Perlara signs rare disease pact with Novartis | October 24, 2016 Issue - Vol. 94 Issue 42 | Chemical & Engineering News
Volume 94 Issue 42 | p. 13 | News of The Week
Issue Date: October 18, 2016 | Web Date: October 19, 2016

Perlara signs rare disease pact with Novartis

Start-up seeks to advance its unusual business model with a big pharma partner
Department: Business
Keywords: start-ups, biotech, pharmaceuticals, rare diseases

In a critical validation for a biotech firm espousing an unusual business model, Perlara has struck a deal to develop lysosomal storage disorder treatments with Novartis. The big pharma company will also make an undisclosed investment in the rare-disease-focused firm.

Credit: Perlara
Photo of Ethan Perlstein.
Credit: Perlara

Launched in 2014 as a public benefit corporation, Perlara is the brainchild of Ethan Perlstein, a molecular biologist who left academia in 2012 to pursue independent research. Perlara seeks an efficient way of developing drugs for rare diseases while living up to the transparency and sustainability tenants of the public benefit model.

Perlara uses the gene-editing tool CRISPR to generate model organisms—yeast, nematodes, fruit flies, and worms—that feature the mutation driving a particular rare disease. Small molecules are tested directly in those organisms, an approach Perlstein believes will yield starting molecules that are safer and less in need of tweaking than the hits that come out of traditional target-based drug screens.

Indeed, in just a year, Perlara has achieved promising results against Niemann-Pick type C (NPC) disease, a fatal disorder driven by a genetic mutation that causes cholesterol and other fat to build up inside lysosomes. Novartis gains access to the biotech’s lead NPC compound, which is currently being tested in mice.

Not including the Novartis investment, Perlara has brought in close to $5 million in seed funding. Its next goal is to raise $10 million in Series A financing—a feat that will require convincing venture capital firms that its model can work for rare disease research.

With the Novartis deal providing helpful affirmation of the company’s technology, Perlstein hopes to secure the funding by the end of this year. The cash will help Perlara toward its next phase: partnering directly with rare disease patient groups in need of treatments.

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Siamak Dana. (October 19, 2016 1:07 PM)
Hello. Ethan;
October 19, 2016
Congratulations are due on your success. I do not understand the fine points of this achievement, but trust Novartis, the Swiss farmaceutucal, has realized the potentials of this to invest money in it. As we all know, the Swiss are not known for generous contribution!
I wish you even more success in future.
Siamak Dana

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