REGENXBIO Releases Schedule of Its ASGCT Oral Sessions and Poster Exhibits

REGENXBIO, a Rockville, Md.-based clinical-stage biotechnology company, is announcing its slate of oral and poster presentations to be made at the American Society of Gene & Cell Therapy (ASGCT)’s 28th annual meeting, which will be held in New Orleans from May 13–17, 2025 (1).

The oral presentations, according to a company press release on May 8, 2025, will report data from late-stage clinical trials of two drug candidates: RGX-121 (clemidsogene lanparvovec), for the treatment of mucopolysaccharidosis (MPS) type II, also known as Hunter syndrome, and RGX-202, for treatment of Duchenne muscular dystrophy (1,2).

Hunter syndrome is a rare recessive disorder with an early age of onset, occurring predominantly in males (2). It initially presents as hydrocephalus, often followed by behavioral changes and central nervous system (CNS) involvement. Duchenne muscular dystrophy causes initial ambulatory difficulty but progresses to the point that patients cannot perform activities of daily life and must use wheelchairs (3).

Olivier Danos, PhD, REGENXBIO executive vice president and chief scientific officer, will make the oral presentation “RGX-121 (clemidsogene lanparvovec): An Investigational AAV [adeno-associated virus] Gene Therapy for the Treatment of Neuronopathic Mucopolysaccharidosis Type II” during a session on “CNS Gene Delivery for Metabolic Diseases: History and Challenges” on Wednesday, May 14 at 8 a.m. CT (1).

The other two REGENXBIO oral presentations involve RGX-202; “Development of a Commercial Manufacturing Process for RGX-202, a Systemically Delivered AAV for the Treatment of Duchenne Muscular Dystrophy” will be presented by Donald Startt, REGENXBIO executive director, Process Development, on Tuesday, May 13 at 1:30 p.m. CT, followed by Danos presenting “RGX-202, an Investigational Gene Therapy for the Treatment of Duchenne Muscular Dystrophy: Interim Clinical Data” on Friday, May 16 at 3:45 p.m. CT (1).

Poster session topics include more on Duchenne muscular dystrophy (in a mouse model), various impacts on AAV endotoxin testing, and multiple analyses of AAV vectors (1).

In June 2022, REGENXBIO opened a manufacturing innovation center for gene therapy manufacturing in Rockville, investing $65 million to enable end-to-end control of gene therapy manufacturing at the site (4). The good manufacturing practice facility enables the company to boost manufacturing of AAV vectors using its proprietary technology, with production able to be scaled up to 2000 L. The facility also implements REGENXBIO’s suspension cell culture platform, a process that the company said at the time had demonstrated increased product purity and yield.

Prior to that, in September 2021, REGENXBIO announced the closing of a collaboration and license agreement with AbbVie to develop and commercialize its gene therapy candidate, RGX-314, for treatment of wet-age-related macular degeneration, diabetic retinopathy, and other chronic retinal diseases (5).

Complete coverage of ASGCT 2025 can be found at this link.

References

1. REGENXBIO. REGENXBIO Announces Presentations at the American Society of Gene & Cell Therapy 28th Annual Meeting. Press Release. May 8, 2025.
2. Hashmi, M. S. and Gupta, V. Mucopolysaccharidosis Type II. In StatPearls, Internet edition; StatPearls Publishing, Treasure Island, Fla., updated July 25, 2023.
3. Venugopal, V. and Pavlakis, S. Duchenne Muscular Dystrophy. In StatPearls, Internet edition; StatPearls Publishing, Treasure Island, Fla., updated July 10, 2023.
4. REGENXBIO. REGENXBIO Opens State-of-the-Art Gene Therapy Manufacturing Facility. Press Release. June 9, 2022.
5. REGENXBIO. AbbVie and REGENXBIO Announce Eye Care Collaboration. Press Release. Sep. 13, 2021.