REGENXBIO's Upcoming Presentation at World Muscle Society Event
REGENXBIO's Participation in International Congress
REGENXBIO Inc. (Nasdaq: RGNX) is excited to announce that its Chief Medical Officer, Steve Pakola, M.D., will be presenting at the International Congress of the World Muscle Society. This significant event will take place in Vienna, Austria, from October 7 to October 11.
Insights on RGX-202 and Its Clinical Data
The upcoming podium presentation will focus on new analyses derived from previous 12-month functional data related to the Phase I/II trial of RGX-202. This session will specifically highlight patient improvements on the North Star Ambulatory Assessment (NSAA), utilizing the established cTAP disease progression model from the Collaborative Trajectory Analysis Project. Notably, RGX-202 maintained a favorable safety profile, with no serious adverse events or concerns raised during the study.
Performance Compared to Control Groups
RGX-202's participants outperformed baseline-matched external natural history controls across all functional measures, further validating the therapy’s potential. This reinforces RGX-202’s positioning as a standout candidate in the therapeutic space for Duchenne muscular dystrophy.
Details of the Presentation
This engaging presentation is titled "RGX-202: An Investigational Gene Therapy for the Treatment of Duchenne Muscular Dystrophy: Interim Clinical Data." Dr. Steve Pakola, who serves as REGENXBIO's Chief Medical Officer, will lead the session during Short Oral Presentations 1, focused on updates from SMA and DMD trials.
The talk is scheduled for October 8 at 3:30 PM CET, accompanied by a poster presentation (P425) for additional insights.
Exploring RGX-202's Innovative Approach
RGX-202 is characterized as a groundbreaking investigational gene therapy aimed at improving functional outcomes in patients with Duchenne muscular dystrophy. What sets RGX-202 apart is its differentiated microdystrophin construct, which effectively encodes critical regions of natural dystrophin, including the C-Terminal (CT) domain.
Designed with unique features such as codon optimization, RGX-202 strives to enhance gene expression, boost protein translation efficiency, and minimize immunogenic responses. This gene therapy is adept at supporting targeted microdystrophin expression across both skeletal and cardiac muscle, facilitated by the NAV AAV8 vector combined with a specialized muscle-specific promoter (Spc5-12). REGENXBIO manufactures RGX-202 utilizing its proprietary, high-yield NAVXpress suspension-based platform.
About REGENXBIO and Its Mission
Founded in 2009, REGENXBIO is at the forefront of biotechnology, dedicated to transforming healthcare through the promising field of gene therapy. The company's mission is to enhance lives by harnessing the curative potential found in their innovative gene therapies. Their late-stage pipeline includes groundbreaking treatments for rare and retinal diseases, with RGX-202 specifically targeting Duchenne muscular dystrophy, alongside other therapies developed under partnerships with Nippon Shinyaku and AbbVie.
REGENXBIO’s AAV platform has successfully treated thousands of patients, showcasing the transformative potential these therapies can offer in reshaping health outcomes. As they continue to push boundaries, the vision for REGENXBIO remains focused on changing healthcare dynamics for countless individuals.
Frequently Asked Questions
What is RGX-202?
RGX-202 is an investigational gene therapy designed to improve outcomes for patients with Duchenne muscular dystrophy.
When will the presentation take place?
The presentation will occur on October 8 at 3:30 PM CET at the World Muscle Society Congress.
Who will present the clinical data on RGX-202?
Dr. Steve Pakola, the Chief Medical Officer of REGENXBIO, will lead the presentation.
What makes RGX-202 stand out in its category?
RGX-202 features a unique microdystrophin construct aimed at enhancing protein function, setting it apart from other therapies.
Where can I find more information about REGENXBIO?
More details about REGENXBIO and its therapies can be found on their official website.
About The Author
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