N=1 Drug Discovery Pact: Mission: Cure’s New Partnership with Perlara

By July 11, 2018News

Mission: Cure believes that new innovations in medical research are key to achieving our goal of curing chronic pancreatitis. For this reason, we strive to leverage and support advances in genomics and precision medicine and aim to collaborate with forward-thinking and mission-driven organizations. Today, we are excited to announce our partnership with Perlara, a rare disease drug discovery platform company working with highly motivated families, foundations and drug developers. Together, we have formed a n=1 drug discovery PerlQuest pact with the goal of discovering promising compounds for rapid testing in patient-matched organoid models of pancreatitis to get treatments to patients faster.

In Stage One of the PerlQuest, Perlara will engineer ∆F508 and 26 other CFTR disease-causing variants in yeast YOR1 models of CFTR deficiency with Mission: Cure’s support. The project will then involve completing drug repurposing and drug discovery screens with the ∆F508 model and another mutant, e.g., class III or class IV.

In the words of Perlara founder and CEO Ethan Perlstein, PhD., we look forward to scientific firsts for rare disease drug discovery.

For more information, check out Perlara’s recent press release on the exciting project: https://prn.to/2L7FTfC