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) is a pioneering biotechnology company dedicated to transforming patient lives through the development and commercialization of innovative gene therapies. The company's core focus lies in the use of recombinant adeno-associated virus (AAV) gene therapy, leveraging its proprietary NAV® Technology Platform. This platform includes exclusive rights to over 100 novel AAV vectors, such as AAV7, AAV8, AAV9, and AAVrh10, which are crucial for delivering therapeutic genes. REGENXBIO aims to address serious diseases with significant unmet medical needs through both in-house development and third-party licensing agreements.
REGENXBIO's mission is realized through its robust pipeline, which targets various challenging conditions:
- Metabolic Diseases: The company is working on therapies for homozygous familial hypercholesterolemia.
- Neurodegenerative Conditions: Their efforts include treatments for mucopolysaccharidosis (MPS), specifically Type II (Hunter syndrome).
- Retinal Diseases: Projects include therapies for wet age-related macular degeneration (wet AMD) and X-linked retinitis pigmentosa.
REGENXBIO has several high-profile partnerships. Collaborations with AbbVie are advancing ABBV-RGX-314, a potential one-time treatment for wet AMD and diabetic retinopathy. They also have licensed their technology to renowned firms like Novartis and Eli Lilly, which are using it to develop therapies for spinal muscular atrophy and various neurodegenerative diseases, respectively.
Recent Achievements and Financial Highlights:
- Completion of enrollment at dose level 2 in the Phase I/II AFFINITY DUCHENNE® trial for RGX-202, aimed at treating Duchenne muscular dystrophy.
- Topline results from the CAMPSIITE® trial for RGX-121, targeting MPS II, showing significant improvement in brain function biomarkers.
- Publication of ABBV-RGX-314 trial results in The Lancet, demonstrating long-term efficacy for wet AMD.
Financial Condition: As of March 31, 2024, REGENXBIO has a strong cash position, with $380.5 million in cash, cash equivalents, and marketable securities. Despite a net loss of $63.3 million for Q1 2024, the company expects its cash reserves to fund operations into 2026, bolstered by proceeds from a recent public offering and potential milestone payments from partners.
REGENXBIO continues to make significant strides in its
REGENXBIO Inc. (Nasdaq: RGNX) has announced its participation in two key investor conferences:
- Morgan Stanley 20th Annual Global Healthcare Conference on September 14, 2022, at 2:55 p.m. ET in New York.
- UBS Biotech South Beach Conference on September 29, 2022, in Miami.
A live webcast of the Morgan Stanley conference will be available on REGENXBIO's website with a 30-day archived replay. The company is committed to advancing gene therapy through its proprietary NAV Technology Platform and aims to progress five AAV Therapeutics by 2025.
REGENXBIO (Nasdaq: RGNX) announced positive interim data for RGX-121, a gene therapy for Mucopolysaccharidosis Type II (MPS II), at the SSIEM Annual Symposium. The Phase I/II/III CAMPSIITE trial revealed significant median reductions in cerebrospinal fluid (CSF) glycosaminoglycans (GAGs) at 48 weeks, demonstrating a favorable safety profile with no serious drug-related adverse events. The company plans to file a Biologics License Application in 2024 via the accelerated approval pathway, aiming to address significant unmet needs in MPS II treatment.
REGENXBIO Inc. (Nasdaq: RGNX) announced key presentations at the Society for the Study of Inborn Errors of Metabolism Annual Symposium from August 30 to September 2, 2022, in Freiburg, Germany. The focus will be on new data from the Phase I/II/III CAMPSIITE™ trial of RGX-121, a gene therapy for Mucopolysaccharidosis Type II, and RGX-111 for Mucopolysaccharidosis Type I. Notable presentations include interim analyses by Dr. Roberto Giugliani and Dr. Raymond Wang, as well as a discussion on neurodevelopment in MPS II by Dr. Maria Escolar.
REGENXBIO Inc. (Nasdaq: RGNX) will present at the H.C. Wainwright 2nd Annual Ophthalmology Virtual Conference on August 17, 2022, at 11:00 a.m. ET. The panel will focus on Unique Delivery Technology Platforms for Better Compliance and Efficacy. A live webcast will be accessible on the company's website, followed by an archived replay for 30 days. REGENXBIO is a clinical-stage biotechnology company harnessing gene therapy through its proprietary NAV Technology Platform, which includes over 100 exclusive AAV vectors, supporting its goal of advancing five AAV Therapeutics by 2025.
REGENXBIO announced its Q2 2022 financial results, reporting revenues of $32.6 million, driven by Zolgensma royalty revenues, up from $22.0 million in Q2 2021. The net loss increased to $68.2 million, or $1.58 per share, compared to $57.6 million a year ago. Key developments include progress in the RGX-314 program for wet AMD and diabetic retinopathy, with BLA filings anticipated in 2024. The company holds $682 million in cash, providing operational runway into 2025, supporting its '5x'25 strategy to have five gene therapies on the market or in late-stage development by 2025.
REGENXBIO (Nasdaq: RGNX) announced plans to file a Biologics License Application (BLA) for RGX-121 in 2024, seeking FDA's accelerated approval for treating Mucopolysaccharidosis Type II (MPS II). The pivotal CAMPSIITE trial is actively enrolling patients aged 4 months to 5 years. RGX-121, a one-time gene therapy, aims to provide a new treatment option as current therapies are inadequate. Preliminary trial results show a well-tolerated treatment with potential positive effects on neurodevelopmental function and significant reductions in glycosaminoglycans (GAGs) in cerebrospinal fluid.
REGENXBIO Inc. (Nasdaq: RGNX) will conduct a conference call on August 3, 2022, at 4:30 p.m. ET to discuss its financial results for the quarter ended June 30, 2022, along with recent operational highlights. Investors and analysts can register for the event via the provided links. REGENXBIO is focused on gene therapy, employing its NAV Technology Platform, which includes over 100 AAV vectors. The company aims to advance five AAV therapeutics into pivotal-stage or commercial products by 2025.
REGENXBIO (Nasdaq: RGNX) has completed enrollment in Cohort 5 of the Phase II AAVIATE trial, evaluating RGX-314 for wet age-related macular degeneration (wet AMD) via suprachoroidal delivery. This marks 85 subjects dosed across five cohorts. The company anticipates releasing additional suprachoroidal data within the year. RGX-314 aims to provide a one-time, in-office treatment option for patients facing vision loss. The trial compares RGX-314 with standard anti-VEGF therapies. REGENXBIO is committed to advancing its gene therapy platform targeting significant retinal diseases.
REGENXBIO Inc. (Nasdaq: RGNX) has inaugurated its new Manufacturing Innovation Center in Montgomery County, Maryland, investing $65 million to enhance gene therapy manufacturing capabilities. This facility will produce NAV Technology-based AAV gene therapies at a scale of up to 2,000 liters. It aims to streamline the transition from research to clinical and commercial readiness, supporting REGENXBIO's goal to develop five AAV therapeutics by 2025. The center is designed to meet global regulatory standards and features advanced production and quality control facilities.
REGENXBIO Inc. (Nasdaq: RGNX) appointed Jennifer Zachary to its Board of Directors effective June 3, 2022. With over 20 years of experience in the pharmaceutical and medical device industries, she will also serve on the Audit Committee. Zachary, currently the Executive Vice President and General Counsel of Merck, brings significant legal and regulatory expertise. Her addition is seen as pivotal for REGENXBIO as it implements its '5x'25' strategy aimed at advancing five AAV Therapeutics to pivotal or commercial stages by 2025.
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