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July 31, 2025 42 mins

Rare disease advocates have long made the case that studying rare diseases can provide insights into more common ones. Actio Biosciences has turned that into a business model. The company is leveraging genetics and precision medicine to develop drugs for rare diseases in the hopes of expanding the indications for them to include more common disease that share underlying biology. We spoke to David Goldstein, founder and CEO of Actio Biosciences, about the company’s Rare Disease Target Atlas, how it identifies the indications it will pursue, and why pricing represents a challenge for such a business model.

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