Finding the Next Rare Disease Drug
Post Date: August 21, 2026 | Publish Date:
Human Genetics is Reshaping Target Evaluation
How do researchers determine which discoveries are most likely to become successful therapies?
That question was at the center of a recent Cincinnati Children’s Innovation Ventures seminar featuring two asset acquisition thought leaders from GondolaBio, a clinical stage biopharmaceutical company, Aileen Li, PhD, Head of Academic Partnering, and Jamie Wu, PhD, Associate, Asset Acquisition.
While the discussion covered rare disease therapeutics and translation broadly, one of the most compelling takeaways was how advances in human genetics are reshaping the way drug developers evaluate therapeutic opportunities.
Genetics as a filter for risk
Li pointed to a statistic that anchors much of GondolaBio’s diligence: targets backed by human genetics evidence successfully reach the market at higher rates compared to targets without it. The reasoning behind that finding, Li explained, comes down to how GondolaBio wants to think about biology itself.
“We ask, can we reduce a complex biology problem into a practical engineering problem?” Li said. “If we can connect the dots in the biology and if we understand the mechanism of the biology, then we are more likely to figure out how to execute and ultimately be successful.” GondlaBio finds that having a solid genetic basis increases chances for success.
That philosophy also shapes GondolaBio’s structure. A sister company of BridgeBio Pharma, GondolaBio launched at the end of 2024 with $300 million in independent funding. The company runs on a portfolio model inspired by MIT’s Andrew Lo, PhD, and BridgeBio co-founder Neil Kumar, PhD. Rather than betting on one or two lead assets, the model suggests building many genetically-anchored programs at once with uncorrelated risk profile, so that we have more shots on goal to bring drugs to patients.
A diligence framework built around genetic evidence
For academic researchers wondering when a discovery is “ready” for industry engagement, Li laid out the framework her team applies to any target or compound:
- Do we know the target of the drug? Do we understand the on-target and off-target profiles of the drug?
- If the drug hits the target and is believed to be safe, will it modify the disease?
- Can we measure the outcome in a clinical study? Are there biomarkers to help us understand what the drug may be doing in patients?
- Can we ultimately seek FDA approval? Is the regulatory path clear? Can we build a solid case to support commercial viability?
Drug development, from ideation to approval, takes a long time and truly takes a village. We rely on expertise from many teams, including basic researchers, innovation groups, clinical centers, patient-foundations, families, among others to help us bring a new drug to the hands of patients.
Science first, deal structure second
Asked what drives the model, Li stated: “We believe that the best drugs are made from the best science.”
GondolaBio’s asset acquisition team is built to pursue ideas to target disease at or near the source. They offer various collaboration models such as sponsored research agreements, co-founder equity, and joint IP development.
If you have any questions about commercialization and collaboration at Cincinnati Children’s, reach out to Innovation Ventures.
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The Research Horizons blog features news and insights about the latest discoveries and innovations developed by the scientists of Cincinnati Children's. This blog does not provide medical advice, diagnosis, or treatment. Email researchnews@cchmc.org with questions or ideas.
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