RGX-121
Mucopolysaccharidosis Type II (MPS II, Hunter syndrome)
Key Facts
Indication
Mucopolysaccharidosis Type II (MPS II, Hunter syndrome)
Phase
Phase 1/2
Status
Active
Company
About REGENXBIO
REGENXBIO is a publicly traded gene therapy leader built around its foundational NAV Technology Platform, comprising exclusive rights to over 100 novel AAV vectors, including the high-performing AAV8 and AAV9. The company's strategy combines advancing its own clinical pipeline, most notably in wet AMD with RGX-314, with a lucrative licensing model that includes partnerships with major biopharma companies like Novartis, Pfizer, and Takeda. With multiple clinical readouts expected in the near term and a strong financial position, REGENXBIO is positioned to be a key player in the next generation of gene therapies.
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