Olivier Danos, PhD, on Refining and Specifying AAV Gene Therapy

“The work that will be presented here is indication agnostic, it’s on the technology. We’re playing our part to try to solve the outstanding problems with AAV technologies, such as the lack of specificity of the vectors as they stand today, the immunogenicity, the toxicities, all these are problems that everybody's trying to solve. And through our programs, we come up with observations, questions and possible solutions tosolve these problems.”

REGENXBIO presented data from multiple of its programs focused on adeno-associated virus vector gene therapies at the American Society of Gene and Cell Therapy (ASGCT) 2023 Annual Meeting, held May 16-20, in Los Angeles, California.

CGTLive spoke with Olivier Danos, PhD, chief scientific officer, REGENXBIO, to learn more about the data presented at the ASGCT meeting. He discussed how the focus of a lot of REGENXBIO’s programs and developing technologies right now is on addressing ongoing challenges with AAV gene therapies, including specificity, immunogenicity, and toxicity. He also touched on the challenges with targeted and achieving sufficient transgene expression even after delivering gene therapy to its intended tissue. This issue is addressed in one presentation that looked at how manufacturing methods and type of target tissue affect biodistribution and expression, and Danos noted that being able to answer these questions may enable the company to create safer and more efficient gene therapies. Another strategy REGENXBIO is pursuing is modifying AAV capsids to efficiently target different tissues and thus creating specialized tissues for the eyes, brain, heart, etcetera. Further stratifying tissue-specific gene therapies, the company also presented clinical data on different routes of gene therapy administration to the eye.

Click here to read more coverage of ASGCT 2023.

REFERENCE

REGENXBIO announces presentations at the American Society of Gene & Cell Therapy (ASGCT) 25th Annual Meeting. News release. REGENXBIO. May 3, 2023. https://www.prnewswire.com/news-releases/regenxbio-announces-presentations-at-the-american-society-of-gene--cell-therapy-asgct-25th-annual-meeting-301538216.html