RGX-111 Gene Therapy in Patients With MPS I

Stopped The FDA placed a clinical hold on RGX-111 investigational gene therapy following an initial single case report of neoplasm (intraventricular CNS tumor) in a participant treated in its phase I/II study four years prior.

Conditions

• Mucopolysaccharidosis Type I (MPS I)
• Hurler Syndrome
• Hurler-Scheie Syndrome

Interventions

• GENETIC: RGX-111

Sponsor

REGENXBIO Inc.