GC1130A Enzyme Replacement | MPS IIIA | Phase I | GC Biopharma/Novel Pharma

May 22, 2024

Page last updated: May 22, 2024

Page reviewed by: Dr. Cara O’Neill, FAAP

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Clinical Trial Summary

Study: Title pending official listing on ClinicalTrials.gov

Trial Listing: Pending

Study opened: Pending

Study design: Pending

Study status: Pending

Route of administration: ICV ERT (intracerebral ventricular enzyme replacement therapy)

Study length: Pending

Study sites: Planned for the U.S., Korea and Japan

Brief Summary of Trial

Pending official listing on ClinicalTrials.gov

Press Releases, web articles, presentations regarding this study

  • In May 2024, GC Biopharma/Novel Pharma issued a press release announcing its Sanfilippo Syndrome treatment had obtained FDA IND Approval
  • In August 2023, SooKyung Shin, R. Ph, M.S, and Dong-Kyu Jin, MD, PhD, of GC Biopharma/Novel Pharma presented to the community during Cure Sanfilippo Foundation’s ADVANCE 2023 conference. Their presentation can be viewed below.

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