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ACG-801 is an enzyme replacement therapy comprising recombinant human acid ceramidase (rhAC), designed to address the genetic deficiency of the naturally occurring lysosomal enzyme acid ceramidase, which normally acts to metabolize ceramide. In CLN3-Batten disease, acid ceramidase deficiency and consequently altered sphingolipid signaling promote the disease phenotype; therapeutically, rhAC targeted both altered sphingolipid metabolism and retina degeneration in CLN3 disease models.
每项发明一条，涵盖美国、欧洲、PCT 与中国的同族专利。
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