BioMarin’s plot to dominate gene therapy

JJ Bienaimé, the less-than-bashful CEO of BioMarin Pharmaceutical, has a less-than-bashful vision for his company: His company will win the arms race in gene therapy.

Thanks to years of infrastructure investment, BioMarin can now churn out 10,000 commercial-grade doses of gene therapy a year, something “nobody else is able to do in the field,” Bienaimé told STAT. In practical terms, that means valrox, BioMarin’s gene therapy for hemophilia A, could treat every eligible patient in the U.S. within about two years of winning approval, according to Bienaimé. If that approval comes later this year, as expected, BioMarin has a chance to seize the market before competitors Roche and Pfizer can sell a single infusion, he said.

And that’s just the first step. Bienaimé looks at BioMarin’s gene therapy division as a perpetual motion machine. Everything the company learned about making valrox went into its next gene therapy, targeting the rare disease phenylketonuria, and will be applicable to one-time treatments yet to come.