IDefine and Unravel Biosciences Announce Drug Repurposing Research Collaboration

FOR IMMEDIATE RELEASE

IDefine and Unravel Biosciences Announce Drug Repurposing Research Collaboration to Accelerate Treatments for Kleefstra Syndrome

ATLANTA, GA  — IDefine – The Kleefstra Syndrome Foundation, a leading patient advocacy organization, and Unravel Biosciences, Inc. (“Unravel”), a therapeutics company leveraging an AI-driven drug discovery platform, today announced a strategic collaboration to accelerate the discovery of potential treatments for Kleefstra syndrome.

Through this partnership, Unravel will analyze samples from 20 Kleefstra syndrome patients using its proprietary rareSHIFT™ program. Patients will provide RNA samples through a non-invasive nasal swab collection method, enabling participation from anywhere in the world without the need for clinical visits or complex logistics. Unravel will use this data to generate Living Molecular Twins™ – digital models that enable AI-powered drug trial simulations to screen more than 40,000 molecules per patient, helping identify potential therapeutic candidates, uncover underlying disease biology, and better predict individual patient responses.

“This collaboration represents an important step forward in identifying potential treatments for people living with Kleefstra syndrome,” said Eric Scheeff, PhD, chief scientific officer of IDefine. “By combining patient-derived data with advanced computational modeling – and enabling participation from anywhere without the burden of travel – we can expand access to research while accelerating progress toward the clinic for a community that is in urgent need of effective treatment options.”

Kleefstra syndrome is a rare genetic neurodevelopmental disorder caused by a mutation or deletion of the EHMT1 gene, which plays a critical role in brain development and function. The condition presents with a wide range of symptoms – including intellectual disability, speech impairment, low muscle tone, seizures, and behavioral challenges – and affects each individual differently, with severity ranging from mild to profound. There are currently no approved treatments for Kleefstra syndrome.

“Unravel’s mission is to integrate individual patient data into the drug discovery process from the very start,” said Richard Novak, PhD, CEO of Unravel Biosciences. “By applying our BioNAV™ platform to Kleefstra syndrome, we can rapidly screen for repurposing candidates and uncover novel shared therapeutic mechanisms for Kleefstra syndrome patients that offer the greatest likelihood of clinical success.”

About IDefine

IDefine is the leading nonprofit patient advocacy organization dedicated to supporting individuals and families impacted by Kleefstra syndrome and accelerating the development of treatments and a cure. Founded in 2020 by parents of children diagnosed with the condition, IDefine brings urgency, determination, and a deeply personal commitment to advancing research and improving outcomes. The organization funds and drives critical research, builds collaborative partnerships with clinicians, researchers, and industry, and fosters a strong, connected community so no family faces Kleefstra syndrome alone. Through its efforts, IDefine is helping to advance scientific understanding and move the field closer to meaningful therapies and, ultimately, a cure. For more information, visit https://www.idefine.org/.

About Unravel Biosciences

Unravel Biosciences is a clinical-stage therapeutics company that integrates AI systems biology computation with rapid clinical validation of discovered targets, leading to four clinical trials starting in 2026. Unravel leverages its proprietary BioNAV™ platform and primary transcriptomics data to create Living Molecular Twins of real patients that can be used to predict therapeutic response. This enables target and drug discovery, preclinical screening, patient stratification, and clinical validation to find treatments for complex diseases using a systematic, data-driven approach called Predictable Medicine™.

Unravel’s platform discovered RVL002, a first-in-class small molecule that targets mitochondrial metabolism and has multiple clinical applications, including neurodegenerative and metabolic disorders, and identified RVL069, a molecule that targets a novel mechanism to treat dystonias. The rareSHIFT™ program provides platform and proprietary datamine access to foundation and biotech partners to accelerate and clinically derisk therapeutics. www.unravel.bio and www.rareshift.org