Vironexis Biotherapeutics Launches First AAV-delivered Cancer Immunotherapy Trial with FDA IND Clearance

Vironexis Biotherapeutics has emerged from stealth mode, announcing its mission to revolutionize cancer treatment by using AAV-delivered T-cell immunotherapy. The company has unveiled its proprietary TransJoin™ AAV Gene Therapy Platform and a diverse pipeline of over ten product candidates targeting blood-based cancers, the prevention of solid tumor metastasis, and a cancer vaccine.

The company's $26 million seed financing was led by Drive Capital and Future Ventures, with participation from Moonshots Capital and Capital Factory. Vironexis has also received FDA IND clearance for its first gene therapy product, VNX-101, which aims to treat CD19+ acute lymphoblastic leukemia. The company is preparing to start a Phase 1/2 clinical trial of VNX-101 in late 2024, marking the debut of AAV-delivered cancer immunotherapy in a clinical setting. VNX-101 has received Fast Track and Rare Pediatric Disease Designations from the FDA.

Samit Varma, co-founder and CEO of Vironexis, expressed excitement about the company's launch and technological advancements. He emphasized the potential for their novel technology to improve upon existing T-cell immunotherapy methods like CAR-T and bispecific antibodies, offering enhanced safety, efficacy, and durability, while also simplifying the manufacturing process and reducing treatment burdens for patients. With an expansive pipeline and a clinic-ready lead program, Vironexis aims to quickly transform cancer treatment.

TransJoin technology enables a patient's body to express engineered transgenes that redirect T cells to tumor cells. This single-dose technology creates a bridge that connects T cells and tumor cells, promoting long-term T-cell-mediated tumor destruction. The foundational technology was licensed from Nationwide Children’s Hospital and is based on research led by Dr. Timothy Cripe, a co-founder of Vironexis. Dr. Cripe is a renowned scientist in gene therapy who also contributed to the development of ZOLGENSMA®.

Dr. Cripe highlighted the potential of AAV as a delivery technology due to its ability to provide long-term therapeutic protein expression. He noted that this capability could address challenges associated with first-generation T-cell immunotherapies and expressed anticipation about translating this approach into clinical outcomes.

Vironexis is also seeking FDA pre-IND input for its second program, VNX-202, aimed at preventing metastatic HER2+ cancers. The company plans to initiate a Phase 1/2 clinical trial for VNX-202 in 2025. Other product candidates include treatments for multiple myeloma, B-cell lymphoma, various metastases prevention, and a cancer vaccine, among others. Vironexis also intends to partner for further development of a treatment for systemic lupus erythematosus.

Molly Bonakdarpour, Partner at Drive Capital, praised Vironexis for its groundbreaking technology, strong preclinical data, and the founding team's expertise. She noted the company's productivity and looked forward to its transition to a clinical-stage entity.

Steve Jurvetson, Co-Founder of Future Ventures, highlighted the broad applicability of the TransJoin technology, which spans treatments across various cancer types and immune disorders. He commended the Vironexis team for rapidly building a comprehensive pipeline.

The TransJoin technology works by enabling the expression of a secreted T cell engager that binds to both tumor cells and T cells. After a single intravenous infusion, the liver continuously secretes the bispecific protein, which redirects T cells to tumor cells, resulting in long-term, consistent tumor cell killing. This approach minimizes toxicity and adverse events.

Vironexis is committed to transforming cancer treatment with its innovative AAV-delivered T-cell immunotherapy. The company aims to overcome current immunotherapy challenges with its TransJoin™ AAV Gene Therapy Platform, offering a promising future for cancer patients.

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