# A father’s crusade in rare disease drug development: a case study of Elpida therapeutics and Melpida

## TL;DR

A father collaborated with experts to rapidly develop a gene therapy for his son's rare disease and created a company to help others in similar situations.

## Contribution

This case study highlights a successful caregiver-driven model for rare disease drug development and identifies actionable lessons for others.

## Key findings

- Melpida was developed in 36 months through collaboration between a caregiver and biomedical professionals.
- Elpida Therapeutics was established as a social purpose corporation to replicate this success for other rare diseases.
- Four key lessons were identified to guide caregivers in developing novel gene therapies.

## Abstract

Therapeutic development for rare diseases is difficult for pharmaceutical companies due to significant scientific challenges, extensive costs, and low financial returns. It is increasingly common for caregivers and patient advocacy groups to partner with biomedical professionals to finance and develop treatments for rare diseases. This case study illustrates the story of Terry Pirovolakis, a father who partnered with biomedical professionals to develop the novel gene therapy, Melpida, within 36 months of the diagnosis of his infant son. We identify the factors that led to the success of Melpida and analyze the business model of Elpida Therapeutics, a social purpose corporation founded by Pirovolakis to reproduce the success of Melpida for other rare diseases. We conclude with four lessons from Melpida to inform caregivers like Pirovolakis on developing novel gene therapies to save their loved ones.