JR-171 (lepunafusp alfa)
Mucopolysaccharidosis I (with CNS involvement)
Phase 1/2Active
Key Facts
Indication
Mucopolysaccharidosis I (with CNS involvement)
Phase
Phase 1/2
Status
Active
Company
About JCR Pharmaceuticals
JCR Pharmaceuticals is a publicly traded, revenue-generating biopharma with a strategic focus on lysosomal storage disorders and other rare diseases. Its key achievement is the development and global commercialization of IZCARGO® (pabinafusp alfa), the first intravenously administered enzyme replacement therapy to demonstrate efficacy in treating both the somatic and central nervous system symptoms of Hunter syndrome (MPS II). The company's future direction is heavily invested in leveraging its J-Brain Cargo® platform to develop new treatments for neurodegenerative and other CNS disorders with high unmet medical need.
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