#United States #New Orleans #Duchenne #REGENXBIO #RGX-121

Introduction

REGENXBIO Inc. has recently announced its participation in the upcoming American Society of Gene & Cell Therapy (ASGCT) 28th Annual Meeting, set to take place in New Orleans from May 13 to May 17, 2025. This significant gathering will spotlight REGENXBIO's cutting-edge advancements in gene therapy, particularly showcasing their innovative approaches and clinical data.

Highlighted Presentations

The presentations from REGENXBIO will illustrate the company's comprehensive capabilities across various stages of gene therapy development, including research, clinical trials, and manufacturing. Key highlights include:

1. RGX-121 for MPS II Treatment: An exploration of the ongoing late-stage clinical trials for RGX-121, also known as clemidsogene lanparvovec, designed to treat Mucopolysaccharidosis Type II. This investigational gene therapy offers hope for patients impacted by this rare genetic disorder.

2. RGX-202 for Duchenne Muscular Dystrophy: Further discussion will delve into RGX-202, an investigational gene therapy aimed at treating Duchenne muscular dystrophy (DMD). The interim clinical data is expected to provide insights into its efficacy and potential as a breakthrough treatment in the market.

3. Manufacturing Innovations: REGENXBIO will also highlight advancements in the manufacturing of RGX-202, focusing on their development of a commercial manufacturing process that emphasizes achieving unparalleled purity levels essential for gene therapy applications.

Oral Presentations

The event will feature several oral presentations:

• - Development of a Commercial Manufacturing Process for RGX-202: Presented by Donald Startt, Executive Director of Process Development, this session will cover the progress in developing a packaging process for AAV-mediated gene therapies.
• - Investigational AAV Gene Therapy for MPS II: Olivier Danos, Ph.D. and Executive VP, will provide insights into the challenges and expectations surrounding the treatment of this metabolic disease with RGX-121.
• - Interim Clinical Data for RGX-202: Also led by Dr. Danos, this session will reflect on the findings from the clinical trials of RGX-202, promising participants the latest updates on its clinical journey.

Poster Presentations

The conference will also showcase various poster presentations, which include:

• - Impact of Container Material on Viral Testing: Understanding how collection container material affects viral testing outcomes by Amanda Zhang.
• - Microdystrophin for DMD: Benjamin Heithoff, Ph.D. will discuss the implications of using AAV-expressed microdystrophin with extended C-terminus to enhance muscle function in DMD mouse models.
• - Novel In Vitro Methods: Research led by Justin Glenn, Ph.D. will showcase the development of in vitro techniques to analyze TLR9 stimulation, a vital aspect of gene therapy efficacy.

About REGENXBIO

Founded in 2009, REGENXBIO is committed to harnessing the therapeutic potentials of gene therapy, particularly focusing on adeno-associated virus (AAV) technologies. Their clinical pipeline includes innovative, one-time treatments for various rare diseases which have the potential to redefine healthcare for numerous patients worldwide. With a commitment to scientific excellence and patient-centric outcomes, REGENXBIO is at the forefront of gene therapy innovations. Thousands of patients have benefited from their therapies, including Novartis’ ZOLGENSMA.

Conclusion

As the ASGCT Annual Meeting approaches, REGENXBIO’s presentations set the stage for invaluable scientific exchange and collaboration within the gene therapy field. Attendees can anticipate a wealth of knowledge regarding the future landscape of treatments for genetic disorders. For more information about REGENXBIO and their pioneering work in gene therapy, visit www.REGENXBIO.com.