CSO Corner, Volume 15: Drug Repurposing with Unravel Biosciences
CSO Corner, Volume 15: Drug Repurposing with Unravel Biosciences
Hello fellow members of our KS family!
Today, I am thrilled to share the latest project in our “Treatment Focused” strategy. We are officially launching a strategic research collaboration with Unravel Biosciences to investigate drug repurposing for KS.
What is Drug Repurposing?
In the world of drug development, creating a brand-new medicine from scratch is a bit like building a new car from the ground up—it takes years of design, safety testing, and massive amounts of funding before it ever hits the road.
Drug repurposing is different. It’s like finding a tool in your garage that was originally designed to tighten a bolt, but realizing it’s also the perfect tool to fix a leaky faucet. We are looking at drugs that have already been “road-tested” and FDA-approved for other conditions to see if they can fix the biological “leaks” we see in Kleefstra Syndrome.
The benefit? We already know a lot about the safety of these drugs, which can potentially shave years off the timeline to get a treatment to our community.
How Unravel Works: The BioNAV™ GPS
So, how do we know which of the thousands of existing drugs might work for KS? This is where Unravel comes in. They use a proprietary platform called BioNAV™.
Think of BioNAV™ as a high-powered, digital GPS for biology. Instead of guessing which drugs might work through trial and error in a lab, Unravel uses artificial intelligence and complex “big data” to map out the unique biological signature of Kleefstra Syndrome. They then cross-reference that map against a massive library of existing drugs to see which ones “fit” the KS signature. It’s about finding the right key for our specific lock.
How it Works: From Nasal Swabs to Simulations
What makes this partnership particularly exciting is how easy it is for our families to participate. Through a non-invasive nasal swab (no complex clinic visits required!), Unravel will analyze samples from 20 KS patients.
- The Signature: We collect the RNA (the instructions the cell is sending out) from those swabs.
- The Simulation: Unravel’s AI platform then screens more than 40,000 molecules against these “digital twins.”
- The Shortlist: The goal is to identify drug candidates—either existing medications or new molecules—that might “flip the switch” back toward a more healthy state.
Sifting Through the Leads: What Happens Next?
It is important to manage our expectations about what “finding a match” actually means. When the AI identifies a molecule that looks promising in a computer simulation, we call that a “lead.” Think of a lead like a candidate who looks perfect on paper for a job. They have the right resume and the right skills, but you wouldn’t hire them without an interview and a background check.
In science, those “interviews” are our follow-up investigations. Once Unravel identifies these potential leads, the work won’t stop; it will actually intensify. We will need to:
- Validate the findings: Test the leads in model systems, such as neurons derived from KS patient cells, to see if it actually provides a benefit.
- Check for safety and efficacy: Ensure the compound does what we think it does without causing unwanted side effects.
- Refine the dose: Figure out exactly how much of a compound is needed to be effective.
But identifying these leads is a massive shortcut—it’s like being given a shortlist of the 5 best candidates instead of having to interview 40,000 people—but we still will have to do the rigorous work to prove they can become genuine treatments.
The Power of Partnership
For years, IDefine has worked to make our community “Research Ready.” Because we have the infrastructure and a motivated community, we can now plug directly into cutting-edge platforms like Unravel’s.
This collaboration is another in our multi-pronged attack on KS, and another result of our “turn up the volume” campaign. There is more to come, stay tuned!
Until next time,
Eric Scheeff, PhD
IDefine Chief Scientific Officer
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