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From One to Many

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Manage episode 363656087 series 60790
内容由RARECast提供。所有播客内容(包括剧集、图形和播客描述)均由 RARECast 或其播客平台合作伙伴直接上传和提供。如果您认为有人在未经您许可的情况下使用您的受版权保护的作品,您可以按照此处概述的流程进行操作https://zh.player.fm/legal

When Terry Pirovolakis’ son Michael was diagnosed with the ultra-rare neurodegenerative disease spastic paraplegia type 50, he set out to raise money and engage researchers in developing a treatment. Now, after successfully dosing Michael with an experimental gene therapy as the first patient in a clinical trial, he has launched Elpida Therapeutics to develop multiple gene therapy programs for children with ultra-rare diseases. We spoke to Pirovolakis about the need Elpida is seeking to address, its unusual business model, and why he hopes to hand off its therapies at no cost to a partner once they win approval.

  continue reading

489集单集

Artwork

From One to Many

RARECast

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published

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Manage episode 363656087 series 60790
内容由RARECast提供。所有播客内容(包括剧集、图形和播客描述)均由 RARECast 或其播客平台合作伙伴直接上传和提供。如果您认为有人在未经您许可的情况下使用您的受版权保护的作品,您可以按照此处概述的流程进行操作https://zh.player.fm/legal

When Terry Pirovolakis’ son Michael was diagnosed with the ultra-rare neurodegenerative disease spastic paraplegia type 50, he set out to raise money and engage researchers in developing a treatment. Now, after successfully dosing Michael with an experimental gene therapy as the first patient in a clinical trial, he has launched Elpida Therapeutics to develop multiple gene therapy programs for children with ultra-rare diseases. We spoke to Pirovolakis about the need Elpida is seeking to address, its unusual business model, and why he hopes to hand off its therapies at no cost to a partner once they win approval.

  continue reading

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