Amicus, Penn expand gene therapy research agreement into five-year collaboration
Apart from getting the FDA to have a change of heart on its Fabry disease therapy, Galafold, last year, Amicus Therapeutics joined forces with the University of Pennsylvania to work on gene therapies for four different disorders — Pompe disease, Fabry disease, CDKL5 deficiency and an undisclosed rare metabolic disorder. On Wednesday, the two expanded their collaboration — to include additional lysosomal disorders and rare diseases.
The partnership was inked to bring together Penn’s AAV vector technology (AAVs are small, non-pathogenic viruses with a genome of single-stranded DNA, which can insert genetic material at a specific site), which is engineered to enhance targeting, safety and gene delivery — with Amicus’ $FOLD technology designed to optimize protein expression, secretion, and the uptake of the target protein.
Under the new five-year agreement, the two entities will also work together on gene therapies for Niemann-Pick Type C (NPC) and two forms of Sanfilippo Syndrome. In addition, Amicus has the opportunity to collaborate with Penn’s Gene Therapy Program (GTP) to develop programs for the majority of lysosomal disorders and 12 additional rare diseases, including Rett Syndrome, Angelman Syndrome, Myotonic Dystrophy and select other muscular dystrophies. Amicus will inject $10 million annually for five years into GTP’s discovery research program — with the ability to extend.
Amicus’ experimental drug for Pompe disease (an inherited lysosomal storage disorder), AT-GAA, did not generate much enthusiasm for accelerated review by the FDA, despite CEO John Crowley’s best efforts last September.
The company’s work with Penn on Pompe disease, meanwhile, has generated positive data.
“We have already seen highly encouraging preclinical results and proof-of-concept in Pompe disease through our existing collaboration…and I believe that we can further expand and accelerate our efforts to rapidly develop gene therapies for many more patients,” said James Wilson, professor of medicine and pediatrics at Penn’s Perelman School of Medicine, in a statement.