A new, freely available checklist for early-stage biotherapeutics programs is aiming to bring more discipline to the high-stakes decisions that determine which drug candidates advance toward the clinic. Catherine Cifuentes-Rojas, Director and Lead for Genetic Medicines and Biologics at Ginkgo Bioworks, released the tool after spending more than 15 years in academic and industry labs, where she observed dozens of go/no-go calls on RNA therapeutics, cell therapies, and biologics candidates.
The checklist is not a proprietary framework tied to any single company's pipeline. Instead, it consists of five plain questions that teams must answer in writing before a program moves to the next stage of investment. Among them: whether a primary assay result replicates in a second, independent system; whether the team has tried to kill its favored hypothesis rather than merely confirm it; and what the in vivo data say, as opposed to in vitro results. It also asks who outside the originating team has reviewed the data, and what evidence would change the team's mind.
The implications for the biotherapeutics industry are significant. Drug development is notoriously capital-intensive, and failures late in development are far more costly than those caught early. By forcing teams to confront disconfirming evidence and external review before committing to expensive in vivo studies or IND-enabling work, the checklist could help organizations avoid sinking resources into programs that lack a solid scientific foundation. Cifuentes-Rojas argues that a no-go decision made early and backed by real data is as much a success as a program that clears every stage.
The checklist also addresses a cultural problem in research settings: junior scientists may hesitate to raise disconfirming data in front of senior colleagues, not because the data are weak but because they lack a structured way to present them. "If the only tool in the room is confidence, the most confident person wins the argument," Cifuentes-Rojas said. "A checklist takes the argument out of it. Either the box is checked with evidence, or it isn't."
She designed the checklist to be completed by a group in under an hour and used at defined decision points: after initial target validation, before committing to in vivo studies, and before a program moves toward IND-enabling pre-clinical work. Answers are recorded and revisited at the next decision point, with dissenting views documented rather than smoothed over. No particular software or platform is required; a shared document suffices.
For an industry where scientific momentum and seniority can overshadow rigorous self-criticism, the checklist offers a simple, low-cost mechanism to improve decision quality. Its release reflects a broader push for more transparent, evidence-driven governance in early-stage biotherapeutic discovery, where the cost of premature advancement can be measured in both dollars and patient risk.
