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 ...

REGENXBIO Inc. (NASDAQ:RGNX) Q1 2024 Earnings Conference Call May 8, 2024 4:30 PM ET Company Participants Patrick Christmas – Chief Legal Officer Ken Mills – Chief Executive Officer Curran Simpson – Chief Operating Officer Vit Vasista – Chief Financial Officer Conference Call Participants Gena Wang – Barclays Annabel Samimy – Stifel Brian Skorney – Baird Luca Issi – RBC Capital Daniil Gataulin – Chardan Operator Good day and thank you for standing by. Welcome to REGENXBIO's First Quarter 2024 Earnings Confe ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2024 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from ___ to ___ Commission File Number 001-37553 REGENXBIO Inc. (Exact Name of Registrant as Specified in its Charter) Delaware 47-1851754 (State ...

EXHIBIT 99.1 REGENXBIO Reports First 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) • Dose level 2 selected as pivotal dose for RGX-202 treatment of Duchenne o New positive interim efficacy and safety data announced for second boy at DL2 o AFFINITY DUCHENNE® trial expansion phase is underway with third and fourth ...

REGENXBIO Inc. (NASDAQ:RGNX) Q4 2023 Earnings Conference Call February 27, 2024 4:30 PM ET Company Participants Patrick Christmas – Chief Legal Officer Ken Mills – Chief Executive Officer Steve Pakola – Chief Medical Officer Vit Vasista – Chief Financial Officer Conference Call Participants Gena Wang – Barclays Gospel Enyindah-Asonye – Morgan Stanley Alec Stranahan – Bank of America Annabel Samimy – Stifel Lisa Walter – RBC Capital Markets Mani Foroohar – Leerink Partners Operator Good day, and thank you fo ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-K (Mark One) ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2023 or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission file number: 001-37553 Indicate by check mark if the registrant is not required to file reports pursuant to Section 13 or Section 1 ...

EXHIBIT 99.1 • ABBV-RGX-314 Subretinal Delivery for the Treatment of Wet AMD o Enrollment is on track in ATMOSPHERE ® and ASCENT™ pivotal trials and these trials are expected to support global regulatory submissions with the U.S. Food and Drug Administration and the European Medicines Agency in late 2025 through the first half of 2026. • ABBV-RGX-314 Suprachoroidal Delivery for Treatment of Wet AMD o REGENXBIO expects to share new program and data updates for the Phase II AAVIATE trial in mid-2024. o In Jan ...