Unravel, TMA Partner to Find Patients for 1st Trial Testing RVL001

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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Unravel Biosciences, which is working toward a first clinical trial to test its investigational therapy RVL001 in people with Rett syndrome, announced that it has partnered with the rare disease health platform TMA Precision Health to identify eligible patients for recruitment into the study.

Under the partnership, Unravel will leverage TMA’s database of rare disease patients from global sources, making it easier to locate the right patients, which can be especially difficult in rare diseases like Rett syndrome.

“TMA’s unique capabilities identifying orphan disease patient populations in global regions historically underrepresented in trials are directly aligned with our mission to rapidly bring effective treatments to patients in need,” Richard Novak, PhD, cofounder and CEO of Unravel, said in a press release.

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Rett syndrome is a neurodevelopmental genetic disorder that affects about one in every 10,000 female births. It has no approved treatments but several experimental therapies are being tested.

“For us at TMA, this partnership is exactly the kind of work we set out to do: enabling companies like Unravel to more fully leverage their drug discovery platforms and power real change for patients without options,” said Joshua Resnikoff, cofounder and CEO of TMA. “We’re incredibly proud to be a part of Unravel’s efforts by using our clinicogenomics database to help drive their clinical work forward.”

Unravel, which spun out from Harvard University’s Wyss Institute for Biologically Inspired Engineering, is a therapeutics company that seeks to identify new therapeutic targets via proprietary computational analyses.

The company’s platform first involves analyses of transcriptomics — a global view of how genes are “turned on or off” — to identify signatures of genetic activity. Then, interactions with more than 40,000 compounds are simulated via computer analyses to identify potential therapies that may normalize the disease signature. Promising candidates are screened using Unravel’s first-in-class frog (Xenopus) models to confirm that they interact with the intended therapeutic target.

The goal of these analyses is to identify medications that are already approved for use and that will interact with a target that holds promise in treating a disease. Then, the company can quickly conduct trials to “de-risk” the target — basically, using an existing medication with an established safety profile to assess whether an experimental target can be safely tested.

That’s the approach the company is employing for the oral liquid therapy RVL001. Unravel did not specify which compound(s) are in the experimental formulation, nor its intended targets.

“Unravel is thrilled to partner with TMA to power this first step to the clinic for RVL001,” Novak said. “By integrating a clinical data-focused trial design with our computational drug and target discovery platform, this collaboration will accelerate and derisk drug development for Rett syndrome.”