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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 (RGNX) reported $22 million in Q2 2021 revenue, up from $16.6 million in Q2 2020, driven by a $6.5 million increase in Zolgensma royalties. The company holds $593 million in cash as of June 30, 2021, positioning it well to fund operations through mid-2023. Ongoing clinical trials for RGX-314 and RGX-121 show promise, with interim data set for presentation at the Retina Society meeting. Meanwhile, the net loss rose to $57.6 million, reflecting increased R&D and G&A expenses. The firm aims to submit an IND for RGX-202 by year-end 2021.
REGENXBIO Inc. (Nasdaq: RGNX) will host a conference call on August 9, 2021, at 4:30 p.m. ET to discuss its Q2 2021 financial results and operational highlights. Interested participants can join the call by dialing (855) 422-8964 or accessing the webcast on the company's website. REGENXBIO focuses on gene therapy using its proprietary NAV® Technology Platform, which includes over 100 adeno-associated viral vectors. The company aims to develop a robust pipeline across various therapeutic areas.
REGENXBIO Inc. (Nasdaq: RGNX) announced its participation in two upcoming virtual investor conferences in June 2021. The Bank of America 2021 Napa Biopharma Virtual Conference will feature a fireside chat on June 16, 2021, at 12:30 p.m. E.T. Additionally, the Raymond James Human Health Innovation Conference is scheduled for June 21, 2021. Investors can access the live webcast of the Bank of America event on REGENXBIO's website, with an archived replay available for 30 days thereafter. REGENXBIO focuses on gene therapy through its proprietary NAV Technology Platform.
REGENXBIO has announced positive interim data from the ongoing Phase I/II trial of RGX-121, a gene therapy for Mucopolysaccharidosis Type II (MPS II). Key findings include no serious drug-related adverse events and evidence of CNS activity in patients up to two years post-administration. Biomarkers indicate strong I2S enzyme activity, and most patients showed continued neurocognitive development. The company has initiated dosing in Cohort 3 at an increased dosage, further enhancing prospects for RGX-121 as a treatment option for MPS II.
REGENXBIO Inc. (Nasdaq: RGNX) will present at three upcoming virtual investor conferences in May 2021. The events include:
- Bank of America Securities 2021 Virtual Health Care Conference on May 12, 2021, at 12:30 p.m. E.T.
- RBC Capital Markets 2021 Global Healthcare Virtual Conference on May 18, 2021, at 10:55 a.m. E.T.
- UBS Global Healthcare Virtual Conference on May 25, 2021, at 11:00 a.m. E.T.
Live webcasts will be accessible on the REGENXBIO website, with replays available for 30 days.
REGENXBIO reported Q1 2021 financial results with revenues increasing to $18.9 million from $17.6 million year-over-year, primarily driven by a rise in Zolgensma royalties. The company holds $657 million in cash and equivalents, sufficient for operations into late 2023. Progress in clinical trials includes ongoing enrollment in pivotal trials for RGX-314 for wet AMD and advancements in RGX-121 and RGX-111 programs. Net loss widened to $50.1 million compared to $40.0 million in Q1 2020, reflecting increased R&D investments.
REGENXBIO Inc. (Nasdaq: RGNX) will host a conference call on May 5, 2021 at 4:30 p.m. ET to discuss its financial results for the quarter ended March 31, 2021, along with recent operational highlights. Investors can join the call by dialing (855) 422-8964 or (210) 229-8819 for international access, with the passcode 7044809. A live or recorded webcast will also be available on their website for about 30 days after the call. REGENXBIO focuses on gene therapy using its proprietary NAV® Technology Platform with over 100 AAV vectors.
REGENXBIO Inc. (Nasdaq: RGNX) announced that it will present four oral and nine poster presentations at the American Society of Gene and Cell Therapy's 24th Annual Meeting, scheduled for May 11-14, 2021. Oral presentations feature topics such as AAV tissue transduction and the RGX-121 gene therapy for mucopolysaccharidosis type II. The poster presentations cover a range of subjects including AAV vector evaluation and gene therapy methodologies. Detailed information about the presentations can be found on the ASGCT website.
REGENXBIO Inc. (Nasdaq: RGNX) announced that Curran M. Simpson, Chief Operations and Technology Officer, will present at the Chardan 5th Annual Genetic Medicines Manufacturing Summit on April 27, 2021, at 3:15 p.m. ET. The event will be held virtually. A webcast of the presentation will be available in the Investors section of REGENXBIO's website and can be accessed at www.regenxbio.com. An archived replay will be accessible for 30 days post-event.
REGENXBIO is focused on advancing gene therapy using its NAV® Technology Platform.
REGENXBIO has announced the dosing of the first patient in Cohort 3 of its Phase I/II trial for RGX-121, a one-time gene therapy targeting MPS II (Hunter Syndrome). This trial, involving a total of nine patients across the U.S. and Brazil, aims to assess safety and efficacy. Previous cohorts indicated positive interim results with reductions in CNS biomarkers and neurocognitive development. RGX-121 leverages the AAV9 vector for gene delivery, with potential systemic benefits. The trial's primary endpoint is establishing safety while evaluating enzyme activity and neurocognitive outcomes.
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