Development of an AAV gene therapy immunotherapy for the treatment of glioblastoma
Grant Award Details
Grant Type:
Grant Number:
TRAN1-15325
Investigator(s):
Disease Focus:
Human Stem Cell Use:
Award Value:
$3,997,919
Status:
Active
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
Pre-IND
Major Proposed Activities
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
Pre-IND
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.