Rare Disease Report

Advocacy Groups as Investors

JANUARY 11, 2017
James Radke, PhD
Perlara PBC, is one of those great rare disease, ‘think outside the box’ success stories.
Originally called Perlstein Labs PBC and founded by Ethan Perlstein, PhD, the premise was to develop animal models to speed up the drug discovery process for rare diseases.
The company started because Dr. Perlstein neither wanted to remain in academia nor work for big pharma – the 2 options he was told he had to pursue. He decided to create a 3rd option – a PBC.
The PBC in the company name refers to it being a Public Benefit Corporation, a new class of corporation that allows companies to pursue profit as well as a strong social or environmental mission. The PBC allows the company to be both academically (conducting science to advance science) and fiscally (develop orphan drugs) based.
And to succeed, Perlara is partnering with both advocacy groups and big pharma. Last fall, the company announced a partnership with Novartis on a drug discovery program for Niemann-Pick type C disease and other lysosomal disorders. And this week, Perlara announced a partnership with 2 patient advocacy groups—the Grace Science Foundation ( NGLY1 Deficiency) and the Wylder Nation Foundation ( Niemann-Pick Type A (NPA)/Acid Sphingomyelinase Deficiency).

The partnerships are under the company’s ‘PeriQuests’ platform.

Advocacy Groups as Investors

PerlQuests are drug discovery collaborations funded directly by patient groups, who in exchange receive joint ownership of the resulting data and inventions.
Dr Perlstein explained, "The goal of the PerlQuest model is to democratize venture philanthropy and make it possible for any patient advocacy group anywhere in the world to be equal partners in their own personalized drug discovery journey."
PerlQuests begin with a natural history study of the disease in model organisms, progress to a drug-repurposing screen, and culminate in a high-throughput drug screening campaign to discover novel compounds suitable for preclinical validation in mice.
Both advocacy groups are thrilled with the new partnership.
Matt Wilsey, President of Grace Science Foundation said, "We've worked closely with the Perlara team since the company's formation. We are proud to be a launch partner of PerlQuests and are excited about the drug leads that will emerge."
Wilsey added, "Perlara's nimble platform allows it to quickly iterate and in turn cut down the time when NGLY1 patients can receive a life-saving treatment."
Steven Laffoon, Co-Founder and President of Wylder Nation Foundation NGLY1 patients can receive a life-saving treatment stated, "Perlara's unique drug discovery platform and business model is a perfect fit for advancing our efforts in discovering existing compounds that can be repurposed, and new novel compounds that can be optimized for treating Niemann-Pick Type A."
To learn more about Perlara, click here.

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