Mucopolysaccharidosis II
Pipeline by Development Stage
Drug Modality Breakdown
Competitive Landscape
9 companies ranked by most advanced pipeline stage
Trial Timeline
Clinical trial activity over time
Showing 15 of 19 trials with date data
Clinical Trials (19)
Total enrollment: 709 patients across 19 trials
An Extension Study of JR-141 to Evaluate the Long-term Safety and Efficacy in MPS II (Hunter Syndrome) Subjects
A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II (STARLIGHT)
An Extension Study of the Long-Term Safety, Tolerability, and Efficacy of Tividenofusp Alfa (DNL310) in Participants With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007
A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and Young Adult Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)
An Extension Study of JR-141 in Patients With Mucopolysaccharidosis Type II
A Study of JR-141 in Patients With Mucopolysaccharidosis II
Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II)
An Extension Study of JR-141-BR21 in Patients With Mucopolysaccharidosis II
A Study of JR-141 in Patients With Mucopolysaccharidosis II (BR21)
Study of Idursulfase-beta (GC1111) in Hunter Syndrome
A Study of Tividenofusp Alfa (DNL310) in Pediatric Participants With Hunter Syndrome
Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II
A Study of JR-141 in Patients With Mucopolysaccharidosis Type II
To Evaluate the Safety and Efficacy of GC1111 (Recombinant Human Iduronate-2-sulfatase) in Hunter Syndrome Patients
Phase I/II Study of Retroviral-Mediated Transfer of Iduronate-2-Sulfatase Gene Into Lymphocytes of Patients With Mucopolysaccharidosis II (Mild Hunter Syndrome)
A Study to Evaluate the Safety, Tolerability, PK and PD of Intracerebroventricular GC1123 in Patients with MPS Ⅱ
Long-term Follow-Up for RGX-121
Mucopolysaccharidosis Type II Natural History
A Study to Test the Possibility of Cross Reaction Induced by the Idursulfase Drug to GSK2788723
Phase Legend
Key Insights
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