Amicus Therapeutics Presents Positive Preclinical Pompe Disease Gene Therapy Data at the American Society of Gene & Cell Therapy (ASGCT) 22nd Annual Meeting

In what could potentially be very good news for Pompe patients, Amicus Therapeutics presents positive data from their preclinical gene therapy program for Pompe disease. The results are presented in the poster session at the ASGCT annual meeting today. This is initial preclinical data from their investigational adeno-associated viral (AAV) gene therapy program for Pompe disease in mice.

This initial preclinical study used a single high dose of AAV in GAA knockout mice with either natural unmodified hGAA (“natural hGAA”) or an Amicus/Penn engineered hGAA transgene with a lysosomal-targeting cell receptor binding motif (“engineered hGAA”). The Amicus/Penn engineered hGAA is designed for optimal expression, secretion, and targeting which enables efficient cross-correction in target tissues (via the binding motif).

The Amicus/Penn hGAA AAV gene therapy demonstrated more uniform cellular uptake and lysosomal targeting compared to natural hGAA AAV gene therapy. Interestingly, the engineered hGAA AAV gene therapy showed glycogen reduction in neuronal cells, which suggests that this may be an effective way to address neuronal aspects of Pompe disease.

Hung Do, PhD, Chief Science Officer of Amicus Therapeutics, stated, “These very important preclinical results validate our capabilities to develop engineered GAA proteins that can efficiently cross-correct target cells and tissues via a gene replacement therapy for Pompe disease. This approach may be applicable to other lysosomal disorders as we continue to combine our Amicus protein engineering expertise, together with Penn’s vector engineering expertise, to develop novel gene therapies.”

John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, added, “Developing a potential cure for Pompe has been a personal and professional goal for many years. These data are profound and it is extremely rewarding to see these preclinical results that show our Amicus engineered GAA is optimized for uptake into target tissues and gets to the right cellular compartments, especially in the central nervous system. These data also provide preliminary and compelling evidence that the Amicus technology to design constructs that enhance protein targeting may be a significant platform for multiple lysosomal disorders. As these data exceed our expectations, our preclinical studies are progressing well ahead of schedule and we now expect to select a clinical candidate in 2019 to move forward into IND-enabling studies.  Our mission has always been to develop potential best in class medicines and that is precisely what we are doing with both our novel, late-stage treatment paradigm AT-GAA as well as this preclinical gene therapy program.”

Preclinical work gives an indication of potential for a new therapy, but it’s early. A lot can change between now and when this is ultimately available. It is good to see their gene therapy program moving forward.

The poster can be found here.

Tagged: gene therapy, preclinical