Regenxbio Stock Price, News & Analysis

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.

REGENXBIO Inc. (Nasdaq: RGNX) has announced its participation in two upcoming investor conferences. The first is the Credit Suisse 31st Annual Healthcare Conference on November 8, 2022, located at Terranea Resort, Rancho Palos Verdes, CA. The second is the Barclays Sixth Annual Gene Editing/Therapy Summit on November 14, 2022, where REGENXBIO will discuss AAV Gene Therapy's current status and future direction at 2:50 p.m. ET. The company focuses on advancing AAV Therapeutics with its proprietary NAV Technology Platform, emphasizing its goal of progressing five products to pivotal-stage or commercial status by 2025.