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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) 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 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.
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