Vironexis emerges with $26M, launches rare disease gene therapies

Vironexis Biotherapeutics has recently come out of stealth mode, revealing its endeavors over the past three years and securing $26 million in funding. The San Diego-based gene therapy company has developed a gene therapy that is ready for clinical trials and has been granted both FDA fast track and rare pediatric disease designations. The seed funding round was led by Drive Capital and Future Ventures, with additional investments from Moonshots Capital and Capital Factory. The financial backing will support Vironexis in its mission to create off-the-shelf, single-dose gene therapies aimed at treating cancer more effectively, safely, and durably.

The company's current focus includes a preclinical pipeline of at least ten candidates, one of which is a cancer vaccine. Most of these candidates target blood-based cancers and the prevention of solid tumor metastasis. These programs are based on the company's proprietary AAV gene therapy platform known as TransJoin. This platform is designed to link T cells and tumor cells to maintain steady serum levels of the therapy, thereby enabling consistent T-cell-mediated tumor cell destruction. Additionally, the low-dose AAV delivery method aims to minimize toxicity and adverse reactions.

Steve Jurvetson, co-founder of Future Ventures, expressed enthusiasm for the TransJoin technology. He highlighted its broad applicability, which extends to treatments for blood-based cancers, solid tumor metastasis prevention, cancer vaccines, and immune disorders. Jurvetson emphasized that the platform's versatility makes it exceptional.

The company's lead candidate, VNX-101, has gained FDA approval to proceed with in-human testing. It will be evaluated as a treatment for patients with CD19+ acute lymphoblastic leukemia. Vironexis plans to begin enrollment for a phase 1/2 trial in the last quarter of this year. VNX-101 has received both fast track and rare pediatric disease designations from the FDA. The fast track designation is intended to expedite drug development and review processes, while the rare pediatric disease designation aims to incentivize the development of drugs for rare pediatric diseases. However, the FDA plans to phase out the rare pediatric designation priority review voucher program after September 30.

Notably, the VNX-101 study will mark the first clinical trial of an AAV-delivered cancer immunotherapy. Vironexis CEO and co-founder Samit Varma expressed optimism about their novel technology, stating it improves upon existing T cell immunotherapy methods like CAR-T and bispecific antibodies. Varma believes their approach could significantly enhance the safety, efficacy, and durability of these drug classes, simplify manufacturing, and reduce the treatment burden on patients.

Founded three years ago, Vironexis licensed its platform science from Nationwide Children’s Hospital in Columbus, Ohio. The research on the TransJoin platform was led by Timothy Cripe, M.D., Ph.D., the hospital’s chief of pediatric hematology/oncology/bone and marrow transplant, and a co-founder of Vironexis. Cripe pointed out the unique benefits of AAV delivery, particularly its ability to provide long-term, continuous expression of therapeutic proteins, which could address the challenges faced by first-generation T cell immunotherapies like CAR-T.

Joining Cripe and Varma in the founding team is Brian Kaspar, Ph.D., a scientist-entrepreneur who previously founded AveXis. AveXis was acquired by Novartis in 2020 and is known for pioneering the AAV gene therapy Zolgensma.

Vironexis is also developing a second program, VNX-202, aimed at preventing metastatic HER2+ cancer, including breast cancer and other tumor types. The company hopes to initiate clinical testing for this candidate next year. Additionally, Vironexis is working on a cancer vaccine for GP350+ nasopharyngeal cancer and an investigational treatment for CD19+ systemic lupus erythematosus, the latter of which they plan to partner for future development.