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) 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.
REGENXBIO (Nasdaq: RGNX) announced presentations on its investigational Duchenne gene therapy RGX-202 at the 2026 Muscular Dystrophy Association Clinical & Scientific Conference in Orlando, March 8-11, 2026.
Events include an interim Phase I/II podium presentation on March 11, a preclinical poster, and a company-hosted symposium on March 9 at 12 p.m. ET. Presentation materials will be posted in the company's Publications section.
REGENXBIO (Nasdaq: RGNX) will participate in three investor conferences in March 2026 with multiple fireside chats and webcasts available.
Events include Leerink Partners Global Healthcare on March 9, 2026 at 9:20 AM ET and Barclays Global Healthcare on March 10, 2026 at 1:30 PM ET in Miami, and RBC Virtual Ophthalmology on March 24, 2026 at 8:45 AM ET. Live webcasts and ~30-day archived replays will be available on the company investor website.
REGENXBIO (Nasdaq: RGNX) will host a conference call on Thursday, March 5, 2026 at 8:00 a.m. ET to discuss fourth-quarter and full-year results for the period ended December 31, 2025, and operational highlights.
Investors can register for the webcast, analysts can request Q&A access, and a replay will be posted about two hours after the call.
REGENXBIO (Nasdaq: RGNX) announced the FDA issued a Complete Response Letter (CRL) on February 7, 2026 for its BLA for RGX-121 (clemidsogene lanparvovec) for Mucopolysaccharidosis II (MPS II). The CRL cited concerns about defining the neuronopathic population, comparability of the external natural-history control, and the CSF HS D2S6 surrogate endpoint.
REGENXBIO said the FDA agreed to the study protocol in principle, the company will request a Type A meeting, pursue paths outlined in the CRL, and aims to resubmit the BLA after providing additional analyses and longer-term clinical data.
Summary not available.
REGENXBIO (Nasdaq: RGNX) said the FDA placed a clinical hold on investigational gene therapies RGX-111 (MPS I) and RGX-121 (MPS II) after a single case of an intraventricular CNS tumor was found in a participant treated with RGX-111.
Preliminary analysis detected an AAV vector genome integration event linked to PLAG1 overexpression; causality is undetermined and the investigation is ongoing. The affected participant is asymptomatic with developmental gains. No neoplasm reported in nine other RGX-111 patients or 32 RGX-121 patients. REGENXBIO awaits the full FDA hold letter.
REGENXBIO (Nasdaq: RGNX) reported positive long-term Phase I/II RGX-202 functional data for Duchenne, with treated patients improving an average of 7.4 NSAA points versus cTAP at 18 months (n=4) and 6.6 points at 12 months. The company expects pivotal topline data in early Q2 2026 and plans a mid-2026 BLA filing under accelerated approval while continuing confirmatory enrollment. REGENXBIO also highlighted an FDA PDUFA date of Feb 8, 2026 for RGX-121, a $100M AbbVie milestone tied to sura-vec first patient dosing, and in-house manufacturing readiness from its Rockville facility.