Construct including CT domain demonstrated higher levels of microdystrophin protein, increased muscle force, and improved resistance to damage in mice lacking dystrophin REGENXBIO's next-generation investigational gene therapy, RGX-202, is the only microdystrophin construct that includes the CT domain Findings support the positive functional data seen in Phase I/II AFFINITY DUCHENNE® trial of R...
REGENXBIO specializes in gene therapies using proven AAV technology. RGX-121, targets Hunter syndrome and is under FDA review, with a PDUFA date set for November 9, 2025. We believe the upcoming FDA decision represents a significant catalyst for RGNX shares, offering a compelling pre-approval investment opportunity.
RGX-202 demonstrating consistent evidence of positively changing disease trajectory for Duchenne All dose level 2 participants exceeded external natural history controls on all functional measures Biomarker data demonstrate consistent, robust microdystrophin expression and transduction levels across all treated ages One new participant aged 2 years at dosing had expression level at 118.6% compa...
$150 million secured at closing extends cash runway into early 2027 REGENXBIO retains additional potential non-dilutive funding opportunities, including monetization of Priority Review Voucher (PRV) and milestones from AbbVie ROCKVILLE, Md. , May 19, 2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced the closure of a non-dilutive, limited recourse royalty bond agreement of up to...
REGENXBIO Inc. (NASDAQ:RGNX ) Q1 2025 Earnings Conference Call May 12, 2025 4:30 PM ET Company Participants Patrick Christmas - Chief Legal Officer Curran Simpson - President & CEO Steve Pakola - Chief Medical Officer Mitch Chan - CFO Conference Call Participants Mani Foroohar - Leerink Partners Gena Wang - Barclays Judah Frommer - Morgan Stanley Annabel Samimy - Stifel Paul Choi - Goldman Sach...
FDA assigns PDUFA target action date of November 9, 2025 RGX-121 on track to be the first gene therapy and one-time treatment for MPS II Partner Nippon Shinyaku to lead commercialization upon potential approval REGENXBIO to lead commercial manufacturing and supply chain ROCKVILLE, Md. , May 13, 2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced the U.S. Food and Drug Administrat...
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