Together, CureSHANK, a nonprofit research foundation, and InnoSer, preclinical research CRO, aim to remove barriers to drug development for Phelan-McDermid syndrome treatment by providing the global research community with accessible and industry-standard preclinical tools and services.
Diepenbeek, Belgium, June 26th, 2025 – InnoSer, a leading preclinical contract research organization (CRO), and CureSHANK, a leading research foundation dedicated to advancing treatments for Phelan McDermid Syndrome (PMS), are partnering to launch a license-free, industry-quality preclinical testing platform for PMS, a severe neurodevelopmental disorder with no cure and no approved treatment options. Through this collaboration, researchers worldwide will gain streamlined access to a novel, license-free mouse model and comprehensive preclinical service platform, paving the way for novel treatments for this rare condition on a faster timeline.
About Phelan McDermid Syndrome (PMS)
Phelan-McDermid Syndrome (PMS) is a rare genetic neurodevelopmental disorder caused by deletions or mutations affecting the SHANK3 gene. PMS, being the most common genetic form of autism, is associated with a range of neurological and developmental challenges, including intellectual disability, delayed speech and motor skills, and social communication difficulties, profoundly affecting quality of life for both patients and their families. It is estimated to affect 1 in 8,000 to 15,000 individuals, with the diagnosis rate climbing steadily year-over-year.
Despite the urgent need for effective treatments, progress in developing therapies for PMS and SHANK3-related neurodevelopmental disorders has been hindered by a lack of license-free, translationally relevant preclinical models and standardized, high-quality preclinical testing infrastructure. Central to overcoming these challenges is the development and validation of translational preclinical models that enable rigorous assessment of therapeutic candidates, reducing drug development timelines, and improving preclinical-to-clinical translational potential.
Removing Commercial Barriers to SHANK3 Research
CureSHANK has developed a novel PMS mouse model designed specifically to eliminate licensing fees and intellectual property barriers commonly associated with existing models, enabling a broader range of researchers to advance therapeutic programs targeting SHANK3-related neurodevelopmental disorders. This model represents a license-free version of the gold-standard Exon 4–22 Shank3 deletion mouse, closely mimicking the patients’ genetic makeup.
To further address the preclinical research gap, CureSHANK has partnered with InnoSer, a CRO renowned for its expertise in rare neurodevelopmental disorder research, to establish an industry-standard preclinical testing service platform. Simultaneously, the mice will also be available directly, without licensing fees or intellectual property constraints, to both non-profit and commercial researchers via OzGene, which has generated the mice for CureSHANK and holds an exclusive license for distribution.
InnoSer to Provide Industry-Quality SHANK3 Preclinical Studies
As CureSHANK’s CRO partner, InnoSer is currently leading the first phase of the collaboration by validating preclinical readouts predictive of clinical responses, helping bridge the gap between preclinical-to-clinical translation. Starting in 2026, InnoSer will provide industry-quality preclinical testing services for PMS, allowing researchers to rapidly test therapeutic candidates without investing in costly infrastructure, standardizing data collection and reducing timelines in early-stage drug development.
This partnership builds on InnoSer’s customizable preclinical studies, including behavioral analysis, biomarker validation, EEG, and pharmacokinetic testing, leveraging their extensive experience in gene therapy testing and rare neurodevelopmental disorders such as STXBP1 encephalopathy. Highlighting the platform’s innovation, InnoSer utilizes an optimized workflow for cost-efficient mouse phenotyping through PhenoTyper™ cages combined with the AHCODA™ data analysis platform.
“Preclinical research is a critical and complex step in developing effective therapies for rare genetic disorders like Phelan-McDermid Syndrome. By combining a license-free mouse model with standardized preclinical services, we’re providing a practical, scalable solution for researchers who want to move quickly and confidently, helping accelerate the journey from discovery to clinical trials with the goal of ultimately bringing new treatments to patients faster,” said Maarten Loos, PhD, director of InnoSer Laboratories Netherlands.
“This partnership marks a significant step forward in our mission to accelerate the discovery of treatments for Phelan-McDermid Syndrome,” said Geraldine Bliss, President of CureSHANK. “By making this model freely available and working with InnoSer to provide rigorous preclinical testing, we are removing critical barriers that have slowed progress in PMS drug development, significantly opening up the playing field.”
The collaborative efforts between InnoSer and CureSHANK position them at the forefront of PMS research, aiming to accelerate therapeutic development for this rare disease. By combining InnoSer’s and CureSHANK’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 CureSHANK:
CureSHANK is a nonprofit research foundation accelerating therapies for PMS and related SHANK3-linked disorders. Through open-science initiatives, strategic partnerships, and community advocacy, CureSHANK is committed to bridging the gap between scientific discoveries and real-world treatments for individuals affected by PMS.
Learn more at https://www.cureshank.org/