RGX-111
Mucopolysaccharidosis Type I (MPS I)
Key Facts
About ReGenX Biosciences
ReGenX Biosciences is a clinical-stage biotechnology company dedicated to improving lives through the development of curative gene therapies. Its core asset is the NAV Technology Platform, a collection of over 100 novel AAV vectors that have become industry standards, powering its own pipeline and over 20 partnered programs, including the commercial therapy Zolgensma®. The company's dual-strategy of internal development and broad licensing has established a significant revenue stream and validates its platform's utility across multiple therapeutic areas.
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