A father’s crusade in rare disease drug development: a case study of Elpida therapeutics and Melpida
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13023_2025_Article_3892.pdf
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Author(s)
Portero, Deanna
Xu, Qingyang
Hussain, Aaliya
Lo, Andrew W.
Date Issued
July 16, 2025
Journal
Orphanet Journal of Rare Diseases
Publisher
BioMed Central
Citation
Portero, D., Xu, Q., Hussain, A. et al. A father’s crusade in rare disease drug development: a case study of Elpida therapeutics and Melpida. Orphanet J Rare Dis 20, 363 (2025).
Version
Final published version
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.
MIT Department
Sloan School of Management. Laboratory for Financial Engineering
Sloan School of Management
Massachusetts Institute of Technology. Computer Science and Artificial Intelligence Laboratory
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Creative Commons Attribution
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DOI of Published Version
https://doi.org/10.1186/s13023-025-03892-0
