AAV-NAGLU

AAV-NAGLU (AMT-110) is an investigational gene therapy originally developed by Institut Pasteur in collaboration with uniQure (formerly Amsterdam Molecular Therapeutics) for the treatment of mucopolysaccharidosis type IIIB (MPS IIIB), also known as Sanfilippo syndrome type B. It is a recombinant adeno-associated viral vector (rAAV2/5) consisting of a serotype 2 AAV genome packaged in a serotype 5 capsid, which encodes the human α-N-acetylglucosaminidase (NAGLU) cDNA. The therapy is designed to deliver a functional copy of the NAGLU gene directly to the central nervous system via intraparenchymal injection, thereby restoring NAGLU enzyme activity and preventing the accumulation of heparan sulfate that leads to progressive neurodegeneration. Although a Phase I/II clinical trial demonstrated long-term safety and persistent enzyme activity in the cerebrospinal fluid of treated children, uniQure discontinued the development of AMT-110 in 2016 as part of a strategic pipeline prioritization.

Other names

rAAV2/5-hNAGLUrAAV2/5-NAGLUAAV5-NAGLUAAV-5-NAGLUAAV 5-NAGLUAAV2/5-hNAGLU