Nicole Paulk, PhD, on Applying AAV Approaches in Oncology


The CEO, founder, and president of Siren Biotechnology discussed the preclinical data she presented at ASGCT’s 2023 meeting on the use of AAV immuno-gene therapy for high-grade gliomas.

“I think this is a really exciting time. This will be the first ever AAV in the oncology space so we'll have a whole new modality to add to the arsenal of things that we can go after cancer with. Especially when we're talking about the brain cancer space in particular, it's a very challenging environment and a very challenging organ because there just hasn't been a lot of successes regardless of modality: small molecules, cell therapies, antibody drug conjugates, monoclonal antibodies, etc. Having a whole new modality class to come at these particular cancers, I think is going to be, hopefully—fingers crossed—a transformative moment so we can get more options for patients.”

Thus far, adeno-associated virus (AAV) vectors have primarily been applied for use in the delivery of gene therapies intended to treat rare, inherited, monogenic diseases. As such, application of AAVs to treat more common, non-inherited diseases like cancers has been overlooked to some extent.

Nicole Paulk, PhD, the CEO, founder, and president of Siren Biotechnology, gave a presentation entitled “AAV immuno-gene therapy delivers vectorized cytokines to effectively treat high grade gliomas” at the American Society of Gene and Cell Therapy (ASGCT) 2023 Annual Meeting, held May 16-20, in Los Angeles, California. The presentation covered 7 years’ worth of data from Paulk’s lab and Siren Biotechnology’s research regarding universal AAV immuno-gene therapies, which enable expression of engineered immunomodulatory payloads, for high grade gliomas. The research utilized both in vitro evaluations of AAVs in primary human brain organoids and in vivo evaluations in cell-line derived xenograft mice, patient-derived xenograft mice, and syngeneic allograft mice.

In an interview with CGTLive™, Paulk discussed the key findings she presented and their implications for the healthcare community. She noted that the company’s lead AAV immuno-gene therapy candidate showed improvements in overall survival in mouse models. She also discussed some of the challenges with using xenograft mouse models in studies like this and how these difficulties are overcome.

Clement N, Morris S, Kuwahara A, et al. AAV immuno-gene therapy delivers vectorized cytokines to effectively treat high grade gliomas. Presented at: American Society of Gene and Cell Therapy (ASGCT) 2023 Annual Meeting. May 16-20, 2023; Los Angeles, CA. Abstract #60.
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