicalPhase 1Phase 2Phase 3

INS1201, Gene Therapy

Duchenne Muscular Dystrophy (DMD)

Phase 1

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

INS1202, Gene Therapy

Amyotrophic Lateral Sclerosis (ALS)

Phase 1

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.

INS1203, Gene Therapy

Stargardt Disease

Pre-clinical

Stargardt disease is a rare genetic eye disease characterized by the buildup of fatty material on the macula, the small part of the retina needed for sharp, central vision. 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.

Synthetic Rescue, Antisense Oligonucleotide

Ataxia Telangiectasia (AT)

Pre-clinical

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.

Synthetic Rescue, Antisense Oligonucleotide

Ataxia with Oculomotor Apraxia Type 1 (AOA1)

Pre-clinical

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.

Other Rare

Pre-clinical

The safety and efficacy of investigational products and/or investigational uses of commercial products have not been approved by any health authority.

Not for promotional use.