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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) 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.
REGENXBIO (Nasdaq: RGNX) will present interim Phase II ALTITUDE trial data on suprachoroidal delivery of surabgene lomparvovec (sura-vec, ABBV-RGX-314) for diabetic retinopathy at the American Academy of Ophthalmology 2025 Annual Meeting. The presentation reports first-time 2-year results in non-proliferative diabetic retinopathy and is delivered by Charles C. Wykoff, M.D., PhD during Section IX: Late breaking developments, part I.
Presentation date/time: October 17, 2025 at 4:46 p.m. ET. Sura-vec is an investigational one-time gene therapy developed in collaboration with AbbVie for wet AMD and DR.
REGENXBIO (Nasdaq: RGNX) announced completion of enrollment in the ATMOSPHERE and ASCENT pivotal trials of subretinal surabgene lomparvovec for wet age-related macular degeneration (wet AMD).
Combined enrollment exceeded 1,200 participants across >200 sites worldwide, making this the largest global gene therapy program reported for an ocular indication. ATMOSPHERE (U.S.) compares sura-vec to ranibizumab; ASCENT (U.S. + 13 countries) compares sura-vec to aflibercept. Primary endpoints are non-inferiority in BCVA at 54 weeks and one year. Company cites durable effects up to 4 years in long-term follow-up and no drug-related serious adverse events in a 60-patient Phase II study. Topline pivotal data expected Q4 2026.
REGENXBIO (Nasdaq: RGNX) announced that Chief Medical Officer Steve Pakola, M.D., will present new analysis of 12-month functional data from the Phase I/II trial of RGX-202 at the International Congress of the World Muscle Society in Vienna, Austria, October 7-11, 2025.
The presentation will highlight that RGX-202 demonstrated a favorable safety profile with no serious adverse events in the Phase I/II study. Notably, pivotal dose participants showed superior performance compared to baseline-matched external natural history controls across all functional measures, suggesting RGX-202's potential as a best-in-class gene therapy for Duchenne muscular dystrophy.
REGENXBIO (NASDAQ:RGNX) has announced positive 12-month pivotal data from its Phase I/II/III CAMPSIITE® trial of RGX-121 for treating MPS II (Hunter syndrome). The trial demonstrated an 82% median reduction in CSF levels of HS D2S6, a key biomarker of MPS II brain disease, sustained through one year.
The study met its primary endpoint with statistical significance (p < 0.0001), showing strong correlation between biomarker levels and neurodevelopmental improvements. Patients demonstrated neurodevelopmental skill acquisition or stability across all sub-scales of the BSID-III assessment at one year.
The FDA completed pre-license and bioresearch monitoring inspections with no observations, and RGX-121 has been well-tolerated in all 26 patients. A decision on the Biologics License Application is expected by February 8, 2026. If approved, RGX-121 would become the first one-time therapy targeting the genetic cause of Hunter syndrome.
REGENXBIO (Nasdaq: RGNX) has announced its participation in four major upcoming investor conferences in September 2025. The company will attend the Wells Fargo Healthcare Conference for one-on-one meetings in Boston on September 4, followed by three fireside chat presentations in New York: the Morgan Stanley Global Healthcare Conference on September 8, and both the Baird Global Healthcare Conference and H.C. Wainwright Global Investment Conference on September 9.
Select fireside chat presentations will be available via webcast through REGENXBIO's investor relations website, with recordings accessible for approximately 30 days after each event.
REGENXBIO (Nasdaq: RGNX) announced that the FDA has extended its review timeline for RGX-121, a potential treatment for Hunter syndrome (MPS II). The PDUFA date has been pushed from November 9, 2025 to February 8, 2026 following the company's submission of additional 12-month clinical data for all 13 patients in the pivotal study.
The FDA's extension comes after completing a pre-license inspection with no observations, and notably, no safety concerns have been raised during the BLA review. RGX-121 holds multiple FDA designations including Orphan Drug, Rare Pediatric Disease, Fast Track, and RMAT. If approved, it would become the first one-time therapy targeting the genetic cause of Hunter syndrome.
REGENXBIO (NASDAQ:RGNX) reported Q2 2025 financial results and significant progress across its gene therapy pipeline. The company's cash position strengthened to $363.6 million, expected to fund operations into early 2027. Key developments include accelerated enrollment in the RGX-202 Duchenne muscular dystrophy pivotal trial, expected to complete by October 2025, with BLA submission planned for mid-2026.
The company reported Q2 2025 revenues of $21.4 million and a net loss of $70.9 million ($1.38 per share). Notable achievements include successful FDA inspections for clemidsogene lanparvovec (RGX-121) for MPS II, with a PDUFA date of November 9, 2025, and advancement of surabgene lomparvovec (sura-vec) for retinal diseases. The company secured a $250 million royalty agreement with Healthcare Royalty and amended its AbbVie collaboration with potential milestone payments of $200 million.