The biopharma business model is misaligned with rare diseases, and children are paying the price.
Recent policy and market changes have sparked a dramatic shift in investment toward therapies with broad commercial demand — leaving hundreds of rare disease programs shelved, often despite promising clinical data. Many of these conditions are pediatric, progressive, and fatal, making delays especially costly.
Craig Martin
Founder, Orphan Therapeutics Accelerator (OTXL)
“There have been huge leaps forward in genetic medicines to treat rare diseases, but the business model to support their advancement hasn’t evolved at the same pace,” shared Craig Martin, who founded the Orphan Therapeutics Accelerator (OTXL) to reimagine how rare disease treatments are financed and advanced.
As a nonprofit, OTXL can incentivize companies to donate programs and negotiate deferred or reduced development costs with industry partners.
In partnership with the American Society for Gene and Cell Therapy, OTXL is also building an AI-powered marketplace to surface shelved assets and match them with aligned funders, opening the door to family offices, impact investors, and others historically excluded from this space.
Soon, OTXL plans to enable fractional investment — another pragmatic, scalable alternative to the current funding model that helps accelerate paths to patients.
To learn more, visit orphantxl.com
