Gene therapy startup approved for unique trial

Vironexis Biotherapeutics has recently emerged from stealth mode with the ambitious plan of initiating what is claimed to be the first-ever clinical trial for a cancer immunotherapy delivered using an adeno-associated virus (AAV). This startup's innovative approach combines the widely-used immunotherapy method in oncology with AAV, a popular gene therapy delivery tool. The objective is to provide patients with a one-time treatment that activates the immune system's T cells, ensuring continuous tumor attack over an extended period.

The company's initial candidate, named VNX-101, is set to be tested in certain patients with acute lymphoblastic leukemia in a Phase 1/2 trial. This candidate has already received two designations from the Food and Drug Administration (FDA) aimed at expediting the development of treatments for critical medical needs.

Vironexis licensed its principal technology, called TransJoin, from Nationwide Children's Hospital in Columbus, Ohio, a prominent center for gene therapy research. The technology, developed under the leadership of Timothy Cripes, a pediatric division chief at the hospital, was published in Science Advances in 2022. Cripes is one of the co-founders of Vironexis, along with Brian Kaspar, who is known for the scientific development of Zolgensma, and CEO Samit Varma.

Interestingly, Vironexis isn't the only company exploring the potential of gene therapy in cancer treatment. Siren Biotechnology also came out of stealth last year with its own immune-gene therapy technology currently in preclinical testing.

Starting with $26 million in seed funding, Vironexis's financial backing comes from leading investors such as Drive Capital and Future Ventures, with additional participation from Moonshots Capital and Capital Factory. The company asserts that its method could lead to therapies that are safer, more effective, longer-lasting, and easier to manufacture and administer compared to existing treatments. However, AAV gene therapies come with their own set of challenges. The manufacturing process is intricate, and the risks associated with gene therapy are considerably different from those encountered with traditional medications.

Overall, Vironexis claims to have more than 10 product candidates in its development pipeline. The company aims to begin clinical trials for VNX-101 in the fourth quarter. Additionally, they are working to secure FDA approval to start a clinical trial for VNX-202 next year, which is intended to prevent metastatic HER2+ cancer. Beyond these, Vironexis is developing a variety of products aimed at treating blood-based cancers, preventing solid tumor metastases, and even a cancer vaccine.

In summary, Vironexis Biotherapeutics is set to revolutionize cancer treatment by merging gene therapy with immunotherapy. Backed by significant seed funding and a robust pipeline of candidates, the company is poised to make significant strides in the field of oncology, offering hope for more effective and enduring cancer treatments.