Regenxbio

RGNX

Market cap $589M

ROCKVILLE, Md. , Oct. 15, 2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced it will participate in the following investor conferences: Chardan's 9th Annual Genetic Medicines Conference Panel "Battleground States for Muscular Dystrophies": Tuesday, October 21 at 2:00 p.m.

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.