The US Food and Drug Administration (FDA) has cleared Siren Biotechnology’s first Investigational New Drug (IND) application for its AAV immuno-gene therapy for cancer, enabling the initiation of its first-in-human clinical trial. 

The IND clearance supports the evaluation of Siren’s lead investigational programme in adult patients with recurrent high-grade glioma.  

Siren believes this marks the first FDA-cleared IND for an adeno-associated virus (AAV)-based therapy in an oncology indication, underscoring the growing potential of gene therapy approaches in cancer. 

“This IND clearance represents a defining moment for Siren as we transition from a preclinical-stage company into the clinic,” said Nicole Paulk, Founder, CEO, and President of Siren Biotechnology. “Reaching this milestone reflects years of focused platform development, rigorous translational work, and close engagement with regulators, and it positions us to begin evaluating our approach in patients.” 

Siren plans to initiate a first-in-human clinical study in adult patients with recurrent high-grade glioma. The research was made possible by funding from the California Institute for Regenerative Medicine (CIRM), a state of California Agency that funds regenerative medicine, stem cell, and gene therapy research. 

The IND clearance follows the FDA’s prior granting of Orphan Drug and Rare Pediatric Disease designations for the programme and reflects the completion of the nonclinical and CMC activities required to support clinical evaluation. 

High-grade gliomas, including glioblastoma, are among the most aggressive and lethal primary brain tumours. Current treatments include surgery, radiation, and chemotherapy, all of which offer limited benefit.