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REGENXBIO Inc. reports news on its AAV gene therapy pipeline and manufacturing platform for rare, neuromuscular and retinal diseases. Recurring updates cover RGX-202 for Duchenne muscular dystrophy; clemidsogene lanparvovec (RGX-121) for MPS II; RGX-111 for MPS I; and surabgene lomparvovec (ABBV-RGX-314) for wet AMD and diabetic retinopathy.
Company announcements also include FDA regulatory communications, clinical and preclinical data presentations, financial results, operational highlights, and collaboration activity with partners including Nippon Shinyaku and AbbVie. News on the NAV and NAVXpress platforms addresses AAV vector technology, product quality, and manufacturing capabilities used across REGENXBIO's investigational programs.
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 ASCENTTM 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.
REGENXBIO announced positive interim data from Phase II trials of RGX-314 for treating wet AMD and diabetic retinopathy. The AAVIATE trial showed RGX-314 was well tolerated among 50 patients, with no serious adverse events. In Cohort 2, a 71.8% reduction in anti-VEGF treatment burden was observed, with 40% of patients injection-free. Cohort 1 of the ALTITUDE trial reported stable visual acuity at three months. These results were presented at the AAO 2021 Annual Meeting, highlighting RGX-314's potential in retinal disease management.
REGENXBIO (Nasdaq: RGNX) has finalized a collaboration and license agreement with AbbVie for the gene therapy RGX-314, targeting wet AMD and diabetic retinopathy. The deal includes a $370 million upfront payment and potential earnings of up to $1.38 billion based on future milestones. REGENXBIO will manage ongoing trials, while AbbVie will lead global clinical development and commercialization. Both companies will share profits from U.S. sales equally, with tiered royalties on international sales. RGX-314 aims to inhibit VEGF, which contributes to retinal fluid accumulation.
REGENXBIO Inc. (Nasdaq: RGNX) announced its participation in two upcoming virtual investor conferences. The Barclays Gene Editing & Gene Therapy Summit is set for November 15, 2021, at 1:30 p.m. ET. Additionally, the Piper Sandler 33rd Annual Virtual Healthcare Conference will take place on December 2, 2021, with a recorded fireside chat available starting November 22, 2021, at 10:00 a.m. ET. A live webcast and recorded presentations can be accessed via REGENXBIO's website for 30 days post-event.
REGENXBIO Inc. (Nasdaq: RGNX) announced that data from its RGX-314 clinical trials will be presented at the American Academy of Ophthalmology 2021 Annual Meeting in New Orleans, LA, from November 12-15, 2021. RGX-314, a potential one-time gene therapy targeting wet age-related macular degeneration and diabetic retinopathy, will be showcased in an oral presentation by Dr. Robert L. Avery on November 12. The therapy utilizes the NAV AAV8 vector to inhibit VEGF, potentially preventing fluid accumulation in the retina.
REGENXBIO and AbbVie announced a strategic collaboration to develop RGX-314, focusing on wet AMD and diabetic retinopathy, with REGENXBIO receiving a $370 million upfront payment. The partnership aims to leverage both companies' expertise for potential milestone payments totaling $1.38 billion. Positive Phase II trial data for RGX-314 showed stable visual acuity and reduced treatment burden. Financially, REGENXBIO reported $30.8 million in Q3 revenues, down from $98.9 million the same period in 2020, and a net loss of $58.4 million. Cash reserves were $533.5 million as of September 30, 2021.