ADARx Pharmaceuticals
ADARx Pharmaceuticals works on 5 rare diseases tracked on Trial Friend, including C3 Glomerulopathy, Geographic Atrophy, Hereditary Angioedema and 2 more, with 7 active clinical trials (6 recruiting).
ADARx is a San Diego biotech building small interfering RNA (siRNA) medicines, drugs that silence a disease-driving gene at the messenger-RNA level, engineered for potency and unusually long-lasting effect. The company kept its pipeline wholly owned while moving three programs into the clinic, and in 2026 filed to go public on Nasdaq.
Its most advanced program is onvuzosiran (ADX-324) for hereditary angioedema, which silences prekallikrein production to prevent the swelling attacks that define the disease. Onvuzosiran carries FDA Fast Track and orphan drug designations and is enrolling the Phase 3 STOP-HAE trial, with topline data expected by the end of 2027. The pitch to patients is dosing measured in months, not days: a long-acting injection that could make prophylaxis nearly invisible in daily life.
The second act is agazisiran, which silences complement factor B, a master switch in the complement immune cascade. That one mechanism is being aimed at several complement-driven rare diseases at once, including IgA nephropathy, C3 glomerulopathy, paroxysmal nocturnal hemoglobinuria, and geographic atrophy, with initial data expected in 2027. A third program targets factor XI for stroke prevention.
ADARx Pharmaceuticals Drug Pipeline
ADARx Pharmaceuticals has 7 active clinical trials across 2 development stages, with 6 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 counts the active and recruiting trials listed on this page, from ClinicalTrials.gov. For companies that work outside rare disease, we list only trials for rare diseases we track, so the company's full pipeline may be larger.
ADARx Pharmaceuticals Clinical Trials (7)
Active and recruiting clinical trials sponsored by ADARx Pharmaceuticals, 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.
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FDA-Approved Drugs
ADARx Pharmaceuticals Trial Locations
ADARx Pharmaceuticals clinical trials are running at 253 sites in 27 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 ADARx Pharmaceuticals's clinical trial and drug development programs
C3 glomerulopathy is a group of rare kidney diseases caused by dysregulation of the alternative complement pathway, leading to uncontrolled C3 deposition in the glomeruli. The two subtypes, C3 glomeru...
Geographic atrophy is the advanced form of dry age-related macular degeneration, characterized by progressive loss of retinal pigment epithelium, photoreceptors, and choriocapillaris in well-defined a...
Hereditary Angioedema is a rare genetic disorder characterized by sudden, severe swelling (angioedema) of skin and mucous membranes affecting the face, hands, feet, gastrointestinal tract, and potenti...
IgA nephropathy is the most common primary glomerulonephritis worldwide, caused by abnormal IgA1 antibodies depositing in the kidney's mesangium, activating complement pathways and triggering inflamma...
Paroxysmal Nocturnal Hemoglobinuria is a rare, life-threatening blood disorder where complement-mediated destruction of red blood cells occurs due to acquired mutations in the PIGA gene. This leads to...
Patient Resources
Organizations and resources related to ADARx Pharmaceuticals's rare disease focus areas
Frequently Asked Questions About ADARx Pharmaceuticals
Common questions about ADARx Pharmaceuticals's rare disease programs, clinical trials, and treatments.