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 (RGNX) is a clinical-stage biotechnology leader advancing novel AAV gene therapies through its proprietary NAV® technology platform. This page provides investors and stakeholders with centralized access to the company’s latest press releases, clinical trial updates, and strategic developments.
Track critical updates across Regenxbio’s pipeline, including progress on RGX-202 for Duchenne muscular dystrophy, RGX-121 for Hunter syndrome, and ABBV-RGX-314 for retinal diseases. Stay informed about regulatory milestones, manufacturing advancements, and partnership announcements with entities like AbbVie.
All content is sourced directly from Regenxbio’s official communications, ensuring accuracy and timeliness. Bookmark this page for streamlined access to essential updates on one-time gene therapies targeting rare genetic disorders.
REGENXBIO (NASDAQ: RGNX) has reported positive interim data from the AFFINITY DUCHENNE® trial of RGX-202, their investigational gene therapy for Duchenne muscular dystrophy. Key highlights include robust microdystrophin expression levels, with a 3-year-old patient showing 122.3% expression compared to control, and a 7-year-old patient showing 31.5%.
The therapy demonstrates the highest reported vector genome copies (4.9-55.4) measured by qPCR across approved or investigational gene therapies. Safety data as of February 21, 2025, shows RGX-202 was well-tolerated with no serious adverse events. Common side effects included nausea, vomiting, and fatigue, which all resolved.
The Phase III portion of the trial is currently enrolling ambulatory patients aged 1 and above, with BLA submission targeted for mid-2026. Previously reported data from November 2024 showed functional improvements in all five initial participants, exceeding external natural history controls. Additional interim functional data is expected in the first half of 2025.
REGENXBIO (RGNX) has reported its Q4 and full-year 2024 financial results, highlighting significant progress in its gene therapy pipeline. The company submitted a Biologics Licensing Application (BLA) for clemidsogene lanparvovec (RGX-121) for MPS II treatment, with potential FDA approval expected in H2 2025.
Key financial metrics include: Cash position of $244.9 million as of December 31, 2024, full-year revenues of $83.3 million, and a net loss of $227.1 million ($4.59 per share). The company secured a strategic partnership with Nippon Shinyaku worth up to $810 million, including $110 million upfront.
Pipeline updates include: RGX-202 pivotal trial for Duchenne Muscular Dystrophy is 50% enrolled with completion expected in 2025 and BLA filing planned for mid-2026. The AbbVie-partnered retinal program continues advancing with pivotal data for surabgene lomparvovec in wet AMD expected in 2026.
REGENXBIO (RGNX) has announced upcoming presentations of new interim biomarker data from their Phase I/II AFFINITY DUCHENNE® trial of RGX-202, a treatment for Duchenne muscular dystrophy. The data will be presented at the 2025 MDA Clinical & Scientific Conference in Dallas from March 16-19, 2025.
Two presentations are scheduled: An interim clinical data presentation by Dr. Carolina Tesi Rocha from Stanford School of Medicine on March 19, and a pre-clinical research presentation on microdystrophin by Dr. Steven Foltz, Senior Scientist at REGENXBIO. Both presentations will be made available on REGENXBIO's website.
REGENXBIO (RGNX) has finalized its strategic partnership with Nippon Shinyaku for the development and commercialization of two gene therapy treatments: RGX-121 (clemidsogene lanparvovec) for Mucopolysaccharidosis II (Hunter syndrome) and RGX-111 for Mucopolysaccharidosis I (Hurler syndrome) in the United States and Asia.
The collaboration aims to advance these potential treatments for rare genetic disorders, with RGX-121 positioned to potentially become the first gene therapy for MPS II, targeting FDA approval by late 2025.
REGENXBIO (RGNX) has scheduled a conference call for Thursday, March 13, at 4:30 p.m. ET to present its fourth quarter and full-year 2024 financial results along with recent operational updates. The presentation will be accessible through a webcast, with a dedicated link for analysts who wish to participate in the Q&A session. Participants are advised to join 15 minutes before the start time, and a replay will be made available on the company's investor website approximately two hours after the call concludes.
REGENXBIO (Nasdaq: RGNX) has announced its participation in five major investor conferences during March and April 2025. The company will engage in fireside chat presentations at:
- TD Cowen's 45th Annual Health Care Conference in Boston (March 5, 1:10pm ET)
- Leerink Partners Global Healthcare Conference in Miami (March 11, 9:20am ET)
- Barclays 27th Annual Global Healthcare Conference in Miami (March 12, 3:30pm ET)
- UBS Virtual CNS Day (March 17, 10:00am ET)
- RBC Inaugural Virtual Ophthalmology Conference (April 3, 8:15am ET)
Select fireside chats will be available via live webcast through REGENXBIO's website, with recordings accessible for approximately 30 days after each presentation.
REGENXBIO (RGNX) has announced upcoming presentations of data from its RGX-121 (clemidsogene lanparvovec) program at the 21st Annual WORLDSymposium™ 2025 in San Diego, CA, scheduled for February 3-7, 2025. The presentations will feature topline results from the pivotal phase of the Phase I/II/III CAMPSIITE® trial for the treatment of mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome.
Two presentations are scheduled: an audiology assessment of CAMPSIITE® study participants on February 5, and an interim clinical study update on February 6. The presentations will be delivered by Nidal Boulos, Ph.D., Director of Clinical Science at REGENXBIO, and Paul Harmatz, M.D., from UCSF Benioff Children's Hospital, respectively.
REGENXBIO and Nippon Shinyaku have announced a strategic partnership for developing and commercializing RGX-121 and RGX-111, gene therapies for MPS II (Hunter syndrome) and MPS I (Hurler syndrome) respectively. The deal includes a $110 million upfront payment to REGENXBIO, with potential milestone payments of up to $700 million ($40M in development/regulatory milestones and $660M in sales milestones).
REGENXBIO will receive double-digit royalties on net sales in the U.S. and Asia, lead manufacturing operations, and retain rights to the RGX-121 Priority Review Voucher. Nippon Shinyaku will lead commercialization efforts in these territories. RGX-121's FDA approval is anticipated by late 2025, with a rolling BLA submission currently underway. The transaction is expected to close by Q1 2025.
AbbVie (ABBV) and REGENXBIO (RGNX) have announced key updates for their ABBV-RGX-314 clinical program. The companies expect pivotal data on the safety and efficacy of subretinal delivery for wet age-related macular degeneration (wet AMD) patients in 2026 through the ATMOSPHERE® and ASCENT™ trials.
Additionally, both companies will plan a Phase 3 clinical program for diabetic retinopathy (DR) treatment using suprachoroidal delivery via the SCS Microinjector®. This gene therapy aims to address the treatment burden of chronic, frequent dosing in retinal diseases, which are among the leading causes of blindness. ABBV-RGX-314 is being developed as the first potential one-time gene therapy for both wet AMD and DR patients.
REGENXBIO has initiated the pivotal phase of AFFINITY DUCHENNE® trial for RGX-202, a gene therapy for Duchenne muscular dystrophy, with FDA alignment on accelerated approval pathway and expected BLA in 2026. The trial reports positive functional data across both dose levels, with improvements in North Star Ambulatory Assessment scores and timed function tests. New biomarker data shows consistent robust expression of RGX-202 microdystrophin in muscle tissue, with the highest reported levels among gene therapies. The pivotal trial will evaluate approximately 30 ambulatory patients aged 1 and older, with no serious adverse events reported to date.
