Expert Interview
Discussing the phase I/II AFFINITY DUCHENNE clinical trial data on RGX-202 from REGENXBIO presented at WMS 2023
Ticker(s): RGNXInstitution: SUNY Downstate Medical Center
- Director of the Muscular Dystrophy Association Clinic and Associate Professor of Neurology at SUNY Downstate Medical Center.
- Treats 8 patients with ATTR-PN, 15 patients for Friedreich’s Ataxia and 90 patients with myasthenia gravis.
- Research Interests in HIV-related neuropathy, inflammatory myopathies and has a clinical specialty in Neuromuscular disease.
Can you explain the mechanism of action of RGX-202 and how it differentiates from other treatments available for Duchenne, especially considering its novel microdystrophin transgene?
Added By: catalin_adminBased on the interim data from the Phase I/II AFFINITY DUCHENNE™ trial, how does RGX-202's efficacy compare to other Duchenne treatments? Are there specific outcomes, like the robust microdystrophin expression, that stand out in its effectiveness?
Given that RGX-202 aims to provide a one-time AAV therapeutic for Duchenne, how does it address the unmet medical needs in Duchenne therapy?
Added By: catalin_adminFrom the data presented, how would you characterize the safety profile of RGX-202? Are there any significant adverse events or side effects that clinicians should be aware of?
- The initial biomarker data indicates robust microdystrophin expression in muscle tissue. How significant are these findings, and what implications might they have for the broader Duchenne patient population?
With the plan to escalate the trial dose by the end of 2023 and initiate a pivotal program in 2024, what are your expectations for the global reception and potential adoption of RGX-202?
Given the unique construct of RGX-202 and its potential impact, how do you see the competitive landscape shaping up for RGX-202 in the Duchenne market? Are there any potential competitors or emerging treatments that might challenge its position?
Given the ongoing medical advancements in Duchenne and the potential of gene therapies, where do you position RGX-202 in the broader treatment paradigm for Duchenne?
Considering the severe and progressive nature of Duchenne, how transformative do you believe RGX-202 could be in changing the standard of care for Duchenne patients?
Added By: catalin_adminWith RGX-202 development program using commercial-ready cGMP material from the REGENXBIO Manufacturing Innovation Center, can you provide insights into the potential scalability and production capabilities for RGX-202?
Added By: catalin_adminAre You Interested In These Questions?
Slingshot Insights Explained
Expert research benefits investors by giving them timely access to unbiased real world perspectives on highly specialized topics. Slingshot Insights' crowdfunded model makes this access available at a fraction of the cost of other expert networks.
Reason
*Slingshot Insights provides access to information, not investment advice. We work to support you and facilitate access to experts; however we are not responsible for monitoring calls for the disclosure of MNPI. You should obtain financial, legal and tax advice from your qualified and licensed advisers before deciding to invest in any security.
