BIO 2026: Alternatives to funding orphan drugs
· brian orelli
AI Briefing
- • Nonprofit and for-profit companies discuss funding orphan drug development, focusing on ultra-rare diseases.
- • Skipping discovery phase with repurposed drugs or rescued assets can keep costs down.
- • The Orphan Therapeutics Accelerator rescues shelved therapeutic assets for ultrarare diseases.
Source Summary
At the BIO International Convention, nonprofit and for-profit companies discussed ways to fund orphan drug development, especially for ultra-rare diseases where development costs can be challenging. One of the simplest ways to keep costs down is to skip the discovery phase and use repurposed drugs or rescue shelved therapeutic assets as the Orphan Therapeutics Accelerator has done. The Cambridge, Mass.-based accelerator was founded in 2024 to rescue shelled therapeutic assets for ultrarare diseases.
Advertisement