Drug intelligence / Profile preview

AAV-hIFNβ

Development stage
Phase 1
Lead developer
Siren Biotechnology
Modality
Gene Therapies, Cytokines & Interferons → Recombinant Proteins and Enzymes
Administration
Intratumoral
01

Overview

AAV-hIFNβ (also known as SRN-101) is an experimental, clinical-stage adeno-associated virus (AAV)-vectored immuno-gene therapy developed by Siren Biotechnology for the treatment of high-grade gliomas, including glioblastoma (GBM). It consists of a recombinant AAV vector (typically AAV9) carrying the gene for human interferon-beta (hIFNβ). Delivered locally via MRI-guided convection-enhanced delivery (CED), the vector transduces tumor and surrounding brain cells to continuously produce and secrete hIFNβ, thereby overcoming the short half-life and systemic toxicities associated with recombinant IFNβ protein. Secreted hIFNβ signals through the type I interferon receptor (IFNAR) to induce tumor cell apoptosis, remodel the immunosuppressive tumor microenvironment, and stimulate a potent anti-tumor immune response. The FDA has granted SRN-101 Orphan Drug, Rare Pediatric Disease, and Fast Track designations for high-grade gliomas.

Other names
AAV-IFN-bAAV-IFN-βAAV-hIFNbAAV-hIFNβAAV9-hIFNbAAV-9-hIFNbAAV 9-hIFNbAAV9-hIFNβscAAV9-hIFNbscAAV-9-hIFNbscAAV 9-hIFNbscAAV9-hIFNβ
02

Targets

IFNAR (Immune system modulation via type I interferon receptor)IFNAR2 (Interferon alpha/beta receptor subunit 2)IRF9 (Interferon regulatory factor 9)JAK1 (Janus kinase 1)STAT1 (Signal transducer and activator of transcription protein 1)STAT2TYK2 (Tyrosine kinase 2)IFNAR1 (Interferon alpha/beta receptor 1)

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