Ozgene’s OzBIG technology has enabled the development of the world’s first Pax7::Foxo1 genetically-engineered mouse model for childhood cancer research. Combining Gen-H’s expertise in BAC vector design with Ozgene’s proprietary goGermline™ technology, the partnership created OzBIG, an innovative platform that empowers researchers to create large and precise gene-targeted mouse models. Leveraging this technology, Ozgene collaborated with the Children’s Cancer Therapy Development Institute (cc-TDI) to develop the Pax7-Foxo1 mouse model, a groundbreaking tool to advance research into alveolar rhabdomyosarcoma (ARMS), a rare and aggressive childhood cancer.

A Model That Mirrors ARMS

OzBIG technology’s unique capabilities directly address the challenges researchers face in replicating complex genetic conditions like ARMS. Alveolar rhabdomyosarcoma is a rare and aggressive childhood cancer driven by the Pax7::Foxo1 gene fusion. To replicate this genetic hallmark, cc-TDI required an exceptionally large and precision targeted genetic payload exceeding 15 Kilobases (Kb). Ozgene’s OzBIG technology, with its unsurpassed capacity to deliver payloads up to 240 Kb, made this feat possible. Through this groundbreaking technology, researchers can now better study the genetic drivers of ARMS and explore potential therapeutic interventions.

“Delivering large genetic payloads with precision is a technological barrier for many researchers,” said Dr. Roger Askew, Sr, Scientific Director of Ozgene. “With OzBIG, we empower researchers worldwide to tackle previously insurmountable challenges. Our collaboration with Gen-H and cc-TDI reflects how innovation in gene targeting can transform disease modelling and therapy development.”

Transformative Impact on Paediatric Oncology

The Pax7::Foxo1 mouse model is more than just a scientific breakthrough; it is a transformative tool for researchers. This model:

• Enables the study of ARMS tumour cell-of-origin, progression and biology.
• Serves as a platform for preclinical testing of targeted therapies.
• Accelerates the development of life-saving treatments for children diagnosed with ARMS.

Dr. Charles Keller, Scientific Director at cc-TDI, highlighted the significance of the model: “The Pax7::Foxo1 model is a critical step forward for alveolar rhabdomyosarcoma research. Ozgene’s expertise and technology have enabled us to create a tool that will accelerate the testing of targeted therapies, ultimately bringing hope to children and families impacted by Pax7::Foxo1 ARMS.”

The Power of Collaboration

The development of this model underscores the importance of scientific partnerships. By working with Gen-H, Ozgene was able to enhance OzBIG’s large-insertion capabilities, delivering a model that is not only reliable but also highly translatable to human disease.

“OzBIG, co-developed with Gen-H, meets the needs of modern biomedical research,” added Dr. Askew. “This collaboration demonstrates the power of combining expertise to achieve breakthroughs that change lives.”

About the Technologies and Collaborators

Ozgene’s OzBIG Technology

OzBIG (big genomic replacement) is a next-generation platform that combines Gen-H’s BAC vector technology with Ozgene’s proprietary goGermline™ technology. This synergy allows for targeted humanization and precise genetic modifications, with an unmatched capacity to deliver payloads up to 240 Kb. It empowers researchers to create optimal preclinical models, bridging the gap between discovery and clinical applications.

Gen-H

Gen-H specializes in precision genetic engineering, offering expertise in BAC vector design and recombineering techniques that enable scarless modification of large DNA constructs. By collaborating with Ozgene, they contribute to the development of complex genetic modifications through platforms like OzBIG, driving advancements in biomedical research.

cc-TDI

The Children’s Cancer Therapy Development Institute is a non-profit biotech organization dedicated to developing targeted therapies for paediatric cancers. Through innovative research, cc-TDI aims to make childhood cancer universally survivable.

• Press Release by Dr. Charles Keller: World’s First Pax7-Foxo1 Genetically Engineered Mouse Developed
• Interview with Dr. Charles Keller by Natalie Marmion: Childrens Cancer Therapy Development Institute – About Us!
• Support cc-TDI’s Mission: Donate Now