ALL-027

ALL-027 is an investigational enzyme replacement therapy (ERT) being developed by Phoenix Nest for the treatment of Sanfilippo syndrome Type D (Mucopolysaccharidosis type IIID; MPS IIID). MPS IIID is a rare, fatal neurodegenerative lysosomal storage disorder caused by a deficiency in the enzyme N-acetylglucosamine-6-sulfatase (GNS), which leads to the toxic accumulation of heparan sulfate in the central nervous system. ALL-027 consists of recombinant human GNS (rhGNS) produced in Chinese hamster ovary (CHO) cells. To effectively cross the blood-brain barrier and reach the brain tissues affected by the disease, the therapy is designed for intracerebroventricular (ICV) or intrathecal administration. The program has received funding from the NIH/NINDS and is currently in preclinical development, supported by data from a completed natural history study (ALL-127).

Other names

rhGNSrecombinant human alpha-N-acetylglucosamine-6-sulfataserecombinant human GNS