A father’s crusade in rare disease drug development: a case study of Elpida therapeutics and Melpida
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...
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| Main Authors: | , , , |
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| Format: | Article |
| Language: | English |
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BMC
2025-07-01
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| Series: | Orphanet Journal of Rare Diseases |
| Online Access: | https://doi.org/10.1186/s13023-025-03892-0 |
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| author | Deanna Portero Qingyang Xu Aaliya Hussain Andrew W. Lo |
| author_facet | Deanna Portero Qingyang Xu Aaliya Hussain Andrew W. Lo |
| author_sort | Deanna Portero |
| collection | DOAJ |
| description | 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. |
| format | Article |
| id | doaj-art-2b40a8ac1e514b7ba437a85019d5a7e8 |
| institution | DOAJ |
| issn | 1750-1172 |
| language | English |
| publishDate | 2025-07-01 |
| publisher | BMC |
| record_format | Article |
| series | Orphanet Journal of Rare Diseases |
| spelling | doaj-art-2b40a8ac1e514b7ba437a85019d5a7e82025-08-20T03:06:05ZengBMCOrphanet Journal of Rare Diseases1750-11722025-07-012011710.1186/s13023-025-03892-0A father’s crusade in rare disease drug development: a case study of Elpida therapeutics and MelpidaDeanna Portero0Qingyang Xu1Aaliya Hussain2Andrew W. Lo3Johns Hopkins Bloomberg School of Public HealthMIT Laboratory for Financial EngineeringMIT Laboratory for Financial EngineeringMIT Laboratory for Financial EngineeringAbstract 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.https://doi.org/10.1186/s13023-025-03892-0 |
| spellingShingle | Deanna Portero Qingyang Xu Aaliya Hussain Andrew W. Lo A father’s crusade in rare disease drug development: a case study of Elpida therapeutics and Melpida Orphanet Journal of Rare Diseases |
| title | A father’s crusade in rare disease drug development: a case study of Elpida therapeutics and Melpida |
| title_full | A father’s crusade in rare disease drug development: a case study of Elpida therapeutics and Melpida |
| title_fullStr | A father’s crusade in rare disease drug development: a case study of Elpida therapeutics and Melpida |
| title_full_unstemmed | A father’s crusade in rare disease drug development: a case study of Elpida therapeutics and Melpida |
| title_short | A father’s crusade in rare disease drug development: a case study of Elpida therapeutics and Melpida |
| title_sort | father s crusade in rare disease drug development a case study of elpida therapeutics and melpida |
| url | https://doi.org/10.1186/s13023-025-03892-0 |
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