Rare disease program, seen as lifeline and pharma boon, set to end

After his son Michael was recognized with a devastating, ultra-rare illness in 2019, Terry Pirovolakis devoted himself to eradicating it.

Pirovolakis, a Canadian IT director, recruited lecturers and raised $3 million to design a gene remedy. He obtained Michael handled, an intervention that appears to have helped the 6-year-old stand on his heels for the primary time and sluggish the course of a illness that may be severely cognitively and bodily disabling.

Then, he based a “public profit company” in California to convey the remedy to approval, together with gene therapies for 4 different illnesses too uncommon for conventional biotech and pharma to care about.