InnoSer Partners with Kabuki Syndrome Foundation to Advance Therapeutic Development for Kabuki Syndrome
InnoSer to serve as the central hub for Kabuki Syndrome Foundation-funded preclinical research in the Kmt2d+/βGeo mouse model. This collaboration aims to empower leading biotech and pharma innovators to perform rigorous studies informing clinical trial readiness of candidate drugs for Kabuki syndrome.
Diepenbeek, Belgium, 15th of May 2025 – InnoSer, a leading preclinical contract research organization (CRO), is proud to announce a collaboration with the Kabuki Syndrome Foundation, a global nonprofit dedicated to driving Kabuki syndrome therapeutic development. This partnership facilitates preclinical research of novel candidate therapies for Kabuki syndrome, a rare genetic disease for which there is currently no treatment available. This partnership builds on InnoSer’s extensive expertise in rare genetic disease models and its proven capabilities in conducting preclinical research studies, helping biotech and pharmaceutical innovators accelerate the discovery and development of candidate therapies, paving the way for future treatments for this rare condition.
Helping Address the Unmet Medical Need for Kabuki Syndrome
Kabuki syndrome is a rare, neurodevelopmental disorder, characterized by a range of developmental and cognitive impairments, including memory deficits and difficulties with visuospatial learning, as well as immune dysfunction and other chronic symptoms. These challenges severely impact individuals and their families, creating a high unmet medical need. With no approved therapies available, this collaboration will accelerate rigorous preclinical testing of candidate therapies for Kabuki syndrome, which is a critical prerequisite before advancing to clinical trials.
The first step of this collaboration is to confirm the therapeutic potential of vafidemstat, an LSD1 inhibitor, for treating Kabuki syndrome in the well-characterized Kmt2d+/βGeo mouse model first characterized by the laboratory of Dr. Hans Bjornsson (1), a leading expert in Kabuki syndrome research. Vafidemstat, developed by Oryzon Genomics, a leading clinical-stage biopharmaceutical company focused on the development of epigenetic-based drugs for CNS diseases and oncology, has already been tested in Phase II clinical trials for multiple neurological indications and is currently moving into Phase III, confirming its tolerability in humans.
InnoSer to Serve as Central Hub for Preclinical Kabuki Syndrome Studies
Going forward, InnoSer will serve as the central hub for preclinical research studies in the Kmt2d+/βGeo mouse model funded through the Kabuki Syndrome Foundation. This centralization, along with the strategic coordination and support from Kabuki Syndrome Foundation, will provide researchers with a rapid and cost-effective solution for outsourcing any future preclinical research studies. Leveraging InnoSer’s expertise in Kmt2d+/βGeo model colony maintenance, together with proven expertise in conducting molecular and behavioral assessments of candidate drugs, this collaboration aims to empower leading biotech and pharma innovators to accelerate the development of Kabuki syndrome therapeutics by providing rapid study execution timelines, a standardized platform to perform rigorous preclinical research, and expert scientific support.
InnoSer further emphasized the significance of this collaboration in helping drive therapeutic innovation for Kabuki syndrome. “This exciting collaboration between InnoSer and the Kabuki Syndrome Foundation underscores the importance of involving patient advocacy organizations and working together to bring new therapies to those affected,” said Maarten Loos, PhD, director of InnoSer Laboratories Netherlands. “Our team’s profound experience in working with rare neurological genetic disease models combined with our strong track record in conducting drug profiling, tolerability, efficacy and behavioral testing studies makes InnoSer an ideal partner for Kabuki Syndrome Foundation in this important initiative.”
KSF has chosen InnoSer as a trusted partner due to their extensive experience in conducting high-quality preclinical studies related to rare genetic conditions, including their successful track record with other neurological diseases. “InnoSer’s expertise in rare neurological genetic disease models, including their proficiency in behavioral and molecular assessments, made them the natural choice for this collaboration,” said Janet Lee, Executive Director of the Kabuki Syndrome Foundation. “This partnership marks a critical step in accelerating therapeutic development for Kabuki syndrome. Insights from the study will help guide future research and move us closer to potential treatments.”
The collaborative efforts between InnoSer and KSF position them at the forefront of Kabuki syndrome research, aiming to accelerate therapeutic development for this rare disease. By combining InnoSer’s and KSF’s expertise, resources, and commitment, this collaboration strives to make a meaningful impact in the lives of individuals affected by this challenging condition.
About InnoSer:
Founded in 2012, InnoSer is an innovative and dynamic CRO supporting the pre-clinical development of drugs from its facilities in Belgium and the Netherlands. InnoSer supports its customers in the critical steps of their drug development journey with well-characterized in vitro and in vivo disease models in addition to general drug development services, allowing to uncover valuable insights into the mode of action, pharmacokinetics, safety, and efficacy of investigational compounds.
About Kabuki Syndrome Foundation:
The Kabuki Syndrome Foundation (KSF) collaborates with a global network of leading clinicians, researchers, and patient advocates to drive high-impact, patient-centered research. Guided by its Medical and Scientific Advisory Board, KSF identifies critical gaps in knowledge, funds targeted research and brings together experts to move science to treatments for the Kabuki syndrome community.
Contact:
Amanda Gamboa, KSF Director of Communications
[email protected]