NEUWAY has established an in-house pipeline targeting severe orphan CNS diseases with very high medical need. The therapeutics will be delivered with NEUWAY’s proprietary delivery technology, EnPC®. This new therapeutic approach has the aim to have a transformative impact on the lives of patients suffering from orphan diseases with very limited treatment options.
Metachromatic leukodystrophy (MLD) is a genetically inherited lysosomal storage disease caused by the deficiency of an enzyme, Arylsulfatase A, which affects the metabolism of lipids needed for proper brain tissue functions. Without this enzyme, sulfatides build up, eventually destroying the isolating myelin sheath of the neuronal tissue and thus their function to transmit signals throughout the central and peripheral nervous systems. The destruction of the nervous system leads to muscle wasting up to paralysis, developmental delays, progressive loss of vision and dementia. Untreated, most children die by age of 4 – 5. The global prevalence of MLD is 1:50.000.
NEUWAY’s lead candidate is an mRNA encoding for the missing enzyme in MLD, Arylsulfatase A. Transported to its site of action, the brain cells, by NEUWAY’s proprietary delivery capsules, EnPCs®, the mRNA is translated into the functional enzyme thus avoiding or reducing the accumulation of toxic intermediates. At this stage of development, repeated i.v. dosing appears to be well-tolerated and does not harm BBB integrity.