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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 reported Q4 and full-year 2020 financial results with revenues of $154.6 million, up from $35.2 million in 2019, primarily due to Zolgensma royalties. The company holds $523 million in cash and equivalents, enhanced by a recent $230 million stock offering. Key developments include active patient enrollment in pivotal trials for RGX-314 targeting wet AMD, with a BLA filing anticipated in 2024. Positive interim data from RGX-121's Phase I/II trial showcases reduced CNS biomarkers. Research on RGX-202 for Duchenne Muscular Dystrophy and other therapies continues with IND applications expected soon.
REGENXBIO Inc. (Nasdaq: RGNX) will participate in several upcoming virtual investor conferences in March 2021. The events include:
- Raymond James 42nd Annual Institutional Investors Conference: March 3, 2021, at 10:50 a.m. ET
- Barclays Global Healthcare Conference: March 9, 2021, at 4:10 p.m. ET
- Morgan Stanley Virtual Healthcare Corporate Access Day: March 16, 2021
Live webcasts for the Raymond James and Barclays events will be available on REGENXBIO's website, with archived versions accessible for 30 days post-event.
REGENXBIO Inc. (RGNX) announced a conference call scheduled for March 1, 2021, at 4:30 p.m. ET, to discuss its financial results for the fourth quarter and full year 2020, along with operational highlights. The call can be accessed via phone or through a webcast on the company's website. REGENXBIO is recognized for its innovative gene therapy solutions using its NAV Technology Platform, which includes a range of adeno-associated virus vectors aimed at therapeutic advancements across various medical fields.
On February 16, 2021, REGENXBIO announced positive interim data from the Phase I/IIa trial of RGX-314, a gene therapy for wet age-related macular degeneration (AMD). The treatment demonstrated durability over three years, with significant reductions in anti-VEGF injection burdens. Cohorts 4 and 5 showed stable visual acuity and decreased retinal thickness. Notably, 67% of patients were anti-VEGF injection-free for up to three years. The ATMOSPHERE trial, aimed at validating these findings, is currently enrolling patients. RGX-314's safety profile remains generally well-tolerated.
REGENXBIO Inc. (Nasdaq: RGNX) announced positive interim data from its Phase I/II trial of RGX-121 for treating Mucopolysaccharidosis Type II (MPS II, Hunter Syndrome) in children. Presented at the 17th Annual WORLD Symposium, the results from eight patients show a favorable safety profile and improvements in biomarker levels, indicating enhanced enzyme activity. RGX-121 aims to deliver the I2S enzyme gene via an AAV9 vector, with plans to commence patient enrollment for Cohort 3 at a higher dose in Q1 2021.
REGENXBIO Inc. (Nasdaq: RGNX) announced its participation in the Angiogenesis, Exudation, and Degeneration 2021 conference on February 12-13, 2021. Two oral presentations will showcase advancements in gene therapy for eye diseases:
- Subretinal Gene Therapy for Exudative AMD by Allen C. Ho, M.D. on February 13 at 9:15 a.m. ET
- Suprachoroidal Gene Therapy for Exudative AMD and Diabetic Retinopathy by Peter A. Campochiaro, M.D. on February 13 at 9:45 a.m. ET
These presentations highlight the company's innovative approaches using its NAV® Technology Platform.
REGENXBIO Inc. (Nasdaq: RGNX) has announced three oral and nine poster presentations for the 17th Annual WORLD Symposium™ from February 8-12, 2021. Key presentations include interim results from the Phase I/II trial of RGX-121 targeting mucopolysaccharidosis type II (MPS II). Notable presentations cover comparative effectiveness of gene therapies and clinical updates on RGX-121. REGENXBIO is focused on advancing its NAV Technology Platform for gene therapy applications, with exclusive rights to over 100 AAV vectors.
REGENXBIO Inc. (Nasdaq: RGNX) has successfully closed its public offering of 4,260,000 common stock shares at $47.00 each, yielding approximately $230.3 million in gross proceeds after the underwriters' full option exercise for an additional 639,000 shares. This offering was managed by BofA Securities, Morgan Stanley, and Barclays. The shares were offered under a shelf registration statement effective since August 8, 2018. The funds raised will support REGENXBIO's ongoing gene therapy developments utilizing its proprietary NAV Technology Platform.
REGENXBIO Inc. (Nasdaq: RGNX) announced its presentation at the 39th Annual J.P. Morgan Healthcare Conference on January 14, 2021, at 11:40 a.m. ET. This virtual conference will showcase the company's advancements in gene therapy. A webcast will be available on REGENXBIO's website, with an archived replay accessible for 30 days. The company utilizes its proprietary NAV Technology Platform to develop innovative gene delivery systems across multiple therapeutic areas.
REGENXBIO Inc. (Nasdaq: RGNX) has priced a public offering of 4,260,000 shares of common stock at $47.00 per share. The offering is expected to generate approximately $200.2 million in gross proceeds before deductions. Closing is anticipated on January 12, 2021, subject to customary conditions. An additional 639,000 shares may be purchased by underwriters within 30 days. This offering is conducted under a shelf registration statement with the SEC, aiming to support REGENXBIO's ongoing gene therapy initiatives.
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