Preclinical Programs | Amicus Therapeutics

Additional Preclinical Programs

Amicus Therapeutics has assembled an industry leading gene therapy portfolio for a broad range of rare genetic diseases including lysosomal disorders and some more prevalent rare diseases. The Company is developing a gene therapy portfolio of intrathecal AAV programs for neurologic lysosomal disorders through a license agreement with The Center for Gene Therapy at The Research Institute at Nationwide Children’s Hospital (NCH).

Amicus also has a broad gene therapy collaboration with the Gene Therapy Program in the Perelman School of Medicine at the University of Pennsylvania (Penn). Amicus is utilizing its protein engineering and glycobiology expertise along with Penn’s adeno associated virus (AAV) gene transfer technologies to develop AAV gene therapies designed for optimal cellular uptake, targeting, dosing, safety and manufacturability. There are six active preclinical programs in development with Penn for Fabry, Pompe, CDKL5 Deficiency Disorder (CDD), Next Generation MPS IIIA, MPS IIIB, and Niemann-Pick Type C. Under the collaboration agreement, Amicus also has worldwide rights to Penn’s next generation gene therapy technologies for a majority of lysosomal disorders as well as 12 more prevalent rare diseases, including Rett Syndrome, Angelman Syndrome, Myotonic Dystrophy and select other muscular dystrophies.