STAT The Readout - Some legit validation for Ethan Perlstein, founder of Perlara — the eponymous-ish biotech startup that’s developing drugs for rare diseases with a rather unorthodox social entrepreneurship model.
Perlara (née Perlstein Labs) just signed a deal with Novartis to develop drugs for lysosomal storage disorders. Novartis will also invest in the company, which is showing preclinical promise with potential therapies for Niemann-Pick type C disease.
Perlara works as a public benefit corporation — with the aim to create a cheaper way to discover new medicines. Think Toms, but with drugs.
So it's sidestepping traditional drug screening methods, and using CRISPR to engineer yeasts, worms, flies, and fish to express genes found in rare diseases. From there, Perlara screens tons of compounds on these simple organisms, and sifts through the data to come up with the best compounds to test in patients.
So far, so good.