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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
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REGENXBIO Inc. (RGNX) will host a webcast to discuss new interim clinical data from the Phase I/II AFFINITY DUCHENNE trial of RGX-202 for Duchenne muscular dystrophy. The primary investigator, Aravindhan Veerapandiyan, M.D., will present the data on March 5, 2024. Additionally, Dr. Veerapandiyan will present at the MDA Clinical & Scientific Conference on March 6, 2024.
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REGENXBIO Inc. announces financial results for Q4 2023, highlighting progress on AAV Therapeutic product candidates. The company plans pivotal trials and BLA filing in 2024, with $314 million in cash to fund operations until H2 2025.
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REGENXBIO Inc. (RGNX) will host a conference call to discuss its Q4 and full-year 2023 financial results and recent operational highlights on February 27, 2024.
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REGENXBIO Inc. (RGNX) announced topline results from the Phase I/II/III CAMPSIITE® trial of RGX-121, a treatment for Mucopolysaccharidosis Type II (MPS II) or Hunter syndrome. The trial met its primary endpoint with statistical significance, supporting the BLA submission in 2024 using the accelerated approval pathway. Patients treated with RGX-121 showed continued improvement in neurodevelopmental skill acquisition up to four years, and the company plans to discuss these results as part of a full rare disease program update on its conference call.
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REGENXBIO Inc. (Nasdaq: RGNX) has completed enrollment in cohort 2 of the Phase I/II AFFINITY DUCHENNE trial of RGX-202, demonstrating strong microdystrophin expression across a wide range of patients. The third patient at dose level 1 showed the largest increase in microdystrophin expression at 83.4% of control. The company is on track to initiate a pivotal trial in the second half of 2024 and plans to discuss the new results in a conference call. Enrollment has been completed at dose level 2, and RGX-202 has been well tolerated with no drug-related serious adverse events in five patients. The company expects to make a pivotal dose determination in mid-2024 and share initial strength and functional assessment data for both dose levels. The Phase I/II AFFINITY DUCHENNE trial is a multicenter, open-label dose escalation and dose expansion clinical study to evaluate the safety, tolerability, and clinical efficacy of a one-time intravenous dose of RGX-202 in patients with Duchenne.
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REGENXBIO Inc. announced presentations on its program for the treatment of mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome, at the 20th Annual WORLDSymposium™ 2024. The topline results from the pivotal phase of the Phase I/II/III CAMPSIITE® trial of RGX-121 for the treatment of MPS II will be presented. The company will also discuss intracisternal administration of investigational AAV9 gene therapies to target the central nervous system in pediatric lysosomal disorders. A conference call will be held to discuss the CAMPSIITE trial pivotal phase topline results and the expedited plan for filing a Biologics License Application using the accelerated approval pathway in 2024.
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REGENXBIO Inc. announced positive interim data from the Phase II AAVIATE trial of ABBV-RGX-314 for the treatment of wet age-related macular degeneration (wet AMD) using suprachoroidal delivery. The treatment demonstrated the potential to sustain constant anti-VEGF therapy and stabilize or improve vision long-term. The data showed that ABBV-RGX-314 was well tolerated in over 100 patients, with no drug-related serious adverse events. The treatment also resulted in an 80% reduction in annualized injection rate and 50% of patients remaining injection-free.
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REGENXBIO Inc. (Nasdaq: RGNX) will present data from the Phase II AAVIATE® trial of suprachoroidal ABBV-RGX-314 for the treatment of wet age-related macular degeneration (AMD) at the Hawaiian Eye and Retina 2024 Meeting. ABBV-RGX-314 is an investigational one-time AAV Therapeutic developed in collaboration with AbbVie for the treatment of wet AMD, diabetic retinopathy, and other chronic retinal conditions.
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REGENXBIO Inc. (RGNX) faces a setback as the U.S. District Court for the District of Delaware granted Sarepta Therapeutics, Inc. summary judgment on invalidity in a patent infringement suit. The lawsuit concerns Sarepta's use of cultured host cell technology covered by a University of Pennsylvania patent to make clinical and commercial supplies of SRP-9001 for the treatment of Duchenne muscular dystrophy. REGENXBIO intends to file an immediate appeal. The ruling does not impact REGENXBIO's current licenses or therapeutic pipeline.
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REGENXBIO Inc. (Nasdaq: RGNX) announced the first patient received dose level 2 of RGX-202, a potential one-time AAV Therapeutic for Duchenne muscular dystrophy. The Phase I/II AFFINITY DUCHENNE trial is on track for pivotal dose determination and initiation of pivotal program in 2024. Initial biomarker data and preclinical efficacy studies show promising results for RGX-202, supporting its potential to be a best-in-class product.
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FAQ
What is the current stock price of REGENXBIO (RGNX)?
The current stock price of REGENXBIO (RGNX) is $10.15 as of November 18, 2024.
What is the market cap of REGENXBIO (RGNX)?
The market cap of REGENXBIO (RGNX) is approximately 510.8M.
What is REGENXBIO’s primary focus?
REGENXBIO specializes in the development and commercialization of gene therapies using its proprietary NAV Technology Platform to address various serious diseases.
What are the key therapeutic areas targeted by REGENXBIO’s platform?
REGENXBIO targets metabolic diseases, neurodegenerative conditions, and retinal diseases through its gene therapy platform.
Who are REGENXBIO’s major partners?
REGENXBIO collaborates with AbbVie, Novartis, and Eli Lilly, among others, to advance its gene therapy programs.
What recent achievements has REGENXBIO reported?
Recent achievements include positive trial results for RGX-202 and RGX-121, publication of ABBV-RGX-314 data in The Lancet, and a strong cash position.
What is ABBV-RGX-314?
ABBV-RGX-314 is a potential one-time gene therapy for wet age-related macular degeneration and diabetic retinopathy, developed in collaboration with AbbVie.
How is REGENXBIO’s financial health?
As of March 31, 2024, REGENXBIO reported a cash position of $380.5 million, expected to fund operations into 2026.
What are the key highlights of REGENXBIO’s financial results for Q1 2024?
Key highlights include $15.6 million in revenues, decreased operating expenses, and a net loss of $63.3 million.
What upcoming milestones is REGENXBIO targeting?
Upcoming milestones include advancing RGX-202 into pivotal trials and filing a BLA for RGX-121 in 2024.
What is the NAV Technology Platform?
The NAV Technology Platform is REGENXBIO's proprietary gene delivery system using novel adeno-associated virus (AAV) vectors for gene therapy.
Where can I find more information about REGENXBIO?
More information can be found on REGENXBIO’s official website at www.regenxbio.com.
REGENXBIO Inc.
Nasdaq:RGNX
RGNX Rankings
RGNX Stock Data
510.79M
49.42M
7.39%
89.06%
10.09%
Biotechnology
Biological Products, (no Disgnostic Substances)
United States of America
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