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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 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.
REGENXBIO Inc. (Nasdaq: RGNX) announced a conference call scheduled for November 2, 2021, at 4:30 p.m. ET. The call will cover the company's financial results for the quarter ended September 30, 2021, and recent operational highlights. Investors can access the call via phone or a webcast on the REGENXBIO website. The company is focused on gene therapy through its NAV® Technology Platform, which includes over 100 exclusive AAV vectors aimed at various therapeutic areas.
REGENXBIO announced positive interim data from the Phase II ALTITUDE trial of RGX-314, a gene therapy for diabetic retinopathy (DR). In Cohort 1, 33% of patients showed a two-step improvement in DR severity after three months, compared to none in the control group. The treatment was well-tolerated with no serious drug-related adverse events. Cohorts 2 and 3 are ongoing at a higher dose of 5x1011 GC/eye. This therapy could significantly benefit the approximately eight million people affected by DR in the U.S.
REGENXBIO (Nasdaq: RGNX) announced positive initial data from the Phase II AAVIATE trial of RGX-314 for wet AMD treatment via suprachoroidal delivery. In Cohort 1, 14 patients showed stable visual acuity and retinal thickness at six months, with a 75.9% reduction in anti-VEGF injections. No drug-related serious adverse events were reported among 50 patients in Cohorts 1-3. The trial is expanding to include a third dose level of RGX-314. A conference call featuring key opinion leaders will provide further insights.
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