ICV-ERT (GM1 Gangliosidosis)

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 02intracerebroventricular enzyme replacement therapy-Novel Pharma-enzyme replacement therapy (ERT)-ICV delivery-Intracerebroventricular (ICV)-GM1 Gangliosidosis