REGENXBIO Inc. (RGNX)

ROCKVILLE, Md. , Oct. 9, 2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that it will present interim data from the Phase II ALTITUDE® trial evaluating suprachoroidal delivery of surabgene lomparvovec (ABBV-RGX-314, sura-vec) for the treatment of diabetic retinopathy (DR) at the American Academy of Ophthalmology 2025 Annual Meeting.

Over 1,200 participants enrolled in ATMOSPHERE® and ASCENT® pivotal trials, representing largest global gene therapy program ever conducted Subretinal surabgene lomparvovec on track to be first gene therapy for wet AMD Topline pivotal data expected in Q4 2026 ROCKVILLE, Md. , Oct. 6, 2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced the completion of enrollment in the ATMOSPHERE® and ASCENT® pivotal studies evaluating surabgene lomparvovec (sura-vec, ABBV-RGX-314) in wet age-related macular degeneration (wet AMD) using subretinal delivery.

ROCKVILLE, Md. , Sept. 29, 2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced Chief Medical Officer, Steve Pakola, M.D.

Regenxbio's RGX-121 gene therapy for Hunter syndrome is the main catalyst, with FDA approval targeted for February 2026. Recent clinical data shows RGX-121 can address neurological decline, a major unmet need, setting it apart from current treatments. Despite a small patient population, premium pricing and first-mover advantage could drive substantial revenue if RGX-121 is approved.

REGENXBIO Inc. (NASDAQ:RGNX ) Morgan Stanley 23rd Annual Global Healthcare Conference September 8, 2025 11:30 AM EDT Company Participants Curran Simpson - President, CEO & Director Steve Pakola - Executive VP & Chief Medical Officer Mitchell Chan - Executive VP & CFO Presentation Unknown Analyst Welcome, everyone, to this session of the Morgan Stanley Global Healthcare Conference. We're excited to have team from REGENX here.

Regenxbio's RGX-121 for Hunter syndrome saw its FDA review delayed to February 8, 2026, but strong 12-month surrogate endpoint data supports its potential as a gene therapy. Late-stage Sura-vec trials for Wet-AMD, in partnership with AbbVie, remain on track for 2026 topline results, bolstering the pipeline's value. Recent partnerships and royalty deals have strengthened Regenxbio's cash position, extending its runway into early 2027 despite ongoing R&D expenses.

12-month pivotal data further demonstrate the ability of one-time RGX-121 treatment to improve outcomes for patients with MPS II >80% reduction in CSF levels of HS D2S6, key biomarker of MPS II brain disease, sustained through 1 year Pivotal patients demonstrate continued skill acquisition or stability, stratified by baseline function, through 1 year Primary endpoint of CSF HS D2S6 reduction at week 16 met; strong correlation to neurodevelopmental outcomes at 1 year, supporting HS D2S6 as surrogate biomarker reasonably likely to predict clinical benefit RGX-121 would be the first and only potential one-time, commercially-available therapy designed to directly address the underlying genetic cause of Hunter syndrome, if approved ROCKVILLE, Md. , Sept. 5, 2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced new, positive data from the Phase I/II/III CAMPSIITE® trial of clemidsogene lanparvovec (RGX-121) for the treatment of patients with Mucopolysaccharidosis Type II (MPS II), also known as Hunter syndrome, at the International Congress of Inborn Errors of Metabolism (ICIEM) 2025.

ROCKVILLE, Md. , Aug. 28, 2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced it will participate in the following investor conferences: 2025 Wells Fargo Healthcare Conference 1x1 Investor Meetings: Thursday, September 4 Location:  Boston, MA Morgan Stanley 23rd Annual Global Healthcare Conference Fireside Chat:  Monday, September 8, 2025 at 11:30 a.m.

The U.S. Food and Drug Administration (FDA) on Monday extended its review timeline for REGENXBIO Inc.'s RGNX Biologics License Application (BLA) for clemidsogene lanparvovec (RGX-121) for Mucopolysaccharidosis II (MPS II), also known as Hunter syndrome.

RGX-121 would be the first and only potential one-time commercially-available therapy designed to directly address the underlying genetic cause of Hunter syndrome, if approved Commercial launch plans remain on track  REGENXBIO plans to present updated pivotal data during the ICIEM meeting in September 2025 ROCKVILLE, Md. , Aug. 18, 2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that the U.S. Food and Drug Administration (FDA) extended its review timeline of the Biologics License Application (BLA) for clemidsogene lanparvovec (RGX-121) for the treatment of Mucopolysaccharidosis II (MPS II), also known as Hunter syndrome.

REGENXBIO Inc. (NASDAQ:RGNX ) Q2 2025 Earnings Call August 7, 2025 8:00 AM ET Company Participants Curran M. Simpson - President, CEO & Director Mitchell Chan - Executive VP & CFO Patrick J.

Regenxbio (RGNX) came out with a quarterly loss of $1.38 per share versus the Zacks Consensus Estimate of a loss of $1.13. This compares to a loss of $1.05 per share a year ago.

RGX-202 in Duchenne muscular dystrophy on track for topline results 1H 2026 and BLA submission mid-2026 Pivotal trial enrollment accelerated, expected to complete in October 2025 ahead of previous guidance Clemidsogene lanparvovec (RGX-121) on track to be first gene therapy and one-time treatment for MPS II; FDA inspections completed successfully Pre-license inspection (PLI) of in-house manufacturing facility, quality systems and processes completed with no observations Bioresearch monitoring information (BIMO) inspection of laboratory and clinical data practices completed with no observations Surabgene lomparvovec (sura-vec, ABBV-RGX-314) on track to be first gene therapy in chronic retinal disease Pivotal data evaluating the safety and efficacy of the subretinal delivery of sura-vec in patients with wet AMD are expected in 2026 Sura-vec using suprachoroidal delivery for diabetic retinopathy advancing to global pivotal program, supported by positive Phase II trial data Conference call today at 8:00 a.m. ET ROCKVILLE, Md.

A pivotal Phase IIb/III clinical trial using suprachoroidal delivery for treatment of diabetic retinopathy will be initiated New Phase II ALTITUDE ® trial data demonstrate a durable safety and efficacy profile observed through two years with a single, in-office injection of surabgene lomparvovec in subjects with non-proliferative diabetic retinopathy REGENXBIO to receive $100 million upon first subject dosed in the Phase IIb/III clinical trial and an additional $100 million upon first subject dosed in a second Phase III clinical trial ROCKVILLE, Md. , Aug. 7, 2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced it will initiate a pivotal Phase IIb/III clinical trial for investigational surabgene lomparvovec (sura-vec, ABBV-RGX-314) in diabetic retinopathy (DR) using suprachoroidal delivery and a corresponding amendment to its eyecare collaboration with AbbVie.

ROCKVILLE, Md. , July 31, 2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that it will host a conference call on Thursday, August 7, at 8:00 a.m.

Construct including CT domain demonstrated higher levels of microdystrophin protein, increased muscle force, and improved resistance to damage in mice lacking dystrophin REGENXBIO's next-generation investigational gene therapy, RGX-202, is the only microdystrophin construct that includes the CT domain Findings support the positive functional data seen in Phase I/II AFFINITY DUCHENNE® trial of RGX-202 ROCKVILLE, Md. , July 10, 2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced the publication of preclinical results comparing a microdystrophin gene therapy construct that included the C-terminal (CT) domain to a microdystrophin construct without the CT domain.