REGENXBIO Showcases Advancements in Gene Therapy Research

Revolutionizing Duchenne Muscular Dystrophy Treatment

REGENXBIO Inc. (NASDAQ: RGNX) has exciting news. The company recently announced that it will present new interim biomarker data from the Phase I/II portion of the AFFINITY DUCHENNE trial. This trial evaluates RGX-202 for treating Duchenne muscular dystrophy, a severe genetic disorder. This presentation will be showcased at the upcoming Muscular Dystrophy Association (MDA) Clinical & Scientific Conference.

Key Presentation Details

During the conference, several significant findings will be introduced. One highlighted session is titled "RGX-202, an investigational gene therapy for the treatment of Duchenne Muscular Dystrophy: Interim clinical data (O75)". This session will cover promising results we have seen so far in the clinical trials of RGX-202.

The session is scheduled to occur on a Wednesday morning, offering a platform for distinguished speakers. Carolina Tesi Rocha, M.D., a Clinical Professor in Neurology at Stanford School of Medicine and Stanford Children's Health, will present this essential data to attendees, emphasizing the innovation and progress made.

Insightful Pre-Clinical Research

Another equally important presentation at the conference will be led by Dr. Steven Foltz, a Senior Scientist at REGENXBIO. His session, titled "Enhanced therapeutic potential of a microdystrophin with an extended C-terminal domain (P143)" focuses on advancements made in pre-clinical research for Duchenne muscular dystrophy therapies.

Expanding the Therapeutic Landscape

The research surrounding microdystrophin holds vital implications for the future of treatment options available for patients suffering from this debilitating condition. Through continuous innovation, REGENXBIO is committed to exploring new therapeutic avenues.

REGENXBIO showcases a growing pipeline of AAV Therapeutics, including its lead product RGX-202. This commitment to harnessing gene therapy showcases a revolutionary approach to treating rare genetic conditions.

REGENXBIO's Mission and Values

Founded in 2009, REGENXBIO aims to enhance lives using the curative potential of gene therapy. Their pioneering work in AAV Therapeutics has made significant strides in developing medicines targeting various diseases.

With treatments now reaching thousands of patients, REGENXBIO's innovative platform not only focuses on conditions like Duchenne muscular dystrophy but also on other conditions such as wet age-related macular degeneration and MPS II. By providing one-time treatments, these therapies represent groundbreaking possibilities for healthcare.

Join the Conversation

The presentations made at the MDA Conference will not only highlight scientific advancements but also inspire dialogue among professionals, researchers, and stakeholders. Interested individuals can find these presentations and other related resources in the Publications section of REGENXBIO's website.

Frequently Asked Questions

What is the significance of the AFFINITY DUCHENNE trial?

The AFFINITY DUCHENNE trial is crucial for testing RGX-202's efficacy and safety in treating Duchenne muscular dystrophy, providing hope for patients.

Who will present at the MDA Conference?

Carolina Tesi Rocha, M.D., and Steven Foltz, Ph.D., will represent REGENXBIO, discussing the latest findings in gene therapy advancements.

Where can I access the presentations from the conference?

Presentations will be available in the Publications section on REGENXBIO's official website.

What is REGENXBIO's main goal?

REGENXBIO's mission is to improve lives through innovative gene therapies that offer potential curative solutions for various diseases.

How does RGX-202 work?

RGX-202 is designed to deliver gene therapy that addresses the underlying causes of Duchenne muscular dystrophy, potentially providing long-lasting benefits for patients.

About The Author

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