Orchard Therapeutics
Rare disease drugmaker · part of Kyowa Kirin · based in London, United Kingdom · founded 2015 · orchard-tx.com
Orchard Therapeutics works on 9 rare diseases we track, including Cerebral Adrenoleukodystrophy, Familial Chylomicronemia Syndrome and 7 more. It has 4 active clinical trials, and 1 is looking for volunteers now. It also has 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 the registrational HURCULES trial (NCT06149403), which compares it with donor stem cell transplant; Orchard said on July 7, 2025 that the last patient had been treated.
Orchard Therapeutics pipeline
Each row lists the diseases Orchard Therapeutics is studying at that stage, from the ones closest to approval down to the earliest work.
Mucopolysaccharidosis IH
Leukodystrophy recruiting
Orchard Therapeutics clinical trials
These are Orchard Therapeutics trials for the rare diseases we track, from ClinicalTrials.gov. Statuses there can lag behind company news, so confirm with the study team before you plan around one.
1 trial looking for volunteers now
The Myelin Disorders Biorepository Project
For Leukodystrophy, White Matter Disease, Leukoencephalopathies, 4H Syndrome and Adrenoleukodystrophy
3 trials underway, not taking new volunteers
OTL-200 in Patients With Late Juvenile Metachromatic Leukodystrophy (MLD)
Tests OTL-200
For Lysosomal Storage Diseases and Metachromatic Leukodystrophy
Gene Therapy With Modified Autologous Hematopoietic Stem Cells for the Treatment of Patients With Mucopolysaccharidosis Type I, Hurler Variant
Tests Frozen autologous CD34+ hematopoietic stem and progenitor cells genetically modified with the lentiviral vector IDUA LVV, encoding for the α-L-iduronidase cDNA, in their final formulation medium.
For Mucopolysaccharidosis IH
A Study to Investigate the Efficacy and Safety of OTL-203 in Subjects With MPS-IH Compared With Standard of Care With Allogeneic HSCT
Tests Experimental: OTL-203, Active Comparator: Allo-HSCT
Pick a condition. One email when a new trial opens, a trial changes or the FDA decides, from any company, not only Orchard Therapeutics. Unsubscribe anytime.
- Trial Alerts, when a new trial opens or a trial changes
- Disease Updates, FDA decisions and condition news, usually 1 or 2 emails a month
FDA-approved drugs from Orchard Therapeutics
From the openFDA database. Tap a drug for side effects, dosing and help paying.
Lenmeldy atidarsagene autotemcel
More about 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 active and recruiting rare disease 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.
Where Orchard Therapeutics trials take place
The trials above run at 31 study sites in 4 countries. Tap a country to see its states, cities and hospitals.
Rare diseases Orchard Therapeutics works on
Each disease page lists every open trial, not only Orchard Therapeutics's, plus approved treatments.
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.
How common: 1 in 20,000 males worldwide; cerebral form develops in approximately 35-40% of boys with X-ALD
Familial Chylomicronemia Syndrome
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.
How common: 1 to 2 per 1,000,000 people globally
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.
How common: 1 in 100,000 to 150,000 males; females with disease are rare
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.
How common: Approximately 1 per 40,000 to 1 per 160,000 live births globally; variable by region
Mucopolysaccharidosis Type I (MPS I) is a lysosomal storage disorder caused by deficiency of the enzyme alpha-L-iduronidase, which breaks down glycosaminoglycans (GAGs).
How common: Approximately 1 per 100,000 live births; Hurler form is the most common phenotype
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.
How common: Approximately 1 per 70,000 to 1 per 100,000 live births; highest prevalence in certain populations including Australia and Scandinavia
Severe Combined Immunodeficiency
Severe combined immunodeficiency is a rare genetic disorder affecting both T cell and B cell immune function, resulting in severe susceptibility to all types of infections.
How common: 1 in 58,000 births in US newborn screening
Thalassemia is an inherited blood disorder where reduced or absent production of one type of hemoglobin chain causes anemia, from mild or none in carriers to severe in the major forms, where it can...
How common: About 1 in 100,000 people have symptoms of beta thalassemia, according to NORD, most often people with Mediterranean, Middle Eastern, African or Asian roots
Where to get support
Patient groups can explain a diagnosis, point you to specialists and tell you about trials before they open. Orchard Therapeutics's own site is orchard-tx.com, and our help paying for treatment page lists drugmaker programs and charity funds.
- NIH GARD - Hunter SyndromeHunter Syndrome and Severe Combined Immunodeficiency
- ALD AllianceCerebral Adrenoleukodystrophy
- United Leukodystrophy FoundationCerebral Adrenoleukodystrophy
- NORD - Familial Chylomicronemia SyndromeFamilial Chylomicronemia Syndrome
- StatPearls - Familial ChylomicronemiaFamilial Chylomicronemia Syndrome
- National Hunter Syndrome SocietyHunter Syndrome
- United Leukodystrophy FoundationMetachromatic Leukodystrophy
- National Center for Advancing Translational Sciences (NCATS)Metachromatic Leukodystrophy