Global collaboration launches streamlined-access to Shank3 cKO research model

Next-generation research tool designed to accelerate therapeutic development for Phelan-McDermid syndrome (PMS) and SHANK3-related neurodevelopmental disorders.

2026-02-09

(Press-News.org) ** FOR IMMEDIATE RELEASE **
Global Collaboration Launches Streamlined-Access to Shank3 cKO Research Model Next-generation research tool designed to accelerate therapeutic development for Phelan-McDermid syndrome (PMS) and SHANK3-related neurodevelopmental disorders. Next-generation research tool designed to accelerate therapeutic development for Phelan-McDermid syndrome (PMS) and SHANK3-related neurodevelopmental disorders. LEIDEN, THE NETHERLANDS - LOS ANGELES - PERTH — February 9, 2026 — InnoSer, a contract research organization (CRO) with preclinical expertise; CureSHANK, a driver of initiatives that accelerate the development of new therapies for PMS and related disorders; and Ozgene, experts in precise genetic engineering, today announced the availability of a new Shank3 conditional knockout (cKO) mouse model created on the C57BL/6J background. The collaboration aims to provide researchers with simplified, predictable access to a high-quality SHANK3 resource while supporting both academic discovery and translational therapeutic programs.

A Genetically Precise Shank3 Exon 4-22 Model The new Shank3 Exon 4-22 deletion model is engineered with loxP sites flanking exons 4–22 of the Shank3 gene. When crossed with Cre-driver lines, these exons can be excised to generate a full Shank3 knockout. The exon-targeting strategy is based on previously published Ex4–22 deletion models (Wang et al., 2016; Drapeau et al., 2018), which demonstrated removal of all major murine Shank3 isoforms and exhibited behavioural, cognitive, and motor phenotypes associated with SHANK3 haploinsufficiency.

Generated using patented goGermline by Ozgene® technology, the line offers genetic accuracy, reproducibility, and improved ethical efficiency. Ozgene will serve as the global distributor, with active colonies in Indianapolis, USA, and optional housing in Perth, Australia, to enable rapid study initiation for researchers worldwide.

“This new model provides a genetically robust platform for advancing research in PMS, SHANK3-related autism spectrum disorder (ASD), and other neurodevelopmental disorders linked to SHANK3 haploinsufficiency," said Dr Frank Koentgen, Founder of Ozgene.

Designed for a Wide Range of Research Applications Because the Ex4–22 deletion removes the coding regions for all major SHANK3 isoforms, this model is expected — based on earlier published lines — to support diverse research objectives, including:

Studies of SHANK3 haploinsufficiency and synaptic biology Preclinical evaluation of gene therapies, antisense oligonucleotides (ASOs), and other modalities aimed at restoring SHANK3 function Development of translational and behavioural biomarkers Modelling rare neurodevelopmental disorders involving SHANK3 loss Fee-for-Service Platform Development by Innoser InnoSer is developing a standardised PMS preclinical testing platform, and results from these studies will be shared once validation is complete. The platform will provide behavioural, cognitive, sensorimotor, and biomarker-based assessments to support therapeutic development programs. This platform is expected to launch in late 2026, and will use the Shank3 cKO model to deliver fee-for-service studies under a commercial use license that does not include any reach-through IP claims nor downstream royalties. InnoSer does not supply animals directly to clients; all animal distribution remains exclusively through Ozgene.

“Preclinical research is a critical and complex step in developing effective therapies for rare genetic disorders like Phelan-McDermid syndrome,” said Maarten Loos, PhD, director of InnoSer Laboratories in the Netherlands. “By combining a mouse model offered through streamlined access 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.”

Access Through Standard Use Licenses Researchers can access the Shank3 Ex4–22 conditional knockout model directly from Ozgene, with multiple options including:

Study-ready experimental cohorts Breeding pairs or trios for internal use Custom background backcrossing Long-term colony management (Australia or USA) Animals are provided under standard use licenses that permit internal breeding and research. These licenses do not allow onward distribution or supply of animals to third parties; distribution is managed exclusively through Ozgene. For researchers purchasing animals from Ozgene, the quoted animal cost is the total cost, with no reach-through IP claims, no downstream royalties, and no additional usage fees for internal use.

The model will also be listed in public databases such as Mouse Genome Informatics (MGI) to increase visibility and accessibility to the research community.

“The SHANK3 community needs tools that are easy to access and straightforward to work with,” said Geraldine Bliss, Co-Founder and President of CureSHANK. “By simplifying how researchers obtain and use this model, and by supporting complementary preclinical services, we hope to accelerate the path to urgently needed therapies.”

Ordering and Technical Information Researchers interested in obtaining the Shank3 cKO model may contact:

Ozgene — Request a quote or scientific details at www.ozgene.com/contact-us CureSHANK — Learn more about access and patient-driven initiatives at www.cureshank.org/academia-industry InnoSer — Preclinical service inquiries and updates on the characterization study at https://www.innoserlaboratories.com/neurology-research-models/phelan-mcdermid-syndrome-shank3-mouse-model ABOUT OZGENE
Founded in 1999, Ozgene specialises in the generation and support of genetically modified mouse models for biomedical research. Leveraging its proprietary goGermline™ technology, OzBIG large humanisation platform, and comprehensive capabilities in model design, validation, custom breeding, and colony management, Ozgene provides ethical, efficient, and high-quality preclinical mouse model services worldwide. Ozgene also offers on-site vivarium care services, partnering with customer facilities to maintain high-quality animal care and operational efficiency through its innovative vivarium management solutions. To learn more, please visit https://www.ozgene.com.

Ozgene Media Contact
Joanne Deys
President
Ozgene LLC
+1 248 762-3626
ozgene@ozgene.com
Social media: LinkedIn

ABOUT CURESHANK
CureSHANK, a 501 (c) (3) research advocacy organization, was founded in 2018 by three parents whose children live with Phelan-McDermid syndrome (PMS), a rare, highly disabling neurodevelopmental disorder currently without cure or FDA-approved therapies. Headquartered in Los Angeles, CA, CureSHANK’s mission is to expedite life-changing therapies for people with PMS and other SHANK3-related disorders by bridging the translational research gap. To learn more, please visit https://www.cureshank.org.

CureSHANK Media Contact
Geraldine Bliss
Co-founder and President, CureSHANK
connect@cureshank.org
https://www.cureshank.org/news
CureSHANK social media: LinkedIn, Instagram, Facebook, X/Twitter, TikTok

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. To learn more, please visit https://www.innoserlaboratories.com/about-innoser/.

InnoSer Media Contact
Casey Kilmore
Senior Marketing and Communications Manager
InnoSer
(+31) 626206909
ckilmore@innoserlaboratories.com

END

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