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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 (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.
REGENXBIO (RGNX) reports Q1 2022 financial results, highlighting a revenue increase to $22.2 million, primarily from Zolgensma royalties. The company has $765 million in cash, providing operational runway into 2025. Key developments include ongoing enrollment in pivotal trials for RGX-314 targeting wet AMD and diabetic retinopathy, and delayed initiation of the AFFINITY DUCHENNE trial. The in-house cGMP facility is now operational, enhancing manufacturing capacity. CEO Kenneth T. Mills expresses optimism about advancing five AAV Therapeutics into pivotal stages by 2025.
REGENXBIO Inc. (Nasdaq: RGNX) announced its participation in two upcoming investor conferences. The Bank of America Securities 2022 Healthcare Conference will take place on May 10, 2022, at the Encore Hotel in Las Vegas, with a presentation scheduled for 4:00 p.m. PT. The UBS Global Healthcare Conference 2022 is set for May 24, 2022, at Lotte New York Palace, with a presentation at 10:45 a.m. ET. Webcasts of both events will be available on the REGENXBIO website for approximately 30 days following their presentations.
REGENXBIO Inc. (Nasdaq: RGNX) will present at the American Society of Gene & Cell Therapy (ASGCT) 25th Annual Meeting from May 16-19, 2022, in Washington, D.C. Key presentations include interim analyses of the RGX-121 and RGX-111 gene therapies for MPS II and MPS I, respectively, and insights on novel AAV vectors. Notable speakers include Nina Hunter, Ph.D., and Roberto Giugliani, M.D., among others. The event highlights REGENXBIO's capabilities in gene therapy, emphasizing its NAV Technology Platform with over 100 AAV vectors developed for various therapeutic areas.
REGENXBIO Inc. (Nasdaq: RGNX) will host a conference call on May 4, 2022, at 4:30 p.m. ET to discuss its financial results for Q1 2022 and operational highlights. Interested parties can access the call via phone or a live webcast on the company's website. REGENXBIO focuses on gene therapy, utilizing its proprietary NAV Technology Platform, which includes rights to over 100 novel AAV vectors applicable across various therapeutic areas.
REGENXBIO Inc. (Nasdaq: RGNX) announced its participation in a fireside chat at Chardan's 6th Annual Genetic Medicines and Cell Therapy Manufacturing Summit, scheduled for April 26, 2022, at 8:30 a.m. ET. The virtual conference will allow investors to access a webcast of the chat through REGENXBIO's website, with an archived version available for approximately 30 days afterward. The company focuses on gene therapy, leveraging its proprietary NAV Technology Platform, which includes over 100 novel AAV vectors, to develop treatments across various therapeutic areas.
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