Value Proposition
· Tumor-selective oncolysis: T‑VEC is an engineered oncolytic HSV‑1 that preferentially replicates in and lyses tumor cells, providing direct cytolytic reduction of cutaneous neurofibromas in preclinical models
· Local, anti-tumor immune activation: preclinical evidence that tumor cell lysis and local GM-CSF expression promotes tumor antigen presentation and subsequent immune responses
· Safe and minimally invasive: intratumoral injections of T-VEC in dermally implanted NF1 xenografts demonstrate treatment of individual symptomatic lesions with minimal systemic exposure, indicating a possible reduction in systemic toxicity compared with systemic drugs
· Reduced risk of permanent skin injury: reduces scarring and permanent skin damage compared to laser, surgical, or chemical therapies
Unmet Need
Cutaneous neurofibromas (cNFs) are extremely common, often numerous, and frequently cause disfigurement, pain, itching, and other negative impacts to quality‑of‑life in NF1 patients. There are currently no broadly effective, safe, and practical local therapies that can be applied to many superficial lesions: surgical excision is impractical for large numbers of lesions, and local ablative approaches (lasers, chemicals) risk scarring and permanent skin injury. Systemic treatments such as MEK inhibitors carry significant systemic toxicities that are disproportionate for benign, localized lesions. Thus, there is a clear need for a locally delivered, tumor‑selective therapy that safely reduces or eradicates symptomatic cNFs while minimizing scarring and systemic adverse effects.
Technology Description
Researchers at Johns Hopkins have demonstrated that the oncolytic HSV-1 T-VEC is an effective treatment for cutaneous neurofibromas in preclinical models (dermally implanted NF1 xenografts). The virus preferentially infects and replicates in tumor cells, causing direct tumor cell lysis and release of tumor antigens; local GM‑CSF expression promotes immune cell recruitment and adaptive responses. Delivered by intratumoral injection into accessible cNF lesions, T‑VEC enables focal treatment of individual symptomatic cNFs with minimal systemic exposure in these models. Preclinical studies also show productive viral propagation in NF1 tumor cells and complete eradication of dermally implanted NF1 xenografts, further supporting clinical evaluation for adults with symptomatic cNFs.
Stage of Development
Preclinical proof-of-concept experiments have been completed.
Data Availability
Data available upon request.
Publication
- WO2026050737
