Perlara announces Glycogen Storage Diseases PerlQuest with the University of Notre Dame
South San Francisco, California, August 15, 2018
Perlara, a rare diseases drug discovery platform company partnering with highly motivated families and organizations, today announced a glycogen storage diseases collaboration with the University of Notre Dame and the Warren Family Research Center for Drug Discovery and Development.
Glycogen storage diseases (GSD) are monogenic recessive inborn errors in metabolism involving genes that synthesize or degrade glycogen in cells and tissues of the body. Cori Disease is a GSD Type III caused by deficiency of the enzyme amylo-alpha-1,6-glucosidase, or AGL. Pompe Disease is a GSD Type II caused by deficiency of the lysosomal enzyme acid alpha-glucosidase, or GAA.
In Stage One of the PerlQuest, Perlara will engineer, phenotype, adapt nematode and fly models of Cori and Pompe diseases to our screening pipeline and screen the Microsource Spectrum collectionto identify repurposable compounds that rescue the disease.
“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,” says Perlara founder and CEO Ethan Perlstein, Ph.D. “With the addition of GSDs, we deepen our commitment to lysosomal diseases and by extension lysosomal biology.”
“We are excited to add Perlara to our collaborative project team seeking therapeutic leads for Cori and Pompe disease,” says University of Notre Dame Professor and Warren Center Researcher Richard Taylor. “Perlara’s expertise in the development and screening of genetically engineered model systems complements our current efforts in this area and will accelerate the identification of small molecules as potential treatments for GSD patients.”
About Perlara PBC
Perlara is the first biotech public benefit corporation (bioPBC) that discovers cures for rare diseases and learns how genes work across diseases to map connections to common ailments. Perlara embarks on journeys of scientific discovery and drug development called PerlQuests™ with families, patient organizations and BioPharma and clinical partners. The PerlArk™ Platform creates disease models using simple animals that share genetic similarity with humans, allowing screening of massive numbers of disease models and drug candidates quickly and at low cost.
For general information, visit www.perlara.com.
About the Warren Family Research Center for Drug Discovery and Development
The Warren Family Research Center for Drug Discovery and Development (Warren Center) at the University of Notre Dame is a state-of-the-art resource for a highly productive and renowned group of drug discovery faculty with expertise and interest in areas such as neurological and central nervous system disorders, infectious disease, cancer and rare diseases. The Warren Center supports the organization of chemical research discoveries for the Notre Dame Chemical Compound Collection and promotes biological evaluation of the products through internal collaborations and external partnerships with the aim of bringing healing therapies to market. Visit drugdiscovery.nd.edu for more information.
About the University of Notre Dame
The University of Notre Dame is a private research and teaching university inspired by its Catholic mission. Located in South Bend, Indiana, its researchers are advancing human understanding through research, scholarship, education, and creative endeavor in order to be a repository for knowledge and a powerful means for doing good in the world. Visit nd.edu for more information.