2024 has been a year of great achievements for Unravel. We are proud to present our Year in Review.
It’s been a busy year for Unravel.
Here’s a quick count of some of our work.
Partners in Progress
We are honored to have initiated collaborations with numerous dedicated organizations to address unmet needs in the rare disease space. Explore some of our impactful partnerships from 2024.
Rett Syndrome and RVL-001: A Clinical Trial and more!
This year, we’ve made great progress working toward a treatment for Rett Syndrome. BioNAV™ identified RVL-001 as a potential treatment for Rett Syndrome, alongside similar findings by other organizations.
In early 2024, Vanderbilt University Medical Center (VUMC) announced its clinical trial to test multiple repurposed drugs for the treatment of Rett syndrome, one of which being vorinostat. A few months later, we happily announced that we are collaborating with VUMC to provide the liquid formulation of vorinostat, RVL-001, for its clinical trial.
Unravel was pleased to collaborate with Quality Chemical Laboratories, Inc. to develop a new manufacturing process for RVl-001 that has lower toxicity and lower manufacturing costs to be tested in clinical trial.
In addition, we are thrilled to conduct our own clinical trial with PECET at the Universidad de Antioquia in Medellin, Colombia. This trial will continue to test the safety and effectiveness of RVL-001 for the treatment of Rett syndrome. As part of this study, we have also translated the widely used Rett syndrome Behavioral Questionnaire (RSBQ) to Spanish, to increase its accessibility to Spanish-speaking populations. In December, our CEO, Richard Novak, and CMO, Neal Muni, took a trip to Medellin in December to fimalize planning for the clinical trial.
Finally, Unravel received an Orphan Drug Designation from the FDA for RVL-001 (vorinostat) to treat Rett syndrome.
Clinical Trial for RVL-001 with the Pitt Hopkins Research Foundation
BioNAV™ also identified RVL-001 as a potential therapeutic for Pitt Hopkins syndrome. The Pitt Hopkins Research Foundation is funding a clinical trial in Colombia to test the efficacy of RVL-001 in Pitt Hopkins patients. Running this collaboration in parallel with the Rett syndrome clinical trial will help to increase the scope of RVL-001 to meet a larger need within the rare disease space.
“A key part of our mission is to rapidly advance promising drugs into clinical testing in multiple orphan disorders, and our partnership with the Foundation will allow us to evaluate the benefit of RVL-001 in this high unmet need patient group.”
Expanded rareSHIFT discovery programs through new business model
Unravel developed rareSHIFT in late 2023 to enable foundations and families access to our innovative drug and target discovery platform aimed at rapid clinical validation. We work with patients to generate RNA data using self-collected nasal swabs, predict potential therapeutic mechanisms, and work with our SqushyWare tadppole models, Jackson Labs mouse models, and clinicals to translate repurposed drugs to the clinic. While the drugs are tools for clinically validating Unravel’s drug targets, they can make a huge impact to patients in the near term.
In 2024, we formalized a not-for-profit business model, enabling scaling the program by lowering costs. Unravel maintains intellectual property and commercial possibilities while sharing all results for noncommercial use with our partners. This partnered approach has already yielded numerous insights into new biological mechanisms while allowing even single families to pursue cutting edge research with urgency. As programs mature, Unravel can transition them to its for-profit biopharma arm to spin out dedicated companies to commercialize new therapeutics for the dicovered targets - backed by clinical validation data.
Accelerating the Path to Clinical Trials with the Jackson Laboratory
Unravel initiated a collaboration with the Jackson Laboratory (JAX), a world leader in preclinical models, to accelerate the development of drugs for high unmet need in the rare disease space. Unravel will use BioNAV™ to predict potential therapies, which will be validated in JAX’s pre-clinical models in a seamless preclinical pipeline, shaving potentially years off of the development process.
Saving Sea Lions with Zymo and Channel Islands Marine Wildlife Institute
Unravel is collaborating with Zymo Research Corporation and Channel Islands Marine & Wildlife Institute (CIMWI) to identify an injectable therapy for sea lions dying of domoic acid (DA) toxicity that could be given on the beach as life-saving treatment. Algae blooms have been increasing as a result of climate change, causing lethal domoic acid toxicity in thousands of animals annually. To learn more about the impact of algae blooms on sea lions, check out this article by the LA Times. CIMWI has taken RNA samples from their sea lion patients that were sequenced by Zymo, and Unravel is now using BioNAV™ to predict treatments using the RNAseq data.
Building on Success: Expanding Our Partnerships
We are honored to have collaborated with many rare disease foundations this year through our rareSHIFT program. With these foundations, we identified drug candidates to treat rare diseases, stratified patients from the same disease into therapeutic response groups, and more. In addition, Unravel conducted 33 personalized studies this year to help patients and families find personalized drug recommendations to treat their rare, ultra-rare, or undiagnosed diseases.
Voices of Impact: Insights from Leaders & Lives Changed
Dr. Manuella Mallamaci, the President of Fondazione CHOPS, exemplifies how leaders' vision and hard work in the rare disease space drive the progress toward therapies.
Rapid Impact through rareSHIFT
Patients are our inspiration. Watch Max’s patient story to learn more about the impact of Unravel Biosciences.
We celebrated Unravel’s new HQ in Medford, MA with state, city, Tufts and Harvard University, and MassBio represenratives
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It all begins with an idea. Maybe you want to launch a business. Maybe you want to turn a hobby into something more.
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It all begins with an idea. Maybe you want to launch a business. Maybe you want to turn a hobby into something more.
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It all begins with an idea. Maybe you want to launch a business. Maybe you want to turn a hobby into something more.
We are beyond grateful for the organizations, investors, advisors, physicians, patients, caregivers, and other believers in our mission and novel approach who made our achievements possible this year. We look forward to continuing to grow our impact in 2025.
