Perlara Enters Collaboration with the University of Notre Dame Focused on Cori Disease and Pompe Disease

Rare Daily Staff

Perlara said it has entered into a collaboration with the University of Notre Dame and the Warren Family Research Center for Drug Discovery and Development focused on Cori Disease and Pompe disease, two glycogen storage diseases.

Glycogen storage diseases are monogenic recessive inborn errors in metabolism involving genes that synthesize or degrade glycogen in cells and tissues of the body. Cori Disease is caused by deficiency of the enzyme amylo-alpha-1,6-glucosidase, or AGL. Pompe Disease is caused by deficiency of the lysosomal enzyme acid alpha-glucosidase, or GAA.

Perlara’s business model is to enter into discovery and drug development collaborations with  families, patient organizations, biopharmaceutical, and clinical partners.

During the first stage of the collaboration, Perlara will engineer, phenotype, adapt nematode and fly models of Cori and Pompe diseases to its screening pipeline and screen a collection of compounds to identify drugs that could be repurposed to treat the diseases.

“Our platform is well suited to evolutionarily and functionally conserved genes and pathways such as those involved in glycogen storage diseases like Cori and Pompe, and is designed for image-based phenotypic readouts of growth, development, morphology, and behavior,” said Ethan Perlstein, founder and CEO of Perlara.  “With the addition of GSDs, we deepen our commitment to lysosomal diseases and by extension lysosomal biology.”

August 15, 2018
Photo: Ethan Perlstein, founder and CEO of Perlara