Perlara signs rare disease pact with Novartis

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.

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.