Development of an AAV gene therapy immunotherapy for the treatment of glioblastoma

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Grant Award Details

Grant Number:

Disease Focus:
Human Stem Cell Use:
Award Value:

Grant Application Details

Application Title:

Development of an AAV gene therapy immunotherapy for the treatment of glioblastoma

Public Abstract:
Translational Candidate

SRN-101, an experimental AAV gene therapy for treating glioblastoma

Area of Impact

Cancer - solid tumors

Mechanism of Action

Following delivery with an AAV, engineered cytokines are expressed from within the tumor to kill the tumor from the inside out, they are then further secreted to stimulate local immune cells to kill remaining tumor cells from the outside in at the tumor margin.

Unmet Medical Need

Brain tumors are the 10th leading cause of death in the US. Glioblastoma is the most common and deadliest brain cancer, with ~13,000 diagnoses annually in the US. The 5-year survival rate is 5%. Here, AAVs deliver cytokines with potent anti-tumor activity.

Project Objective


Major Proposed Activities

  • Rodent studies to determine Maximum Tolerated Dose and PK/PD studies
  • Full CMC and process development for both plasmid and viral production at GLP/GMP
  • Production of master cell banks for cGMP plasmids and human cells used for viral production
Statement of Benefit to California:
This TRAN1 award will support the development of a novel AAV immuno-gene therapy – SRN-101 – for treating patients with glioblastoma. Glioblastoma is the most common primary brain tumor in adults and second in children. This award has the potential to bring direly needed effective therapies to the 1,400 Californians diagnosed with glioblastoma each year. Siren's headquarters and employees are based here in CA so this award will support the CA economy both directly and indirectly.