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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
REGENXBIO (RGNX) announced positive interim data for RGX-111, a one-time gene therapy targeting severe Mucopolysaccharidosis Type I (MPS I), at the WORLD Symposium. The Phase I/II trial shows RGX-111 is well-tolerated with no serious adverse events. Key findings include positive CNS biomarker activity and encouraging neurodevelopmental gains among participants. RGX-111 is designed to deliver the IDUA gene via AAV9 vector, aiming for long-term treatment effects. The company expects to manufacture commercial-scale material for further clinical development in 2023, aligning with its '5x'25 strategy to advance multiple gene therapies.
REGENXBIO announced promising interim data from the Phase I/II/III CAMPSIITE trial for RGX-121, a potential one-time gene therapy for Mucopolysaccharidosis Type II (MPS II). Results show RGX-121's excellent tolerability with no serious adverse events reported across three dosage levels. Notably, patients in Cohort 3 exhibited significant reductions in CSF GAGs, nearing normal levels at 48 weeks. Furthermore, improvements in neurodevelopmental functions were observed up to three years post-treatment. REGENXBIO plans to file for Biologics License Application in 2024, using the accelerated approval pathway.
REGENXBIO (NASDAQ: RGNX) announced a conference call scheduled for February 28, 2023, at 4:30 p.m. ET. This call will cover the company’s financial results for Q4 and the full year ended December 31, 2022, along with recent operational highlights.
REGENXBIO, a clinical-stage biotechnology company, focuses on gene therapy through its proprietary NAV Technology Platform, which comprises over 100 novel AAV vectors. The company aims to advance five AAV Therapeutics into pivotal-stage or commercial products by 2025.
REGENXBIO Inc. (RGNX) announced new interim data from trials of its gene therapies RGX-121 and RGX-111 for MPS II and MPS I, respectively. These results will be showcased at the 19th Annual WORLD Symposium™ from February 22-26, 2023, in Orlando, Florida. The company will present three oral and eight poster presentations, including interim analyses from both clinical trials. Additionally, REGENXBIO will host a symposium session on February 24, discussing AAV gene therapy for neuronopathic MPS II. The event underscores REGENXBIO's commitment to advancing gene therapy solutions and enhancing treatment options for rare diseases.
REGENXBIO (RGNX) announced promising interim results from a Phase II bridging study of RGX-314, a gene therapy for wet AMD, using its NAVXpress manufacturing platform. The study involved 60 patients and highlighted that RGX-314 produced via this new process showed similar safety and efficacy profiles to prior methods. The company aims for these results to support Biologics License Application (BLA) submission in 2024. With no severe adverse events linked to RGX-314, the therapy may significantly reduce treatment burdens for patients. A live webcast discussing these findings occurred on February 11, 2023, showcasing advancements toward RGX-314's commercialization.
REGENXBIO Inc. (NASDAQ: RGNX) announced three key presentations on RGX-314 at the Angiogenesis, Exudation, and Degeneration 2023 Conference, scheduled for February 10-11, 2023. These presentations will showcase data from a Phase II bridging study assessing RGX-314's pharmacodynamics, safety, and efficacy for treating neovascular age-related macular degeneration (AMD), utilizing cGMP material from the NAVXpress™ platform. Presenters include Dr. Charles Wykoff, Dr. Allen Ho, and Dr. Peter Campochiaro, highlighting long-term follow-up and new delivery methods for AMD and diabetic retinopathy, which could support RGX-314's future commercialization.
REGENXBIO has commenced the Phase I/II AFFINITY DUCHENNE trial for RGX-202 to treat Duchenne muscular dystrophy. This innovative gene therapy utilizes a novel microdystrophin and the NAV® AAV8 vector, aiming to improve muscle strength in affected boys. The trial will evaluate safety, tolerability, and efficacy with a focus on pediatric patients aged 4 to 11. Additionally, REGENXBIO is enrolling patients for the observational AFFINITY BEYOND study, which aims to assess AAV8 antibody prevalence. This initiative aligns with the company's '5x'25' strategy to advance five gene therapies by 2025, highlighting a commitment to addressing unmet medical needs in the Duchenne community.
REGENXBIO Inc. (Nasdaq: RGNX) announced leadership promotions, appointing Curran Simpson as Chief Operating Officer and Shiva Fritsch as Chief Communications Officer. These changes follow a transformative year for the company, including the launch of a gene therapy Manufacturing Innovation Center and progress under the '5 x '25' initiative to advance five AAV therapeutics into pivotal stages by 2025. Simpson, with extensive biopharmaceutical experience, will oversee critical operational functions, while Fritsch will manage communications and human resources.
REGENXBIO Inc. (Nasdaq: RGNX) announced it will present at the 41st Annual J.P. Morgan Healthcare Conference on January 10, 2023, at 11:15 a.m. PT in San Francisco, CA. The presentation will include a live webcast accessible on the company's website, with an archive available for 30 days after the event. REGENXBIO is focused on gene therapy, utilizing its proprietary NAV Technology Platform and aims to progress five AAV Therapeutics into pivotal or commercial stages by 2025.
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