From One to Many
from RARECast
When Terry Pirovolakis’ son Michael was diagnosed with the ultra-rare neurodegenerative disease spastic paraplegia type 50, he set out to raise money and engage researchers in developing a treatment. Now, after successfully dosing Michael with an experimental gene therapy as the first patient in a clinical trial, he has launched Elpida Therapeutics to develop multiple gene therapy programs for children with ultra-rare diseases. We spoke to Pirovolakis about the need Elpida is seeking to address, its unusual business model, and why he hopes to hand off its therapies at no cost to a partner once they win approval.
Embed this episode
What this episode covers
Terry Pirovolakis, who drove the development of a gene therapy for his son’s ultra-rare disease, discusses his newly launched Elpida Therapeutics, its unusual business model, and why it hopes to hand off its therapies at no cost to a partner once they win approval.
No transcript yet.