STAT+: A rare disease program, portrayed as both lifeline and pharma handout, is set to expire
After his son Michael was diagnosed with a devastating, ultra-rare disease in 2019, Terry Pirovolakis dedicated himself to eradicating it.
Pirovolakis, a Canadian IT director, recruited academics and raised $3 million to design a gene therapy. He got Michael treated, an intervention that seems to have helped the 6-year-old stand on his heels for the first time and slow the course of a disease that can be severely cognitively and physically disabling.
Then, he founded a “public benefit corporation” in California to bring the treatment to approval, along with gene therapies for four other diseases too rare for traditional biotech and pharma to care about.
