Orchard Therapeutics
Orchard Therapeutics works on 9 rare diseases tracked on Trial Friend, including Cerebral Adrenoleukodystrophy, Familial Chylomicronemia Syndrome, Hunter Syndrome and 6 more, with 3 recruiting clinical trials and 1 FDA-approved rare disease drug.
Orchard Therapeutics, acquired by Kyowa Kirin in January 2024, develops hematopoietic stem cell gene therapies for rare genetic diseases. Lenmeldy (atidarsagene autotemcel) received FDA approval in March 2024 for early-onset metachromatic leukodystrophy. OTL-203 for MPS I (Hurler syndrome) is in clinical development.
Focus areas at Orchard Therapeutics
As a rare disease specialist, Orchard Therapeutics has active clinical trial programs and drug development efforts across 9 rare diseases, including Cerebral Adrenoleukodystrophy, Familial Chylomicronemia Syndrome, Hunter Syndrome, Metachromatic Leukodystrophy, Mucopolysaccharidosis Type I, and 4 additional rare conditions. 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 Orchard Therapeutics, 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.
Orchard Therapeutics is headquartered in London, United Kingdom, founded in 2015, a subsidiary of Kyowa Kirin. 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.
Orchard Therapeutics Drug Pipeline
Orchard Therapeutics 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 Orchard Therapeutics'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.
Orchard Therapeutics Clinical Trials (3)
Active and recruiting clinical trials sponsored by Orchard Therapeutics, 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.
Orchard Therapeutics FDA-Approved Drugs (1)
Medications developed or marketed by Orchard Therapeutics that have received U.S. Food and Drug Administration approval. Drug data is sourced from the openFDA database and includes brand names, generic names, approval dates, and matched rare disease indications. Where a drug treats a condition covered by Trial Friend, the disease name links directly to that disease page.
| Drug Name | Brand Name | Rare Diseases | Approval Date |
|---|---|---|---|
| ATIDARSAGENE AUTOTEMCEL | LENMELDY intravenous | — |
Orchard Therapeutics Trial Locations
Orchard Therapeutics clinical trials are running at 7 sites in 4 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 (9)
Diseases targeted by Orchard Therapeutics's clinical trial and drug development programs
Cerebral adrenoleukodystrophy is the most severe form of X-linked adrenoleukodystrophy, caused by mutations in the ABCD1 gene that impair breakdown of very-long-chain fatty acids. These accumulate in ...
Familial Chylomicronemia Syndrome is a rare inherited metabolic disorder where the body cannot break down certain fats (triglycerides) normally, causing dangerously high blood fat levels. People with ...
Hunter syndrome is an X-linked lysosomal storage disorder caused by deficiency of iduronate-2-sulfatase enzyme, leading to accumulation of glycosaminoglycans in cells and tissues. This multisystem dis...
Metachromatic Leukodystrophy (MLD) is a lysosomal storage disorder resulting from deficiency of the enzyme arylsulfatase A (ARSA), which breaks down sulfatides, critical components of myelin. Deficien...
Mucopolysaccharidosis Type I (MPS I) is a lysosomal storage disorder caused by deficiency of the enzyme alpha-L-iduronidase, which breaks down glycosaminoglycans (GAGs). This results in accumulation o...
Sanfilippo syndrome, formally mucopolysaccharidosis type III (MPS III), is a lysosomal storage disorder in which the body cannot break down a sugar chain called heparan sulfate. It builds up mainly in...
Patient Resources
Organizations and resources related to Orchard Therapeutics's rare disease focus areas
Frequently Asked Questions About Orchard Therapeutics
Common questions about Orchard Therapeutics's rare disease programs, clinical trials, and treatments.