Siren Biotechnology has announced that its lead asset, SRN-101, an adeno-associated viral (AAV) immuno-gene therapy, has received both Orphan Drug and Rare Pediatric Disease designations from the FDA for the treatment of high-grade gliomas (HGGs), including pediatric-type diffuse HGGs. This regulatory support aims to accelerate the development of a new therapeutic option for these aggressive brain cancers, which currently have limited treatment options. The company believes SRN-101 represents a significant advancement in addressing the unmet needs of both adult and pediatric patients suffering from HGGs.
Novel Approach to High-Grade Glioma Treatment
SRN-101 is designed to harness the body’s immune response to target and destroy tumor cells. The therapy utilizes a recombinant AAV vector to deliver an engineered cytokine, aiming to stimulate a potent anti-tumor immune response. Preclinical data has demonstrated that Siren Biotechnology’s AAV immuno-gene therapies exhibit significant anti-tumor effects in brain cancer models. The company also received a $4 million CIRM TRAN1 grant to further support the development of these therapies.
According to Dr. Nicholas Butowski, MD, Neuro-Oncologist and Director of Translational Research in Neuro-Oncology at the University of California, San Francisco, SRN-101 has the potential to address a critical gap in effective HGG therapies by leveraging the body’s immune response in a targeted, innovative way, potentially representing a major breakthrough in how one of the most devastating brain cancers is treated.
Siren Biotechnology's Pipeline and Platform
SRN-101 is the first asset and indication to be revealed from Siren Biotechnology’s universal AAV immuno-gene therapy platform. The company is also developing additional technology innovations and exploring other solid tumor indications. Siren Biotechnology's CEO and Founder, Dr. Nicole K. Paulk, PhD, stated that the team has worked tirelessly to develop an approach that demonstrates remarkable promise for the treatment of high-grade gliomas but also has transformative potential for use in additional indications.
About High-Grade Gliomas
High-grade gliomas are aggressive brain tumors that pose significant challenges in treatment. Despite advancements in medical care, the prognosis for patients with HGGs remains poor, underscoring the urgent need for innovative therapeutic strategies. The FDA's Orphan Drug and Rare Pediatric Disease designations for SRN-101 highlight the critical need for new treatment options for these patient populations.
