A new startup attempting to develop AAV gene therapies to treat cancer emerged from stealth Thursday morning with $26 million in seed funding — and a plan to quickly get into the clinic.
Vironexis Biotherapeutics is aiming to get a Phase 1/2 trial up and running in October for what it describes as an “off-the-shelf” gene therapy, a term that has historically been connected to allogeneic CAR-T. Such therapies have proven difficult to develop, as they continue to face questions about their durability. And the FDA has not approved any.
But rather than giving patients re-engineered white blood cells from donors, Vironexis’ gene therapy is designed to deliver an engineered gene. The gene prompts the body to continuously create a protein that links tumor-fighting T cells on one side and tumor cells on the other. CEO Samit Varma likened the process to an immunotherapy that the body produces itself.
What Vironexis means by “off-the-shelf” is that there is no individualized manufacturing process for its therapy like for autologous CAR-T, where a patient’s own white blood cells are removed, genetically modified and then readministered, Varma told Endpoints News. Instead, its process allows the therapy to be made in bulk and stored in freezers at treatment sites.
“The human immune system is the most powerful drug in the world, and so we’re leveraging that power by introducing this gene that can then identify these tumor cells,” Varma said.
Timothy CripeTimothy Cripe, a co-founder whose research at Nationwide Children’s Hospital helped launch the company, also said he believes the approach is safer than traditional CAR-T. With the program, called VNX-101, the immune-fighting reaction ramps up more slowly as the protein continues to be expressed and accumulates over time.
The first kind of cancer Vironexis wants to attack is CD19-positive acute lymphoblastic leukemia, a setting where two autologous CAR-Ts are currently approved: Novartis’ Kymriah and Gilead and Kite’s Tecartus. Varma said the FDA granted the program IND clearance and that the company already has manufactured enough of VNX-101 to begin the trial next month.
Vironexis also plans to expand to other areas at a steady march, and it expects another program called VNX-202 to start a basket trial for HER2-positive cancers in 2025. Design-wise, VNX-202 contains the same AAV delivery vehicle as VNX-101, but researchers replaced the gene responsible for the anti-CD19 proteins with a gene for HER2.
Varma said its approach can allow the company to go into many different cancer settings with few modifications. So far, Vironexis has conducted preclinical testing for BCMA-, PSMA- and GD2-positive cancers, among several others. They’re also “actively” in discussions for a Series A, he said, and hopes to raise one “within the next several months.”
Brian KasparAlso co-founding Vironexis is Brian Kaspar, the former AveXis founder and CSO who helped develop the SMA gene therapy Zolgensma and was let go after it was acquired by Novartis following a data manipulation scandal in 2019. Kaspar has denied any wrongdoing.
In an interview Wednesday, Kaspar said he hopes Vironexis can follow the commercialization path AveXis and Novartis laid out with Zolgensma, which he added other gene therapy companies have struggled with. Varma also noted that he’s worked with Kaspar at multiple companies since AveXis and that the due diligence always comes back clean.
“This is actually our fourth company, post-AveXis departure, that Brian and I have done together, and in each one of those, it’s been a massive amount of diligence,” Varma said. “It’s been investigated by probably 12 different top law firms in the country, every time we did an acquisition or anytime we raised money.”
Drive Capital and Future Ventures led the seed round, with Moonshots Capital and Capital Factory also participating.