A next-generation research tool designed to accelerate therapeutic development for Phelan-McDermid syndrome and SHANK3-related neurodevelopmental disorders

Perth, Australia; USA; The Netherlands — Ozgene, CureSHANK, and InnoSer announce the availability of a new license-free Shank3 conditional knockout (conKO) mouse model, created on the widely used C57BL/6J background. The model provides a genetically robust platform for advancing research into Phelan-McDermid syndrome (PMS), SHANK3-related autism spectrum disorder (ASD), and other neurodevelopmental disorders linked to SHANK3 haploinsufficiency.

This collaboration brings together CureSHANK’s leadership in SHANK3-focused research, Ozgene’s expertise in precise genetic engineering, and InnoSer’s advanced preclinical expertise.

A genetically precise, license-free Shank3 Exon 4–22 Deletion Model

The newly released Shank3 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.

This 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.

The CureSHANK model replicates this well-established genetic design and is provided license-free, eliminating prior barriers to SHANK3-targeted therapeutic development.

The model was generated using Ozgene’s patented goGermline by Ozgene® technology, ensuring genetic accuracy, reproducibility, and improved ethical efficiency.

Collaborative roles

CureSHANK

CureSHANK supports global access to license-free SHANK3 research tools and drives initiatives that accelerate the development of new therapies for PMS and related disorders.

Ozgene

Ozgene generated the new conditional knockout line using its goGermline™ technology and will serve as the global distributor. An active colony is maintained in Indianapolis, USA, with the option to house the line in Perth, Australia, enabling rapid study initiation for researchers worldwide.

InnoSer

InnoSer is performing comprehensive phenotypic characterization of the model and is developing a standardised PMS preclinical testing platform, expected to launch in 2026. This platform will provide behavioural, cognitive, sensorimotor, and biomarker-based assessments to support therapeutic development programs.

Availability

Researchers can access the Shank3 Ex4–22 conditional knockout model through Ozgene, with multiple options including:

• Study-ready experimental cohorts
• Breeding pairs or trios
• Custom background backcrossing
• Long-term colony management (Australia or USA)

Preclinical services using this model will be available through InnoSer in 2026, following completion of characterization studies.

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

Contact and ordering information

Researchers interested in obtaining the Shank3 conditional knockout model or additional technical information may contact:

• Ozgene — Request a quote or scientific details at ozgene@ozgene.com
• CureSHANK — Learn more about access and patient-driven initiatives at 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/