Perlara signs rare disease pact with Novartis

Abstract

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