Neurogene: Genes over ERT for monogenic CNS diseases

Neurogene is developing disease-modifying gene therapies for two monogenic, pediatric disorders in which enzyme replacement is not feasible or hasn't been explored.

Aspartylglucosaminuria (AGU) is a lysosomal storage disorder caused by autosomal recessive AGA mutations that lead to toxic accumulation of N-acetylglucosamines and cellular dysfunction in multiple tissue and cell types, including neurons. The disease is characterized by development delay and intellectual disability as well as psychomotor regression. ...