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ICV-ERT (Morquio Syndrome Type B)
- Development stage
- Preclinical
- Lead developer
- Novel Pharma
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes
- Administration
- Intracerebroventricular
01
Overview
ICV-ERT (Morquio Syndrome Type B) is an investigational enzyme replacement therapy (ERT) being developed by Novel Pharma for the treatment of Morquio Syndrome Type B (MPS IVB), also known as Mucopolysaccharidosis Type IVB. MPS IVB is a rare lysosomal storage disorder caused by a deficiency of the enzyme beta-galactosidase, which leads to the accumulation of keratan sulfate and GM1 gangliosides. While systemic ERTs are available for some lysosomal storage diseases, they often fail to cross the blood-brain barrier effectively. This program utilizes an intracerebroventricular (ICV) delivery route to administer recombinant beta-galactosidase directly into the cerebral ventricles, aiming to address the central nervous system manifestations of the disease that are typically inaccessible via intravenous administration.
- Other names
- MPS IVBMorquio Syndrome Type B ERTICV-ERT (MPS IVB)Intracerebroventricular enzyme replacement therapy (MPS IVB)
02
Targets
IGF2R (Cation-independent mannose-6-phosphate receptor)LCT (Lactase-phlorizin hydrolase)GLB1 (Galactosidase Beta 1)
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