Siren Biotechnology Launch Universal AAV Imuuno-Gene Therapy and Present Pre-Clinical Data at ASGCT

Anna Osborne
18 May 2023
Clinical Trials
Gene Editing
Gene Therapy
Raw Materials
Viral/Non-Viral Vectors
Siren Biotechnology has unveiled its groundbreaking mission to pioneer universal AAV immuno-gene therapy for cancer treatment, launching out of stealth today and presenting its first preclinical data at the ASGCT 26th Annual Meeting.

The innovative approach combines AAV gene therapy with cytokine immunotherapy, using the AAV vectors as delivery vehicles to target and modify immune cells, enhancing their ability to recognize and destroy cancer cells.

By leveraging the potential of AAVs, Siren Biotechnology seeks to develop a universal treatment approach that can be applied across different types of cancers, revolutionizing cancer treatment.

Siren’s presentation, “AAV immuno-gene therapy delivers vectorized cytokines to effectively treat high-grade gliomas,” given by AAV gene therapy expert and Siren’s Founder and Chief Executive Officer, Nicole Paulk shared details on the Company’s novel highly targeted method of destroying cancer cells.

In the preclinical studies, Siren Biotechnology demonstrated the effectiveness of their universal AAV immuno-gene therapy in various cancer models. Evaluating AAV9 vectors expressing 12 different engineered immunomodulatory interferon cytokine payloads.

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Results showed impressive tumor regression and durable anti-tumor responses, demonstrating:

  • High-grade glioma (HGG) organoids exhibited rapid reduction in tumor size.
  • Treatment of 450 mice across three prominent HGG mouse models resulted in significant tumor regression and extended survival.
  • Tumor cells underwent widespread apoptosis within 48 hours of treatment.
  • Consistent tumor eradication was observed, with no evidence of residual proliferating tumor cells and absence of cytokine payload expression by Day 7 post-treatment.

The preclinical data presented by Siren also showcased the therapy’s excellent safety profile.

The company’s work in advancing immuno-gene therapy for cancer holds promise for future clinical investigations, which will be crucial in validating the therapy’s efficacy and safety in human subjects.

“This novel approach represents a new, big, bold idea to fight cancer by combining the potential of AAV gene therapy and cytokine immunotherapy into a single modality. Our vision is for Universal AAV Immuno-Gene Therapy to become the standard of care for any solid tumor cancer. Bolstered by the results shared today at ASGCT, we’ve started our vision-led journey with an initial focus on brain and eye cancers. Attempts to treat these cancers with systemic drugs have largely failed. The unmet need for these patients is dire, and our opportunity to help is massive,” comments Dr. Paulk.

Source: Siren Biotechnology Press Release

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