Welcome to our dedicated page for Regenxbio news (Ticker: RGNX), a resource for investors and traders seeking the latest updates and insights on Regenxbio stock.
REGENXBIO Inc. (Nasdaq: RGNX) generates frequent news as it advances a late-stage pipeline of adeno-associated virus (AAV) gene therapies for rare and retinal diseases. Company updates often focus on clinical trial progress for RGX-202 in Duchenne muscular dystrophy, clemidsogene lanparvovec (RGX-121) for MPS II, RGX-111 for MPS I, and surabgene lomparvovec (ABBV-RGX-314, sura-vec) for wet age-related macular degeneration and diabetic retinopathy.
News releases highlight functional and biomarker data from ongoing studies, such as long-term North Star Ambulatory Assessment results in the AFFINITY DUCHENNE® trial of RGX-202 and analyses of cerebrospinal fluid biomarkers in MPS II. REGENXBIO also reports on pivotal trial enrollment milestones, including completion of enrollment in the ATMOSPHERE® and ASCENT® pivotal trials for subretinal sura-vec in wet AMD and progress in the ALTITUDE® trial for diabetic retinopathy using suprachoroidal delivery.
Investors following RGNX news can also expect regular disclosures on regulatory interactions and key dates, such as the FDA’s extension of the Prescription Drug User Fee Act (PDUFA) action date for the RGX-121 Biologics License Application and anticipated timelines for pivotal data readouts and BLA submissions. The company’s collaboration and license activities with partners like AbbVie and Nippon Shinyaku, including amendments to development and milestone structures, are typically announced through press releases and corresponding Form 8-K filings.
In addition, REGENXBIO issues announcements about participation in major healthcare and investor conferences, where management presents clinical updates and strategic priorities. For a fuller picture of RGNX stock, readers can use this news feed to track clinical data presentations, manufacturing and commercial readiness updates, financial results press releases and other material events described in the company’s communications.
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.
REGENXBIO (NASDAQ:RGNX) announced the advancement of surabgene lomparvovec (sura-vec) into a pivotal Phase IIb/III clinical trial for diabetic retinopathy (DR) using suprachoroidal delivery. The decision follows positive two-year data from the Phase II ALTITUDE® trial, demonstrating durable safety and efficacy with a single injection.
Under an amended collaboration with AbbVie, REGENXBIO will receive $100 million upon first subject dosing in the Phase IIb/III trial and another $100 million for the first subject in a second Phase III trial. The Phase II ALTITUDE trial showed favorable safety results, with no drug-related serious adverse events or intraocular inflammation observed through two years at dose level 3.
REGENXBIO (Nasdaq: RGNX) has scheduled a conference call for Thursday, August 7, 2025, at 8:00 a.m. ET to discuss its second quarter 2025 financial results and recent operational developments. The company will host a webcast that can be accessed through a registration link.
Analysts can participate in the Q&A session by dialing (646) 307-1963 (domestic) or (800) 715-9871 (international) with passcode 9571992. A replay will be available on REGENXBIO's investor website approximately two hours after the call concludes. Participants are encouraged to join 15 minutes before the start time.
REGENXBIO (NASDAQ:RGNX) has published significant preclinical results for RGX-202, their novel gene therapy for Duchenne Muscular Dystrophy. The research, published in Molecular Therapy Methods and Clinical Development, demonstrates that their unique microdystrophin construct containing the C-terminal (CT) domain shows superior performance compared to versions without it.
The studies in mdx mice revealed that the CT domain-containing construct achieved higher protein levels, increased muscle force, and improved resistance to damage. RGX-202 is currently the only microdystrophin gene therapy candidate that includes the CT domain, making it closest to natural dystrophin. These findings support positive interim results from the ongoing Phase I/II AFFINITY DUCHENNE® trial, with REGENXBIO planning a BLA submission in mid-2026 using the accelerated approval pathway.
REGENXBIO (NASDAQ: RGNX) has secured a strategic royalty monetization agreement with Healthcare Royalty (HCRx) worth up to $250 million. The company received $150 million upfront, extending its cash runway into early 2027. The non-dilutive agreement includes rights to royalties from ZOLGENSMA sales and milestone payments from various partnerships.
Additional funding includes $50 million tied to ZOLGENSMA sales milestones (by April 2027) and another $50 million upon mutual agreement. HCRx will receive quarterly interest payments from royalty revenue and warrants to purchase 268,096 shares at $14.92. REGENXBIO retains rights to other funding sources, including a potential Priority Review Voucher for RGX-121 and milestones from AbbVie and Nippon Shinyaku.
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