Rare Daily Staff

Ethan Perlstein, CEO of Perlara, announced on social media this week that the company will wind down its operations.

The company, which sought to accelerate the discovery of treatments for rare genetic diseases through the use of model systems, had developed a platform that allowed families, patient organizations, and researchers to enter into partnerships with the company to engage in drug discovery projects.

“The science was working and although from the outside it looked like the business was working the PerlQuest model had an Achilles heel painfully revealed when an early backer and partner bailed on a licensing deal after months of negotiations, triggering a fatal tail spin,” Perlstein wrote in a Facebook post. “We scrambled and schemed to do everything we could to salvage operations.”

Originally founded in February 2014 as Perlstein Lab, Perlara was the first biotech Public Benefit Corporation. Perlstein said the company was successful at finding clinically actionable, repurposable drugs (as well as new molecular entities) for two of its PerlQuests. Nevertheless, it couldn’t secure financing to advance these clinical-stage lead programs and ready the next ones.

Perlstein said he will do what he can to advance the discoveries made at Perlara into the clinic. He will work with the families involved in these discoveries on clinical, regulatory, and business development fronts to advance drugs approved here or abroad to clinical trials in the United States. The company’s most advanced programs were in congenital disorders of glycosylation (PMM2).

He said he would make available all data and discoveries from the company’s NGLY1 PerlQuest on an open-source basis.

“It sounds crazy, but Perlara felt like the closest thing to having a child with a rare disease,” he wrote. “So much was sacrificed for this company. The darkest moments were the guilt, shame, anger of failure.”

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