Page last updated: August 13, 2026 Page reviewed by: Dr. Cara O'Neill, FAAP For web accessibility options: Click/tap the floating blue icon on the right.Trial Summary “JR-446 is a novel drug developed based on the J-Brain Cargo technology, which has been clinically...
Enzyme Replacement Therapy
GC1130A Enzyme Replacement | MPS IIIA | Phase I | GC Biopharma
Page last updated: July 29, 2025 Page reviewed by: Dr. Cara O'Neill, FAAP For web accessibility options: Click/tap the floating blue icon on the right.Clinical Trial Summary Study:Â Phase 1 Study of GC1130A in Pediatric Patients With Sanfilippo Syndrome Type A (MPS...
DNL126 Enzyme Replacement | MPS IIIA | Phase I-II | Denali
Page last updated: February 6, 2026 Page reviewed by: Dr. Cara O'Neill, FAAP For web accessibility options: Click/tap the floating blue icon on the right.Clinical Trial Summary Study: “A Phase 1/2, Multicenter, Open-Label Study to Evaluate the Safety, Tolerability,...
JR-441 Enzyme Replacement | MPS IIIA | Phase I-II | JCR
Page last updated: August 30, 2024 For web accessibility options: Click/tap the floating blue icon on the right. Trial Information Phase I/​II Study of JR-441 in Patients With Mucopolysaccharidosis Type IIIA A Phase I/ II, open-label, randomized, 2-arm study, designed...
TA-ERT Enzyme Replacement | MPS IIIB | Phase I-II | Spruce Biosciences
Page last updated: June 12, 2026 For web accessibility options: Click/tap the floating blue icon on the right.Clinical Trial Summary Study: Intracerebroventricular Tralesinidase Alfa in Children With Mucopolysaccharidosis Type IIIB (TrAnsform) Trial Listing: Read this...