EXHIBIT 99.1 REGENXBIO Reports Second Quarter 2024 Financial Results and Recent Operational Highlights • Company remains on track for its first BLA filing in 2024 and is accelerating progress toward pivotal trial initiation for Duchenne (H2 2024) and diabetic retinopathy (H1 2025) o New, positive data from Phase II AFFINITY DUCHENNE ®trial of RGX-202 demonstrates consistent high expression of microdystrophin across treated patients in all age groups o Successful End-of-Phase II meeting with FDA for RGX-202 ...

Robust microdystrophin expression observed in new data from pivotal dose Patients aged 5.8 and 8.5 years at dosing had expression levels at 77.2% and 46.5% of control, respectively Consistent high expression of microdystrophin across treated patients in all age groups continues to support plans for accelerated approval Expects to initiate pivotal trial in Q4 2024 ROCKVILLE, Md., Aug. 1, 2024 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today reported new, positive interim safety and efficacy data in the Ph ...

REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. Since its founding in 2009, REGENXBIO has pioneered the development of AAV Therapeutics, an innovative class of gene therapy medicines. REGENXBIO is advancing a pipeline of AAV Therapeutics for retinal and rare diseases, including ABBV-RGX-314 for the treatment of wet AMD and diabetic retinopathy, being developed in collaboration with AbbVie, RGX-202 for the treatment of Duche ...

Regenxbio (RGNX) shares rallied 18.3% in the last trading session to close at $12.71. This move can be attributable to notable volume with a higher number of shares being traded than in a typical session. This compares to the stock's 18.3% loss over the past four weeks. For Regenxbio, the consensus EPS estimate for the quarter has remained unchanged over the last 30 days. And a stock's price usually doesn't keep moving higher in the absence of any trend in earnings estimate revisions. So, make sure to keep ...

Regenxbio (RGNX) shares ended the last trading session 6.4% higher at $12.79. The jump came on an impressive volume with a higher-than-average number of shares changing hands in the session. This compares to the stock's 19.4% loss over the past four weeks. Earlier this month, the company completed a successful pre-biologics license application (BLA) meeting with the FDA for its pipeline candidate, RGX-121, which is being developed for the treatment of Mucopolysaccharidosis Type II (MPS II). The company expe ...

"We believe RGX-202 has unique, differentiating features that support its potential to be a best-in-class product and we are pleased to expand its clinical development to reach a wider range of boys with Duchenne in need of treatment options," said Curran Simpson, Chief Operating Officer of REGENXBIO and President and CEOelect. "Supported by the strong safety profile and positive microdystrophin data demonstrated in the AFFINITY DUCHENNE trial, today's news marks significant steps in rapidly accelerating RG ...

Aligned with FDA on content of BLA and plans for submission: Submission of a rolling BLA using the accelerated approval pathway expected to start in Q3 2024 Confirmatory trial expected to begin in H2 2025 FDA confirmed RGX-121 commercial bulk drug is comparable to clinical material Positive biomarker, neurocognitive and systemic data will be part of BLA submission The FDA continues to be aligned with REGENXBIO's plan to use cerebrospinal fluid (CSF) levels of heparan sulfate (HS) D2S6, a key biomarker of br ...

Curran Simpson, Chief Operating Officer, Appointed as President and CEO Co-Founder Kenneth T. Mills to Step Down as President and CEO; Named Chairman of the Board Mr. Simpson is a seasoned biopharmaceuticals leader with over 35 years of industry experience. As the Chief Operating Officer (COO) at REGENXBIO since January 2023, he has been directly responsible for key business functions including Research & Clinical Development, Corporate Strategy, Manufacturing & Quality, Regulatory, and Commercial Operation ...

REGENXBIO Inc. (NASDAQ:RGNX) is a biotech company developing AAV therapies for common and rare diseases. Its proprietary NAV platform creates one-dose AAV treatments that offer lasting therapeutic effects by targeting the root cause of diseases. AAV vectors deliver gene therapies to specific cells, maximizing efficacy and avoiding adverse immune responses. RGNX's pipeline includes promising candidates such as ABBV-RGX-314 for age-related AMD and DR, RGX-202 for DMD, and RGX-121 for Hunter syndrome. RGXN's s ...

ROCKVILLE, Md., June 5, 2024 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced it will participate in the Goldman Sachs 45th Annual Global Healthcare Conference on Wednesday, June 12, 2024. Goldman Sachs 45th Annual Global Healthcare Conference Fireside Chat: Wednesday, June 12, 2024 at 8:00 a.m. EDT Location: Miami, FL A live webcast of the fireside chat can be accessed in the Investors section of REGENXBIO's website at www.regenxbio.com. An archived replay of the webcast will be available for ...