MPS I (mucopolysaccharidosis type I) is a lysosomal storage disorder caused by deficiency of alpha-L-iduronidase, leading to accumulation of glycosaminoglycans and progressive multisystem disease. Severity ranges from severe Hurler phenotype to attenuated forms.
A class action lawsuit was filed against REGENXBIO covering the period from February 9, 2022 to January 27, 2026, following FDA clinical holds on gene therapies RGX-111 and RGX-121 after a CNS tumor was discovered in a trial participant.
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The FDA placed clinical holds on Regenxbio's RGX-111 and RGX-121 gene therapies following a tumor case in an MPS I patient. The company's stock fell nearly 18% on the news.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07361536 |
Cardiac Structure and Function in MPS |
RECRUITING | |
| NCT05665036 |
Safety and Efficacy of Encapsulated Allogeneic MPS-1 Therapy |
WITHDRAWN | PHASE1/PHASE2 |
| NCT04958070 |
The Intensively Follow-up Examinations for Asymptomatic MPS I Infants in Taiwan |
UNKNOWN | |
| NCT04532047 |
PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders) |
RECRUITING | PHASE1 |
| NCT03333200 |
Longitudinal Study of Neurodegenerative Disorders |
RECRUITING | |
| NCT02702115 |
Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-318 in Subjects With MPS I |
TERMINATED | PHASE1/PHASE2 |
| NCT02067650 |
Ultrasound Findings of Finger, Wrist and Knee Joints in Mucopolysaccharidosis |
COMPLETED | |
| NCT01572636 |
Laronidase (Aldurazyme TM) Enzyme Replacement Therapy With Hematopoietic Stem Cell Transplant for Hurler Syndrome |
TERMINATED |
