Olivier Danos, PhD, on Enhancing Gene Therapy for DMD

“We've started a clinical trial on Duchenne muscular dystrophy using an AAV with the micro dystrophin. We're bringing into the busy arena of gene therapy for DMD a new configuration, a product which we think has interesting differentiation. We're using AAV8 as a serotype and using our own microdystrophin, which has been designed and optimized very carefully. For instance, this is a microdystrophin which is slightly larger than the ones that have been used so far.”

REGENXBIO is advancing its gene therapy for Duchenne muscular dystrophy (DMD), RGX-202, with a clinical trial that is currently ongoing and recruiting patients. The company 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. One presentation looked at how manufacturing methods and type of target tissue affect biodistribution and expression. REGENXBIO is also looking into 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.

CGTLive spoke with Olivier Danos, PhD, chief scientific officer, REGENXBIO, to learn more about the company’s programs and technologies. He discussed RGX-202 and what characteristics differentiate it from other gene therapies for DMD in development or on the market. He also discussed further research to be done with gene therapy for DMD and what research trends are happening in the field.

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