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Novel Pharma

South Korea

Ownership: Private
Development stage: Other

Overview

Novel Pharma specializes in rare disease therapeutics, particularly lysosomal storage disorders and central nervous system diseases. It is best known for Hunterase (idursulfase beta), an FDA-approved enzyme replacement therapy for Hunter syndrome, and for Hunterase ICV-ERT, the first treatment for severe-type Hunter syndrome delivered directly into the cerebrospinal fluid. The company is expanding its pipeline in rare neurodegenerative diseases, leveraging its expertise in biologics and disease biology.

Therapeutic areas: NeurologyRare Diseases
Modalities: Enzyme replacement therapyBiologic (recombinant protein)
Industry: Biotech

Drug pipeline

1 asset

ICV-ERT (Morquio Syndrome Type B)

Preclinical

Partnerships

Association for Research of MPS and Rare Diseases (ARMRD)

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