Regenxbio Stock Price, News & Analysis

REGENXBIO Inc. (Nasdaq: RGNX) announced promising interim results from the Phase II ALTITUDE™ trial for RGX-314, aimed at treating diabetic retinopathy (DR). Presented at the Angiogenesis conference, data shows nearly 50% of patients in Cohort 1 achieved a significant improvement on the Diabetic Retinopathy Severity Scale after six months post-injection. The treatment was well tolerated with minimal adverse events. The trial continues to enroll patients in subsequent cohorts, suggesting positive potential for RGX-314 as a viable option for patients suffering from DR.

REGENXBIO presented positive interim data for RGX-111, a potential one-time gene therapy for MPS I, at the 18th Annual WORLD Symposium on Feb 9, 2022. RGX-111 showed good tolerability with no serious drug-related adverse events across two dosing cohorts. Biomarker and neurodevelopmental assessments revealed encouraging results, indicating biological activity in the CNS after treatment. The Phase I/II trial has expanded to enroll up to six additional patients. RGX-111 aims to address the limitations of current MPS I treatments, highlighting its potential in gene therapy.

REGENXBIO (Nasdaq: RGNX) reported positive interim results from the Phase I/II trial of RGX-121 for treating Mucopolysaccharidosis Type II (MPS II). The gene therapy was well-tolerated, showing no drug-related serious adverse events across 13 patients. Significant biomarker reductions were observed, particularly in CSF Heparan sulfate and D2S6 levels. Neurodevelopmental improvements continued for up to two years post-administration.

The expansion of Cohort 3 using commercial-scale cGMP material is planned for the first half of 2022.

REGENXBIO Inc. (Nasdaq: RGNX) announced its participation in a fireside chat at the 11th Annual SVB Leerink Global Healthcare Conference on February 16, 2022, at 8:40 a.m. ET. The event will be held virtually, and a webcast will be accessible on the company's website, with an archived replay available for 30 days post-event. REGENXBIO focuses on gene therapy, utilizing its proprietary NAV Technology Platform, which includes over 100 novel AAV vectors in developing a pipeline of therapeutic candidates.

REGENXBIO (Nasdaq: RGNX) announced three oral presentations at the Angiogenesis, Exudation, and Degeneration 2022 Conference, occurring from February 11 to 12, 2022. These presentations will include updated interim data from the ongoing Phase II ALTITUDE™ trial of RGX-314, targeting diabetic retinopathy. Notable presentations are scheduled for February 12, covering gene therapy advancements led by experts from renowned institutions. REGENXBIO's NAV® Technology Platform underpins its gene therapy pipeline, reflecting a commitment to innovative treatments.

REGENXBIO Inc. (Nasdaq: RGNX) announced presentations at the 18th Annual WORLD Symposium™ in San Diego from February 7-11, 2022. The company will showcase interim data from Phase I/II clinical trials for RGX-121 and RGX-111, targeting mucopolysaccharidosis types I and II. Notably, five oral and seven poster presentations are scheduled, featuring key researchers and significant advancements in treatment methodologies. This event aims to highlight the company's progress in gene therapy, showcasing its innovative approaches to serious genetic disorders.

REGENXBIO announced the initiation of the ASCENT^TM Phase III clinical trial, evaluating RGX-314 as a one-time gene therapy for wet AMD. The trial aims to enroll 465 patients across the U.S. and Canada, comparing RGX-314 delivery to standard aflibercept treatment. A Biologics License Application (BLA) is expected to be submitted to the FDA in 2024 based on results from ASCENT and the ongoing ATMOSPHERE trial. The trial's primary endpoint focuses on non-inferiority to aflibercept based on visual acuity changes after one year.

REGENXBIO has received FDA clearance for its IND application to initiate the clinical trial of RGX-202, a novel gene therapy targeting Duchenne muscular dystrophy. This therapy utilizes a microdystrophin transgene delivered through the proprietary NAV® AAV8 vector. The AFFINITY DUCHENNE trial will test the safety and efficacy of RGX-202 in pediatric patients, with dosing expected to start in the first half of 2022. The trial incorporates an innovative design with a comprehensive immunosuppressive regimen to enhance safety and tolerability.

On November 22, 2021, REGENXBIO announced the FDA granted Orphan Drug Designation for RGX-202, a potential one-time gene therapy targeting Duchenne muscular dystrophy. RGX-202 features a novel microdystrophin transgene aimed at enhancing muscle resistance to damage. The company plans to submit an Investigational New Drug (IND) application by year-end 2021. Commercial-scale cGMP material has been produced, supporting clinical development. Orphan drug status provides key advantages like tax credits and marketing exclusivity.