Rare Disease Parent Co-Founds Gene Therapy Company Focused on Ultra-Rare Populations

Rare Daily Staff

Elpida Therapeutics, a social purpose corporation founded to develop gene therapies for patient populations too small to attract the interest of biopharmaceutical companies, launched with about $20 million in cash and in-kind contributions.

As a social purpose corporation, Elpida said it looks to combine sustainability along with broader socially responsible objectives. Elpida co-founder and CEO Terry Pirovolakis, whose son Michael was treated with an experimental gene therapy Melpida to treat his ultra-rare neurodegenerative disease spastic paraplegia 50.

Elpida will focus on advancing programs that traditional biotech companies are finding difficult to bring through to completion. The company is working to bring in programs that were deprioritized by biotech companies or that did not receive biotech investment to advance due to small patient populations, but which have excellent data on which to build a program and springboard the science and opportunity. Melpida is the company’s lead experimental therapy, and it is building a pipeline of similarly situated pre-clinical CNS gene therapy programs. Elpida will seek to advance a total of five programs to round out the initial program pipeline.

“After working on a successful clinical program for my son who has an ultra-rare disease called SPG50 that was developed and delivered in a clinical trial in less than three years leveraging numerous key partners and industry experts, I knew there had to be a way to help other small foundations and families speed up the path that I embarked upon when I set out to save my son,” said Pirovolakis. “ElpidaTx’s mission will be to turn around the current significant unmet needs of patients and leverage scientific advancements and gene therapy maturity which will put cures in reach of families and children who desperately need them using a model that will allow us to help more children, while being self-sustaining and replicable.”

Elpida Co-Founder Laura Hameed, said the launch “marks a new era in how the biotechnology ecosystem can think about the advancement of ultra-rare diseases programs.” She said she believes the social purpose model may be the ideal model of the future for the advancement of expensive gene therapy programs for diseases with very small patient populations.

Photo: Terry Pirovolakis, co-founder and CEO of Elpida Therapeutics