On April 27, 2026 Siren Biotechnology reported the publication of its first peer-reviewed manuscript in Molecular Therapy Oncology, alongside upcoming presentations at the American Society of Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper) 2026 Annual Meeting.
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The publication reports preclinical data supporting Siren’s Universal AAV Immuno-Gene Therapy platform and its application to high-grade glioma, a setting with significant unmet need and limited therapeutic options.
Publication Highlights Platform Potential and Translational Depth
The study demonstrates that localized delivery of AAV-encoded cytokines enables sustained intratumoral expression and drives robust anti-tumor activity across multiple preclinical models of high-grade glioma, including human organoids and orthotopic in vivo systems.
Across these studies, treatment was associated with tumor regression and prolonged survival, alongside evidence of tumor-localized immune activation and transcriptional reprogramming.
The study includes extensive in vivo validation across multiple orthogonal glioma models, representing one of the most comprehensive preclinical datasets reported to date for AAV-based approaches in oncology.
The publication also establishes a foundation for the continued clinical development of Siren’s platform in high-grade glioma.
"This paper represents the most complete view to date of how our platform performs across systems that matter for translation," said Nicole K. Paulk, PhD, Founder and CEO of Siren Biotechnology. "We were deliberate about building a dataset that goes beyond a single model or readout, and instead shows reproducible consistency across orthogonal approaches. That level of rigor is important as we continue advancing into the clinic."
Access the full open-access manuscript here.
Upcoming ASGCT (Free ASGCT Whitepaper) 2026 Presentations
Siren Biotechnology will present new data spanning preclinical and CMC advances at the ASGCT (Free ASGCT Whitepaper) 2026 Annual Meeting in Boston, MA.
MRI-Guided Convection-Enhanced Delivery of an AAV-hIFNβ Vector Achieves Targeted Brain Retention and Durable Transgene Expression in a Large-Animal Model
Wednesday, May 13, 2026, 5:00 – 6:30 pm EST, Poster Hall, Abstract #2241
End-to-End Device Compatibility and In-Use Stability Assessment of an AAV Gene Therapy Delivered by Convection-Enhanced Delivery
Friday, May 15, 2026, 8:15 – 8:30 am EST, MCEC Room 162AB
"Together, these presentations extend the platform story into delivery, manufacturability, and real-world use considerations," added Dr. Paulk. "We’re excited to share data that we believe helps define what effective AAV-based therapies in oncology can look like."
This research was additionally made possible by funding from the California Institute for Regenerative Medicine (CIRM), a state of California Agency that funds regenerative medicine, stem cell, gene therapy research, and clinical trials (Grant number: TRAN1-15325).
(Press release, Siren Biotechnology, APR 27, 2026, View Source [SID1234664826])