Terry PirovolakisCEO at Elpida TherapeuticsSpeaker
Profile
CureSPG50, a Canadian charity, was founded by Terry Pirovolakis and his wife Georgia after there child received a Spastic Paraplegia Type 50 (SPG50) diagnosis in April 2019. By obtaining $4.5 million from generous donors and unwavering community support, Mr. Pirovolakis joined forces with academic sponsors to successfully demonstrate efficacy in both cells and mice, as well as preliminary safety in mice. He then assembled a team of experts to develop a tox vector, carry out toxicology studies in rats and NHPs, produce clinical-grade drugs, and, within three years, treat his own child through a Canadian CTA. The team proceeded to submit a Phase I/II application to the FDA, treating a second patient and anticipating a third to be treated by the time this proposal is assessed.
Elpida Therapeutics SPC: A social purpose corporation registered in California, was established by Mr. Pirovolakis in response to this significant unmet need, with the intention of advancing non-commercially viable, ultra-rare conditions from proof-of-concept to BLA to provide treatments for underprivileged children. This is accomplished through philanthropy and in-kind partnerships with top biotech, CDMO, and CRO organizations in the gene therapy field. Elpida operates as a non-profit, using all profits to support further programs. Presently, Elpida Therapeutics is working on SPG50, CMT4J, with three additional programs to be chosen via an application process and a scientific expert committee to advance academic and foundation programs.
Agenda Sessions
Innovation Station - Rapid Fire Case Studies Directly from the Thought Leaders Innovating in the Rare Disease Space
, 2:30pmView SessionBrain Storming Session - Hot Topic Break Down
, 3:45pmView Session