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ICV-ERT (GM1 Gangliosidosis)
- Development stage
- Preclinical
- Lead developer
- TEGA Therapeutics
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes
- Administration
- Intracerebroventricular
01
Overview
ICV-ERT (GM1 Gangliosidosis), also known as NP-02, is an enzyme replacement therapy (ERT) being developed by Novel Pharma for the treatment of GM1 gangliosidosis. This rare, fatal lysosomal storage disorder is caused by a deficiency in the enzyme beta-galactosidase, leading to the toxic accumulation of GM1 gangliosides in the central nervous system (CNS). The therapy utilizes a recombinant version of the human beta-galactosidase enzyme, which is delivered directly into the brain's ventricles via an intracerebroventricular (ICV) route. This delivery method is intended to bypass the blood-brain barrier, ensuring that the therapeutic enzyme reaches the affected neural tissues to reduce substrate accumulation and mitigate neurodegeneration. The program is part of Novel Pharma's broader pipeline of ICV-delivered ERTs for brain-affecting lysosomal storage diseases.
- Other names
- intracerebroventricular enzyme replacement therapy (GM1 Gangliosidosis)NP-02NP02NP 02
02
Targets
GLB1 (Galactosidase Beta 1)
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