Startup aims to combat cancer with 'off-the-shelf' gene therapy, raises $26M seed round

14 September 2024
A new biotech startup, Vironexis Biotherapeutics, has emerged with the objective of developing AAV gene therapies to combat cancer. Launched with $26 million in seed funding, the company aims to commence a Phase 1/2 clinical trial for its innovative gene therapy in October. This therapy is described as "off-the-shelf," a term historically linked to allogeneic CAR-T therapies, which have faced challenges in development due to questions about their durability and a lack of FDA approvals.

Unlike traditional CAR-T therapies that involve re-engineering white blood cells from donors, Vironexis' approach focuses on delivering an engineered gene. This gene prompts the body to continuously produce a protein that bridges tumor-fighting T cells and tumor cells. According to CEO Samit Varma, this method functions like an immunotherapy that the body generates on its own.

The term "off-the-shelf" for Vironexis refers to the absence of an individualized manufacturing process, which is common in autologous CAR-T therapies where a patient’s own white blood cells are modified and reinfused. Instead, their therapy can be mass-produced and stored at treatment sites, allowing for quicker and more accessible treatment options.

Varma emphasized the power of the human immune system, suggesting that their gene therapy can leverage this power by introducing a gene that identifies tumor cells. Timothy Cripe, a co-founder whose research at Nationwide Children’s Hospital contributed to the startup's launch, believes this approach is safer than traditional CAR-T. Their program, VNX-101, allows for a gradual ramp-up of the immune response as the protein accumulates over time.

The initial target for Vironexis' therapy is CD19-positive acute lymphoblastic leukemia, a cancer type for which two autologous CAR-T therapies, NovartisKymriah and Gilead's Tecartus, are currently approved. Varma disclosed that the FDA has granted IND clearance for their program and that sufficient quantities of VNX-101 have been manufactured to begin the trial next month.

Vironexis has plans to expand its focus to other cancer types. Another program, VNX-202, is expected to start a trial for HER2-positive cancers in 2025. VNX-202 uses the same AAV delivery vehicle as VNX-101, but with a gene targeting HER2 instead of CD19. According to Varma, the adaptability of their approach allows the company to address various cancer types with minimal modifications. So far, preclinical tests have been conducted for BCMA-, PSMA-, and GD2-positive cancers, among others.

The company is also preparing for a Series A funding round, aiming to secure additional investments within the next several months. Vironexis' co-founders include Brian Kaspar, the former AveXis founder and CSO who played a key role in developing the SMA gene therapy Zolgensma. Despite being let go after Novartis acquired AveXis due to a data manipulation scandal, Kaspar has denied any wrongdoing and maintains a clean record of due diligence in his ventures.

In an interview, Kaspar expressed his ambition for Vironexis to follow the commercialization path set by AveXis and Novartis with Zolgensma, a path that has proven challenging for other gene therapy companies. Varma also highlighted his extensive collaboration with Kaspar in multiple companies since their time at AveXis, emphasizing the rigorous due diligence they undergo.

Drive Capital and Future Ventures led the seed round of funding, with additional participation from Moonshots Capital and Capital Factory.

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