Siren Biotechnology Becomes Clinical-Stage Company As FDA Approves IND For Its AAV-Based Cancer Therapy Targeting Recurrent High-Grade Glioma

Siren Biotechnology, known for its work in developing Universal AAV Immuno-Gene Therapy for cancer, announced that the U.S. Food and Drug Administration has cleared the Company’s first Investigational New Drug application. This approval allows Siren to move forward with its first clinical trial in humans and marks the Company’s official transition into a clinical-stage biotechnology organization. 

The IND clearance enables the clinical evaluation of Siren’s lead investigational therapy in adults with recurrent high-grade glioma, an aggressive and difficult-to-treat brain cancer. The approval is a major regulatory milestone for the Company and, according to Siren, is believed to be the first FDA-cleared IND for an AAV-based gene therapy specifically targeting an oncology indication. This milestone highlights the growing interest and progress in using gene therapy to treat cancer.

Nicole K. Paulk, PhD, Founder, CEO, and President of Siren Biotechnology, said the clearance marks an important turning point for the organization. She noted that the progress reflects years of platform development, extensive translational research, and close collaboration with regulatory agencies. She added that the Company is now prepared to begin evaluating its therapeutic approach in patients for the first time.

Siren plans to launch a first-in-human clinical study enrolling adult patients with recurrent high-grade glioma. The program builds on the Company’s Universal AAV Immuno-Gene Therapy platform, which is designed to deliver immune-modulating genetic payloads directly into tumors in a durable and localized manner.

This FDA clearance follows the earlier granting of Orphan Drug and Rare Pediatric Disease designations for the program. It also confirms the successful completion of the nonclinical and manufacturing work required to support clinical testing. The development of this research was supported by funding from the California Institute for Regenerative Medicine, a state agency that supports stem cell, regenerative medicine, and gene therapy research. The program received support under grant number TRAN1-15325.