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.
On the same day that FDA Commissioner Martin Makary spoke in a fireside chat during the 2025 Biotechnology Innovation Organization’s International Convention, the agency unveiled a pilot commissioner’s national priority voucher program that will enable companies to receive a shortened FDA review time of one to two months.
On the same day that FDA Commissioner Martin Makary spoke in a fireside chat during the 2025 Biotechnology Innovation Organization’s International Convention, the agency unveiled a pilot commissioner’s national priority voucher program that will enable companies to receive a shortened FDA review time of one to two months.
For years, the biopharma industry has spent increasing amounts of money on R&D without improving success rates, leaving many executives searching for new, more predictable drug development paths.
The COVID-19 pandemic sent the world into a tailspin, raising ongoing concerns about biosecurity, a subject that encompassed the better part of the morning June 16, the first day of the Biotechnology Innovation Organization’s annual conference in Boston.
As a growing roster of nations moves to protect individual genomic and other health data in the name of privacy under the General Data Protection Regulation in the EU and similar laws elsewhere, chief aggregators of such data, drug developers, are struggling.
As a growing roster of nations moves to protect individual genomic and other health data in the name of privacy under the General Data Protection Regulation in the EU and similar laws elsewhere, chief aggregators of such data, drug developers, are struggling.
With just 10% of therapeutic programs successfully moving from first toxicity dose to market, drug developers are on a constant hunt for new tricks to put the odds in their favor. Strategies abound, from biomarker-guided patient selection to deeper regulatory engagement. But big pharma executives and an academic expert weighing the challenge at the BIO International Convention said June 14 they see another tool slowly gaining traction with the potential to decrease timelines and boost chances for approval: in-silico modeling.
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