Perlstein Lab, B Corp is a mission-driven biotech company that will develop a scalable evolutionary drug discovery platform for rare/orphan diseases.
1 in 12 people in the United States and in the world suffer from a rare disease. But most rare diseases afflict fewer than 6,000 patients. Of the more than 7,000 diagnosed rare diseases, only 450 have FDA approved therapies, which leaves hundreds of millions of patients globally with unmet medical needs. At the current glacial pace of orphan drug discovery – 10-20 approvals/year – it will take another 450 years before we have treatments for all rare diseases.
Perlstein Lab is the first orphan disease-focused biotech company founded as a Benefit corporation (B corp), a new corporate structure that enables us to balance profit motive with patient interests. We are building a scalable and personalized screening platform that leverages shared ancestry between humans and simple model organisms in order to generate high quality orphan drug candidates faster, cheaper and more systematically than current ad hoc approaches. We will initially focus on the ~50 recessive lysosomal storage diseases (LSDs), which afflict over 1M worldwide with an average of 50 different mutations per LSD gene. Several LSDs have enzyme replacement therapies, but the rest have no approved drugs.