March 23, 2026 —
Siren Biotechnology has announced that the U.S. Food and Drug Administration (FDA) has cleared an Investigational New Drug (IND) application submitted by the University of California, San Francisco (UCSF) to initiate a Phase 1 clinical trial of SRN-101, an investigational AAV-based gene therapy for patients with recurrent high-grade glioma.
The IND clearance enables the launch of a single-center, investigator-initiated Phase 1 study at UCSF led by Dr. Nicholas Butowski, Professor of Neurological Surgery and Neuro-Oncology. The study will evaluate the safety and biological activity of SRN-101 in adults with recurrent high-grade glioma, one of the most aggressive forms of brain cancer.
SRN-101 is an experimental gene therapy developed by Siren Biotechnology that utilizes an adeno-associated virus (AAV) vector to deliver therapeutic genes directly to tumor cells in the brain. AAV vectors are widely used in gene therapy due to their favorable safety profile and ability to enable targeted gene delivery and sustained gene expression.
The upcoming Phase 1 study will generate important early clinical and translational data that may guide future development of the therapy. Siren Biotechnology is supporting the trial by providing the investigational study drug and collaborating with the UCSF research team.
Recurrent high-grade gliomas remain among the most difficult cancers to treat, with limited therapeutic options available. Standard treatments typically include surgery, radiation therapy, and chemotherapy, but recurrence rates remain high and survival outcomes are poor. As a result, researchers are increasingly exploring innovative approaches such as AAV-mediated gene therapy to target tumor biology more directly.
The clinical trial has also received support from the California Institute for Regenerative Medicine (CIRM), a state agency that funds research and clinical development in regenerative medicine, stem cell therapy, and gene therapy. Funding from CIRM helped enable the translation of the research into a clinical study.
Investigators hope that evaluating SRN-101 in patients with recurrent high-grade glioma will help determine whether AAV gene therapy approaches can offer new therapeutic possibilities for individuals facing this devastating disease.
With the IND clearance now secured, the study represents an important step toward advancing AAV-based gene therapy strategies for brain cancer, an area where innovative treatment options are urgently needed.
