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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 Inc. (Nasdaq: RGNX) announced its participation in the SVB Securities Global Biopharma Conference on February 16, 2023, at 12:00 p.m. ET. A live webcast will be available on the company's website, with a replay archived for 30 days. REGENXBIO is a biotechnology company focused on gene therapy, utilizing its proprietary NAV Technology Platform, which includes over 100 AAV vectors for therapeutic development. The company aims to advance five AAV Therapeutics into pivotal or commercial stages by 2025, under its "strategy.
This engagement highlights the company's commitment to innovation and progress in biotechnology.
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.
REGENXBIO Inc. (Nasdaq: RGNX) has announced progress on its AAV Therapeutics for treating CLN2 disease, the most common form of Batten disease. The first patient has been dosed with RGX-181 in a Brazil-based study, showing no severe adverse effects. Additionally, REGENXBIO received approval for RGX-381 from the UK Health Authority, with plans for a Phase I/II clinical trial in the first half of 2023. Both therapies aim to provide long-term benefits for patients suffering from debilitating neurodegenerative symptoms related to CLN2 disease.
REGENXBIO (RGNX) announced the completion of dosing in the expanded Cohort 2 of its Phase I/II trial for RGX-111, an investigational gene therapy for severe Mucopolysaccharidosis Type I (MPS I). Eight patients have participated in this trial, aimed at evaluating RGX-111's safety and potential effectiveness. The company plans to manufacture commercial-grade material to further develop RGX-111. Interim trial updates are expected in the first half of 2023. RGX-111 leverages the NAV AAV9 vector to deliver the IDUA gene directly to the CNS.
REGENXBIO Inc. (Nasdaq: RGNX) announced its participation in two investor conferences. The first is the Piper Sandler 34th Annual Healthcare Conference on December 1, 2022, at 9:10 a.m. ET in New York, NY. The second event is the BofA Securities 2022 Virtual Biotech SMID Cap Conference, with a fireside chat on December 8, 2022, at 10:50 a.m. ET. Live webcasts can be accessed on REGENXBIO's website, with replays available for 30 days. The company is a leader in gene therapy, focusing on advancing its 5x'25 strategy to develop five AAV Therapeutics by 2025.
REGENXBIO (Nasdaq: RGNX) reported third-quarter financial results, highlighting a net loss of $75.5 million, compared to a $58.4 million loss in Q3 2021. Revenues decreased to $26.5 million, chiefly due to falling Zolgensma royalties. The company maintains a robust cash position of $617 million, expected to fund operations through 2025. Progress on the 5x25 strategy continues, with RGX-314 trials for wet AMD and diabetic retinopathy demonstrating positive interim data. BLA filings for RGX-314 and RGX-121 are anticipated in 2024, affirming ongoing commitment to gene therapies.
REGENXBIO (Nasdaq: RGNX) announced positive interim results from the Phase II ALTITUDE trial of RGX-314, aimed at treating diabetic retinopathy (DR) via suprachoroidal delivery. The results showed that RGX-314 was well tolerated in 50 patients across the first three cohorts, with no serious drug-related adverse events. Clinically meaningful improvements were noted, including 54% of patients achieving any DRSS improvement, compared to 20% in the control group. The trial will expand to include a third dose level and new cohorts, with the goal of further evaluating RGX-314's potential.
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