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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) 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.
REGENXBIO (Nasdaq: RGNX) will present at the 44th Annual J.P. Morgan Healthcare Conference.
Date & time: Wednesday, January 14, 2026 at 10:30 a.m. PT. Location: Westin St. Francis, San Francisco, CA.
A live webcast will be available in the Investors section of the company's website and an archived replay will be posted for approximately 30 days after the presentation.
REGENXBIO (Nasdaq: RGNX) will participate in the Piper Sandler 37th Annual Healthcare Conference with a fireside chat on Tuesday, December 2, 2025 at 8:30 a.m. ET in New York, NY.
A live webcast will be available in the Investors section of the company's website and an archived replay will be accessible for approximately 30 days following the event.
REGENXBIO (Nasdaq: RGNX) reported Q3 2025 results and operational milestones on November 6, 2025. Key clinical timelines include RGX-202 (Duchenne) topline expected early Q2 2026 with a planned BLA submission mid-2026, and RGX-121 (MPS II) with an FDA PDUFA date of February 8, 2026. Enrollment completed for the RGX-202 pivotal trial (n=30) and for sura-vec wet AMD pivotal trials (>1,200 participants); sura-vec wet AMD topline expected Q4 2026. Financials: cash $302.0M (Sept 30, 2025), revenues $29.7M Q3, R&D $56.1M Q3, and net loss $61.9M Q3. The company expects cash to fund operations into early 2027. Commercial manufacturing batches for RGX-202 were produced and process qualification is near completion.
REGENXBIO (Nasdaq: RGNX) completed enrollment in the pivotal AFFINITY DUCHENNE trial for RGX-202 in October 2025 and has manufactured the first batches intended for commercial supply at its Rockville Manufacturing Innovation Center.
Key facts: pivotal enrollment = 30 participants; Phase I/II microdystrophin range 20%–122%; no SAEs or AESIs reported as of May 7, 2025; capacity = 2,500 doses/year; topline pivotal data expected in early Q2 2026 with a BLA submission targeted for mid-2026, and potential commercial launch in 2027.
REGENXBIO (Nasdaq: RGNX) will host a conference call on Thursday, November 6, 2025 at 8:00 a.m. ET to discuss third quarter financial results for the period ended September 30, 2025 and operational highlights.
Listeners can register for a live webcast via the company website, analysts may use a separate link to join the Q&A, and a replay will be available on the investor website approximately two hours after the call concludes. Participants are advised to join 15 minutes early.
REGENXBIO (Nasdaq: RGNX) announced participation in multiple investor conferences in October–November 2025. The company will join Chardan's 9th Annual Genetic Medicines Conference panels on Oct 21, 2025 at 2:00 PM ET and 2:45 PM ET in New York, a Guggenheim Securities fireside chat on Nov 10, 2025 at 9:00 AM ET in Boston, and a Stifel fireside chat on Nov 11, 2025 at 1:20 PM ET in New York. Live webcasts of select events will be available in the Investors section at www.regenxbio.com, with archived replays available for approximately 30 days after each event.