- RGX-202, a potential one-time AAV Therapeutic for the treatment of Duchenne that includes an optimized transgene for a novel microdystrophin, continues to be well-tolerated in three patients from dose level 1 (1x1014 GC/kg)
- Initial biomarker data in two patients who completed three-month assessment demonstrate robust microdystrophin expression with localization to the muscle cell membrane
- Patient aged 4.4 years old had expression level at 38.8% of control
- Trial dose escalation expected by end of 2023
- Pivotal dose determination and initiation of pivotal program anticipated in 2024
- Plan to use RGX-202 microdystrophin as a surrogate endpoint to support a Biologics License Application filing using the accelerated approval pathway
- RGX-202 development program uses commercial-ready cGMP material from the REGENXBIO Manufacturing Innovation Center
- Conference call today, Tuesday, October 3, 2023, at 4:30 p.m. ET
REGENXBIO will host a conference call Tuesday, October 3 at 4:30 p.m. ET with principal investigator, Dr. Aravindhan Veerapandiyan, to discuss these results and the RGX-202 program.
Listeners can register for the webcast via this link. Analysts wishing to participate in the question and answer session should use this link. A copy of the slides being presented will be available via the Company's investor website. Those who plan on participating are advised to join 15 minutes prior to the start time. A replay of the webcast will also be available via the Company's investor website approximately two hours after the call's conclusion.
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