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Guided Cures  origins

Perlara 1: a rewarding journey of discovery

Perlara launched in 2014 as the first biotech Public Benefit Corporation (bioPBC). Our commitment to sustainability, open science and business and patient equity has guided our approach from day one. Visit the archived Perlara 1 website to see our roots.


Our base in the Bay Area allowed us to assemble an amazing team of scientists and cutting-edge laboratory to fullfill our mission to accelerate the discovery of cures for rare genetic diseases and uncover underlying mechanisms that enable the development of treatments that work across a range of diseases and individuals.  We developed the PerlArk™ Drug Discovery Platform, based on creating patient avatars using simple animals that share genetic similarity with humans, allowing us to screen massive numbers of disease models and drug candidates quickly and at low cost.


Perlara 2: success reveals new vistas

What we have learned is that our scientific and business approach works and has borne fruit with Maggie’s Pearl, a  joint venture between Perlara and Maggie’s Cure.  We have developed the first platform therapy for a group of related genetic inborn errors of metabolism called congenital disorders of glycosylation (CDG). Our cure candidate, Epalrestat, an aldose reductase inhibitor approved in Japan in 1992 for neuropathy in adults with diabetes, targets the root cause of PMM2-CDG, the most common CDG affecting over 1000 patients worldwide. 


Based on the success of our foundational research and Maggie's successful n-of-1 treatment, we launched a pivotal Phase 3 trial for up to 40 PMM2-CDG children (ages 2-17) at a single clinical trial site at the Mayo Clinic. The clinical protocol includes telemedicine visits. Research into treatments for other CDGs is planned. 


Perlara 1 also taught us that the success of our approach relies on the scientific acumen and leadership of our Perlara Cure Guides™. By eliminating the need for an expensive and geocentric laboratory, we are free to do what we do best— guide you and globally distributed research teams through the planning and development of your best cure and treatment paths that can synergistically lead to finding more potential cures for more diseases.


Perlara 1.0 proved that you could create joint ventures with individual families battling the long tail of disease. Perlara 2.0 will prove that you can build a fully decentralized biotech that partners with entrepreneurial families anywhere in the world and creates medicines spanning all therapeutic modalities regardless of the number of patients.


Ethan Perlstein

Founder and CEO


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