Perlara announces n=1 drug discovery pact with Mission: Cure

SAN FRANCISCO, July 11, 2018 /PRNewswire/ -- Perlara, a rare diseases drug discovery platform company partnering with highly motivated families, foundations and drug developers, today announced a PerlQuest partnership to cure chronic pancreatitis with Mission: Cure, a nonprofit organization whose mission is to find a cure and improve the quality of life for those affected by chronic pancreatitis and in the process, demonstrate a new, faster model for curing disease using innovative financing based on patient outcomes.

Chronic pancreatitis is a painful and debilitating disease that affects about 150,000 children and adults in the US and over 1 million worldwide and is a risk factor for pancreatic cancer. The cystic fibrosis transmembrane conductance regulator (CFTR) gene is a common genetic driver of pancreatitis in patients of all ages. CFTR belongs to an evolutionarily conserved family of ABC-transporter-class ion channel proteins with homologs throughout the animal kingdom, including the gene YOR1 encoded by the model organism budding yeast, Saccharomyces cerevisiae.

In Stage One of the PerlQuest, Perlara will engineer ?F508 and 26 other CFTR disease-causing variants in yeast YOR1 models of CFTR deficiency and then complete drug repurposing and drug discovery screens with the ?F508 model and another mutant, e.g., class III or class IV. The goal is to discover compounds for rapid testing in patient-matched organoid models.

"We're thrilled to partner with an innovative and nimble patient advocacy group like Mission: Cure," says Perlara founder and CEO Ethan Perlstein, PhD. "We look forward to a scientific first for rare disease drug discovery: creating yeast patient avatars of shared and private loss-of-function CFTR mutations and then testing screening hits from yeast directly on pancreas-on-a-chip models derived from these patients."

"Mission: Cure was founded to use innovative financing based on patient outcomes to find a cure for chronic pancreatitis," says Mission: Cure co-founder and co-director Megan Golden.  "Our partnership with Perlara is key to discovering the underlying genetic pathways that impact pancreatic disease and will be instrumental in the discovery and development of effective therapies utilizing precision medicine technology."

"We are excited about the prospects that the new yeast models will bring," says Mission: Cure co-director Linda Martin. "Perlara has the expertise and platform needed to be successful in creating a very good yeast model that can be used for fast, effective drug discovery and screening. We hope to be able to move forward with further studies to repurpose existing drugs and/or develop effective, personalized treatments for chronic pancreatitis patients with CFTR and other genetic mutations."

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 PerlQueststm with families, patient organizations and BioPharma and clinical partners. The PerlArktm 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 Mission: Cure

Mission: Cure is a nonprofit organization fiscally sponsored by Cures Within Reach and supported by The National Pancreas Foundation to discover life-altering therapies and bring them to patients using outcomes-based financing and impact investing. For specific information about pancreatitis and venture philanthropy, please visit www.mission-cure.org.

SOURCE Perlara