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. (Nasdaq: RGNX) generates frequent news as it advances a late-stage pipeline of adeno-associated virus (AAV) gene therapies for rare and retinal diseases. Company updates often focus on clinical trial progress for RGX-202 in Duchenne muscular dystrophy, clemidsogene lanparvovec (RGX-121) for MPS II, RGX-111 for MPS I, and surabgene lomparvovec (ABBV-RGX-314, sura-vec) for wet age-related macular degeneration and diabetic retinopathy.
News releases highlight functional and biomarker data from ongoing studies, such as long-term North Star Ambulatory Assessment results in the AFFINITY DUCHENNE® trial of RGX-202 and analyses of cerebrospinal fluid biomarkers in MPS II. REGENXBIO also reports on pivotal trial enrollment milestones, including completion of enrollment in the ATMOSPHERE® and ASCENT® pivotal trials for subretinal sura-vec in wet AMD and progress in the ALTITUDE® trial for diabetic retinopathy using suprachoroidal delivery.
Investors following RGNX news can also expect regular disclosures on regulatory interactions and key dates, such as the FDA’s extension of the Prescription Drug User Fee Act (PDUFA) action date for the RGX-121 Biologics License Application and anticipated timelines for pivotal data readouts and BLA submissions. The company’s collaboration and license activities with partners like AbbVie and Nippon Shinyaku, including amendments to development and milestone structures, are typically announced through press releases and corresponding Form 8-K filings.
In addition, REGENXBIO issues announcements about participation in major healthcare and investor conferences, where management presents clinical updates and strategic priorities. For a fuller picture of RGNX stock, readers can use this news feed to track clinical data presentations, manufacturing and commercial readiness updates, financial results press releases and other material events described in the company’s communications.
REGENXBIO Inc. (Nasdaq: RGNX) announced that it will present four oral and nine poster presentations at the American Society of Gene and Cell Therapy's 24th Annual Meeting, scheduled for May 11-14, 2021. Oral presentations feature topics such as AAV tissue transduction and the RGX-121 gene therapy for mucopolysaccharidosis type II. The poster presentations cover a range of subjects including AAV vector evaluation and gene therapy methodologies. Detailed information about the presentations can be found on the ASGCT website.
REGENXBIO Inc. (Nasdaq: RGNX) announced that Curran M. Simpson, Chief Operations and Technology Officer, will present at the Chardan 5th Annual Genetic Medicines Manufacturing Summit on April 27, 2021, at 3:15 p.m. ET. The event will be held virtually. A webcast of the presentation will be available in the Investors section of REGENXBIO's website and can be accessed at www.regenxbio.com. An archived replay will be accessible for 30 days post-event.
REGENXBIO is focused on advancing gene therapy using its NAV® Technology Platform.
REGENXBIO has announced the dosing of the first patient in Cohort 3 of its Phase I/II trial for RGX-121, a one-time gene therapy targeting MPS II (Hunter Syndrome). This trial, involving a total of nine patients across the U.S. and Brazil, aims to assess safety and efficacy. Previous cohorts indicated positive interim results with reductions in CNS biomarkers and neurocognitive development. RGX-121 leverages the AAV9 vector for gene delivery, with potential systemic benefits. The trial's primary endpoint is establishing safety while evaluating enzyme activity and neurocognitive outcomes.
REGENXBIO reported Q4 and full-year 2020 financial results with revenues of $154.6 million, up from $35.2 million in 2019, primarily due to Zolgensma royalties. The company holds $523 million in cash and equivalents, enhanced by a recent $230 million stock offering. Key developments include active patient enrollment in pivotal trials for RGX-314 targeting wet AMD, with a BLA filing anticipated in 2024. Positive interim data from RGX-121's Phase I/II trial showcases reduced CNS biomarkers. Research on RGX-202 for Duchenne Muscular Dystrophy and other therapies continues with IND applications expected soon.
REGENXBIO Inc. (Nasdaq: RGNX) will participate in several upcoming virtual investor conferences in March 2021. The events include:
- Raymond James 42nd Annual Institutional Investors Conference: March 3, 2021, at 10:50 a.m. ET
- Barclays Global Healthcare Conference: March 9, 2021, at 4:10 p.m. ET
- Morgan Stanley Virtual Healthcare Corporate Access Day: March 16, 2021
Live webcasts for the Raymond James and Barclays events will be available on REGENXBIO's website, with archived versions accessible for 30 days post-event.
REGENXBIO Inc. (RGNX) announced a conference call scheduled for March 1, 2021, at 4:30 p.m. ET, to discuss its financial results for the fourth quarter and full year 2020, along with operational highlights. The call can be accessed via phone or through a webcast on the company's website. REGENXBIO is recognized for its innovative gene therapy solutions using its NAV Technology Platform, which includes a range of adeno-associated virus vectors aimed at therapeutic advancements across various medical fields.
On February 16, 2021, REGENXBIO announced positive interim data from the Phase I/IIa trial of RGX-314, a gene therapy for wet age-related macular degeneration (AMD). The treatment demonstrated durability over three years, with significant reductions in anti-VEGF injection burdens. Cohorts 4 and 5 showed stable visual acuity and decreased retinal thickness. Notably, 67% of patients were anti-VEGF injection-free for up to three years. The ATMOSPHERE trial, aimed at validating these findings, is currently enrolling patients. RGX-314's safety profile remains generally well-tolerated.
REGENXBIO Inc. (Nasdaq: RGNX) announced positive interim data from its Phase I/II trial of RGX-121 for treating Mucopolysaccharidosis Type II (MPS II, Hunter Syndrome) in children. Presented at the 17th Annual WORLD Symposium, the results from eight patients show a favorable safety profile and improvements in biomarker levels, indicating enhanced enzyme activity. RGX-121 aims to deliver the I2S enzyme gene via an AAV9 vector, with plans to commence patient enrollment for Cohort 3 at a higher dose in Q1 2021.
REGENXBIO Inc. (Nasdaq: RGNX) announced its participation in the Angiogenesis, Exudation, and Degeneration 2021 conference on February 12-13, 2021. Two oral presentations will showcase advancements in gene therapy for eye diseases:
- Subretinal Gene Therapy for Exudative AMD by Allen C. Ho, M.D. on February 13 at 9:15 a.m. ET
- Suprachoroidal Gene Therapy for Exudative AMD and Diabetic Retinopathy by Peter A. Campochiaro, M.D. on February 13 at 9:45 a.m. ET
These presentations highlight the company's innovative approaches using its NAV® Technology Platform.
REGENXBIO Inc. (Nasdaq: RGNX) has announced three oral and nine poster presentations for the 17th Annual WORLD Symposium™ from February 8-12, 2021. Key presentations include interim results from the Phase I/II trial of RGX-121 targeting mucopolysaccharidosis type II (MPS II). Notable presentations cover comparative effectiveness of gene therapies and clinical updates on RGX-121. REGENXBIO is focused on advancing its NAV Technology Platform for gene therapy applications, with exclusive rights to over 100 AAV vectors.