Hope for rare diseases: new funding model aids treatment
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Lee and Lori Greenwood’s daughter, Noa, received a diagnosis of Canavan disease, a rare genetic condition, and quickly entered a clinical trial for a new therapy.
Canavan prevents children from developing beyond a six-month-old’s abilities, but Noa began reaching milestones her parents had been told she would never achieve after receiving treatment. Previously, pharmaceutical companies often avoided funding treatments for rare diseases like Canavan because the number of potential patients was too small to justify the investment.
BridgeBio addressed this problem by creating a different approach to funding. Instead of focusing on single rare disease treatments, the company invested in a portfolio of 150 different therapies, spreading the financial risk. This strategy allowed them to pursue cures for conditions that traditional pharmaceutical companies deemed unprofitable. Noa’s progress demonstrates the potential of this new model to help individuals with rare diseases.
Approximately 450 million people globally live with rare diseases, and BridgeBio’s approach offers a path toward developing treatments for many of them. The company hopes its model will encourage further investment in therapies for conditions affecting small patient populations.
