Disc Medicine
Disc Medicine works on 5 rare diseases tracked on Trial Friend, including Essential Thrombocythemia, Myelodysplastic Syndromes, Myelofibrosis and 2 more, with 3 recruiting clinical trials.
Disc Medicine is a hematology company developing disease-modifying treatments for erythropoietic porphyrias and other rare hematologic disorders. The company's lead candidate bitopertin (a GlyT1 inhibitor for erythropoietic protoporphyria) received a Complete Response Letter from the FDA in February 2026. Disc Medicine also develops DISC-0974 for myelofibrosis-associated anemia and DISC-3405, an anti-TMPRSS6 monoclonal antibody in Phase 1b for sickle cell disease.
Focus areas at Disc Medicine
As a rare disease specialist, Disc Medicine has active clinical trial programs and drug development efforts across 5 rare diseases, including Essential Thrombocythemia, Myelodysplastic Syndromes, Myelofibrosis, Polycythemia Vera, Sickle Cell Disease. These programs may span orphan drug designation, novel therapeutic mechanisms, and precision medicine approaches targeting the underlying causes of each disease.
The clinical trials section below shows all active and recruiting studies sponsored by Disc Medicine, sourced live from ClinicalTrials.gov. Each trial includes its current recruitment status, study phase (Phase 1 through Phase 4), conditions under investigation, and the number of active trial sites. The FDA-approved drugs section lists treatments that have received U.S. Food and Drug Administration approval, with brand names, generic names, approval dates, and matched rare disease indications from the openFDA database.
Disc Medicine is headquartered in Watertown, United States, founded in 2017. The company dedicates its research and development resources exclusively to rare and orphan diseases, working to bring innovative treatments to patients with conditions that have historically had limited or no treatment options.
Disc Medicine Drug Pipeline
Disc Medicine has 3 active clinical trials across 2 development stages, with 3 currently recruiting participants. Clinical trials advance through phases: Phase 1 tests safety in a small group, Phase 2 evaluates effectiveness and side effects, Phase 3 confirms benefit in a larger population, and Phase 4 monitors long-term safety after FDA approval.
Note: This pipeline includes all of Disc Medicine's active interventional trials, not only those targeting rare diseases. We show the full pipeline because a company's broader research activity, therapeutic expertise, and development infrastructure directly shape its ability to advance rare disease programs. A strong overall pipeline often signals deeper clinical operations, faster enrollment capabilities, and greater commitment to bringing new treatments to patients.
Disc Medicine Clinical Trials (3)
Active and recruiting clinical trials sponsored by Disc Medicine, sourced live from ClinicalTrials.gov. Each trial card shows the study phase, current recruitment status, conditions under investigation, study locations, eligibility criteria, and a direct link to the full ClinicalTrials.gov record. You can also download a one-page PDF summary to share with your doctor.
Note: Recruitment statuses on ClinicalTrials.gov may not immediately reflect recent FDA decisions, sponsor announcements, or enrollment changes. Always confirm a trial's current status directly with the study coordinator.
FDA-Approved Drugs
Disc Medicine Trial Locations
Disc Medicine clinical trials are running at 57 sites in 2 countries. Click any country to drill down by state, city, and individual research facility. Proximity to a trial site is one of the most important factors in deciding whether to participate.
Rare Disease Focus Areas (5)
Diseases targeted by Disc Medicine's clinical trial and drug development programs
Essential Thrombocythemia is a myeloproliferative neoplasm characterized by overproduction of platelets by bone marrow, resulting in very high platelet counts. Patients are at increased risk for blood...
Myelodysplastic syndromes are a group of blood cancers in which the bone marrow fails to produce enough healthy blood cells. Immature blood cells do not develop properly and either die in the bone mar...
Myelofibrosis is a myeloproliferative neoplasm in which bone marrow develops scarring (fibrosis), leading to anemia, enlarged spleen, and abnormal blood cell counts. Most cases involve JAK2, CALR, or ...
Polycythemia Vera is a myeloproliferative neoplasm in which bone marrow produces too many red blood cells, leading to thick blood that carries oxygen less effectively and increases clot risk. Most pat...
Sickle cell disease is an inherited blood disorder where hemoglobin polymerizes under low oxygen, causing red blood cells to become rigid, sickle-shaped, and prone to hemolysis and vaso-occlusion. Thi...
Patient Resources
Organizations and resources related to Disc Medicine's rare disease focus areas
Frequently Asked Questions About Disc Medicine
Common questions about Disc Medicine's rare disease programs, clinical trials, and treatments.