Vironexis Biotherapeutics emerged from stealth with plans to start what it claims will be the first-ever clinical trial testing a cancer immunotherapy delivered with the help of an adeno-associated virus, or AAV.
The startup’s technology combines an AAV — a common gene therapy delivery tool — with an immunotherapy approach now widely used in oncology. The idea is to give patients a one-time treatment that spurs the immune system’s T cells into continuously attacking tumors over the long term.
Vironexis’s first prospect is known as VNX-101 and it will be tested in certain people with acute lymphoblastic leukemia in a Phase 1/2 trial, the company said Thursday. VNX-101 has received a pair of designations from the Food and Drug Administration meant to encourage and speed up development of treatments for pressing medical needs.
The company licensed its core technology, dubbed TransJoin, from Nationwide Children’s Hospital in Columbus, Ohio, a leading hub for gene therapy research. Nationwide’s Timothy Cripe, a pediatric division chief, led work on the technology that was published in Science Advances in 2022. Cripe is one of the company’s founders, along with Brian Kaspar — the scientific founder of the company that developed Zolgensma — and CEO Samit Varma.
Vironexis is not the only company thinking about harnessing the power of gene therapy to treat cancer in a different way. Siren Biotechnology emerged from stealth last year with its own “immune-gene therapy” technology in preclinical testing.
Vironexis is starting off with $26 million in seed financing led by Drive Capital and Future Ventures. Moonshots Capital and Capital Factory also participated in the funding.
The company says its approach has the potential to produce safer, more effective, longer-lasting and more easily manufactured and administered therapies than those currently available. But AAV gene therapies have issues of their own; manufacturing is complex and the approach offers risks that are much different than pills a patient can stop taking if they experience issues.
All told, Vironexis says it has more than 10 product candidates in the pipeline. It plans to advance VNX-101 into the clinic in the fourth quarter and is working to obtain FDA permission to start a clinical trial for VNX-202 to prevent metastatic HER2+ cancer next year. It also has a range of other products designed to treat blood-based cancers and prevent solid tumor metastases, as well as a cancer vaccine.
