Sponsor

Orchard Therapeutics (Europe) Limited

United Kingdom 245 Hammersmith Road, W6 8PW, United Kingdom

About the sponsor

Orchard Therapeutics (Europe) Limited supports clinical research in Metachromatic Leukodystrophy, including late juvenile MLD and broader ARSA deficiency manifestations. The funded studies focus on neurological outcomes, disease progression, and restoration of enzyme activity in affected tissues.

MLD
Late juvenile MLD
Motor impairment
Residual ARSA activity

Research activity in this area is centered on gene therapy approaches designed for inherited neurodegenerative disease.

About Specializations

Orchard Therapeutics (Europe) Limited runs clinical trials in conditions such as Metachromatic leukodystrophy, Mucopolysaccharidosis I, among others.

All trials

Ongoing trials

Clinical trials registered by this sponsor – none is enrolling right now.

Quick filters

6 clinical trials

Metachromatic leukodystrophy

Not yet recruiting

Long‑Term Monitoring of Malignancy Risk in Early‑Onset Metachromatic Leukodystrophy Patients Treated with atidarsagene autotemcel

Registered drugNo placebo

Long‑Term Monitoring of Malignancy Risk in Early‑Onset Metachromatic Leukodystrophy Patients Treated with atidarsagene autotemcel

Registered drugNo placebo

France

Participants:0–17 years · 18–64 years
Substances:Atidarsagene Autotemcel
Sponsor:Orchard Therapeutics (Europe) Limited

Contact Research Centre

Mucopolysaccharidosis I

Not recruiting

Study on Gene Therapy with Autologous Stem Cells and Drug Combination for Children with Mucopolysaccharidosis Type I Hurler Variant

Registered drugNo placebo

Study on Gene Therapy with Autologous Stem Cells and Drug Combination for Children with Mucopolysaccharidosis Type I Hurler Variant

Registered drugNo placebo

Italy

Participants:0–17 years
Substances:Busulfan
Sponsor:Orchard Therapeutics (Europe) Limited

Contact Research Centre

Metachromatic leukodystrophy

Not recruiting

Study on Gene Therapy with Atidarsagene Autotemcel for Patients with Metachromatic Leukodystrophy

Registered drugNo placebo

Study on Gene Therapy with Atidarsagene Autotemcel for Patients with Metachromatic Leukodystrophy

Registered drugNo placebo

Italy

Participants:0–17 years
Substances:Atidarsagene Autotemcel
Sponsor:Orchard Therapeutics (Europe) Limited

Contact Research Centre

Mucopolysaccharidosis I

Not recruiting

Study on the Effectiveness and Safety of OTL-203 for Patients with Hurler Syndrome (MPS-IH) Compared to Standard Treatment with Stem Cell Transplantation

Registered drugNo placebo