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LIN-2004
- Development stage
- Preclinical
- Lead developer
- Linno Pharmaceuticals
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes
01
Overview
LIN-2004 is an engineered galactocerebrosidase (GALC) enzyme being developed by Linno Pharmaceuticals as an enzyme replacement therapy (ERT) for the treatment of Krabbe disease (globoid cell leukodystrophy). Krabbe disease is a rare, fatal lysosomal storage disorder caused by a deficiency in GALC activity, which leads to the accumulation of psychosine and other galactolipids, resulting in progressive demyelination and neurodegeneration. LIN-2004 is designed to restore GALC activity, thereby reducing the toxic accumulation of psychosine and galactolipids. It is currently in the IND-enabling stage of development.
02
Targets
GALC (Galactosylceramidase)
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