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.

The eighth version of the Prescription Drug User Fee Act (PDUFA) is up for renewal next year, but the bulk of the negotiations between the drug development industry and the U.S. FDA is largely complete. At the BIO International Convention, representatives from the industry and trade organization provided an overview of what was agreed upon during the 127 negotiating sessions that were needed to arrive at the commitment letter, which will be sent to Congress later in 2026.

SK Biopharmaceuticals Co. Ltd. is doubling down on AI-powered drug discovery through a new collaboration with Insilico Medicine Inc. valued at more than $2.5 billion. The milestone-heavy deal, announced at the BIO International Convention in San Diego June 22, will pair Insilico’s Pharma.AI platform with SK Biopharm’s central nervous system (CNS) drug development and commercialization capabilities, underscoring Korean and global biopharma efforts to embed AI across the entire R&D lifecycle.