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REGENXBIO Inc. reports news on its AAV gene therapy pipeline and manufacturing platform for rare, neuromuscular and retinal diseases. Recurring updates cover RGX-202 for Duchenne muscular dystrophy; clemidsogene lanparvovec (RGX-121) for MPS II; RGX-111 for MPS I; and surabgene lomparvovec (ABBV-RGX-314) for wet AMD and diabetic retinopathy.
Company announcements also include FDA regulatory communications, clinical and preclinical data presentations, financial results, operational highlights, and collaboration activity with partners including Nippon Shinyaku and AbbVie. News on the NAV and NAVXpress platforms addresses AAV vector technology, product quality, and manufacturing capabilities used across REGENXBIO's investigational programs.
REGENXBIO (Nasdaq: RGNX) dosed the first patient in the Phase IIb/III NAAVIGATE trial of investigational surabgene lomparvovec in diabetic retinopathy using suprachoroidal delivery, triggering a $100 million milestone payment from AbbVie.
NAAVIGATE will evaluate a 1.0x1012 GC/eye dose in non-proliferative DR, with a primary endpoint of >2-step DRSS improvement at one year. The Phase IIb portion plans to enroll about 135 U.S. participants. REGENXBIO also plans to present long-term ALTITUDE and wet AMD data at ASRS 2026 and expects pivotal ATMOSPHERE and ASCENT topline data in Q4 2026.
REGENXBIO (Nasdaq: RGNX) completed dosing in the confirmatory study of RGX-202 for Duchenne muscular dystrophy, marking completion of the registrational development program.
This supports initiating a BLA in Q3 2026 under the accelerated approval pathway, targeting potential FDA approval in 2H 2027, backed by safety data from 63 patients and pivotal efficacy data from 30 patients.
REGENXBIO (Nasdaq:RGNX) announced agreement with the FDA on the path to resubmit its BLA for NAVSUNLI™, a potential one-time gene therapy for MPS II (Hunter syndrome). No additional studies or new patients are required, and the FDA plans an expedited review.
A Type A meeting is expected in July, with BLA resubmission targeted for Q3 2026.
REGENXBIO (Nasdaq: RGNX) will participate in two upcoming investor events: the 2026 RBC Capital Markets Global Healthcare Conference fireside chat on May 20, 2026 at 9:30 a.m. ET in New York, and the Stifel 2026 Virtual Ophthalmology Forum fireside chat on May 26, 2026 at 9:30 a.m. ET.
Live webcasts and 30-day replays will be available in the Investors section of REGENXBIO's website.
REGENXBIO (Nasdaq:RGNX) reported Q1 2026 results and key clinical milestones. Positive topline Phase III AFFINITY DUCHENNE® data for RGX-202 met its primary endpoint with high statistical significance, supporting a potential accelerated approval in 2027.
Q1 2026 revenue was $6.4M versus $89.0M a year ago, with a net loss of $90.1M. Cash and securities totaled $150.5M, expected to fund operations into early 2027. A $100M milestone from AbbVie is expected upon first patient dosing in the NAAVIGATE DR study in Q2 2026.
REGENXBIO (Nasdaq: RGNX) reported positive topline results from the pivotal Phase III portion of the AFFINITY DUCHENNE study of gene therapy RGX-202 for Duchenne muscular dystrophy.
The primary endpoint was met with high statistical significance (p<0.0001); 93% of patients (n=30) achieved >10% microdystrophin expression at Week 12, with mean expression of 71.1%. RGX-202 showed a favorable safety profile and statistically significant correlation between microdystrophin expression and one-year functional improvement (NSAA, timed tests; n=9). REGENXBIO plans to pursue FDA accelerated approval and is preparing for a potential 2027 commercial launch.
REGENXBIO (Nasdaq: RGNX) will present multiple oral and poster abstracts at the ASGCT 2026 annual meeting in Boston, May 11–15, 2026.
Oral highlights include NAVXpress manufacturability advances and an encore of topline pivotal data for RGX-202 (Duchenne). Multiple posters cover NVG82, capsid engineering, reduced liver/DRG transduction, and single-nuclei transcriptomics.
REGENXBIO (Nasdaq: RGNX) will host a live webcast on May 14, 2026 at 8:00 a.m. ET to discuss topline results from the pivotal trial of RGX-202, its next-generation investigational gene therapy for Duchenne muscular dystrophy.
The company will also report first quarter 2026 financial results and operational highlights pre-market on May 14. The live webcast and an archived replay (available for ~30 days) are accessible via the investor section at regenxbio.com.
REGENXBIO (Nasdaq: RGNX) reported positive interim Phase I/II AFFINITY DUCHENNE data for RGX-202 (data cut Jan 5, 2026). Pivotal-dose participants (2x10^14 GC/kg, n=7) showed improved function vs external controls (NSAA +4.9 vs cTAP at 1 year; older 8+ = +5.2), stable cardiac MRI measures, consistent microdystrophin expression, and no SAEs or AESIs. Liver markers remained below concerning thresholds and new biomarker data showed 51.2% expression in one patient at Week 12. Topline pivotal data expected Q2 2026 with a planned pre-BLA meeting mid-2026.
REGENXBIO (Nasdaq: RGNX) reported Q4 and full-year 2025 results and operational highlights on March 5, 2026. Key clinical milestones include expected pivotal topline data for RGX-202 in early Q2 2026 and sura-vec wet AMD pivotal data in Q4 2026. Cash, cash equivalents and marketable securities were $240.9M at year-end, with runway into early 2027. 2025 revenues rose to $170.4M, R&D expenses increased to $228.3M, and net loss narrowed to $193.9M for the year. The FDA issued clinical holds on RGX-111 and RGX-121 and a CRL for RGX-121; the company plans to address regulator concerns.