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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 Inc. (Nasdaq: RGNX) has announced progress on its AAV Therapeutics for treating CLN2 disease, the most common form of Batten disease. The first patient has been dosed with RGX-181 in a Brazil-based study, showing no severe adverse effects. Additionally, REGENXBIO received approval for RGX-381 from the UK Health Authority, with plans for a Phase I/II clinical trial in the first half of 2023. Both therapies aim to provide long-term benefits for patients suffering from debilitating neurodegenerative symptoms related to CLN2 disease.
REGENXBIO (RGNX) announced the completion of dosing in the expanded Cohort 2 of its Phase I/II trial for RGX-111, an investigational gene therapy for severe Mucopolysaccharidosis Type I (MPS I). Eight patients have participated in this trial, aimed at evaluating RGX-111's safety and potential effectiveness. The company plans to manufacture commercial-grade material to further develop RGX-111. Interim trial updates are expected in the first half of 2023. RGX-111 leverages the NAV AAV9 vector to deliver the IDUA gene directly to the CNS.
REGENXBIO Inc. (Nasdaq: RGNX) announced its participation in two investor conferences. The first is the Piper Sandler 34th Annual Healthcare Conference on December 1, 2022, at 9:10 a.m. ET in New York, NY. The second event is the BofA Securities 2022 Virtual Biotech SMID Cap Conference, with a fireside chat on December 8, 2022, at 10:50 a.m. ET. Live webcasts can be accessed on REGENXBIO's website, with replays available for 30 days. The company is a leader in gene therapy, focusing on advancing its 5x'25 strategy to develop five AAV Therapeutics by 2025.
REGENXBIO (Nasdaq: RGNX) reported third-quarter financial results, highlighting a net loss of $75.5 million, compared to a $58.4 million loss in Q3 2021. Revenues decreased to $26.5 million, chiefly due to falling Zolgensma royalties. The company maintains a robust cash position of $617 million, expected to fund operations through 2025. Progress on the 5x25 strategy continues, with RGX-314 trials for wet AMD and diabetic retinopathy demonstrating positive interim data. BLA filings for RGX-314 and RGX-121 are anticipated in 2024, affirming ongoing commitment to gene therapies.
REGENXBIO (Nasdaq: RGNX) announced positive interim results from the Phase II ALTITUDE trial of RGX-314, aimed at treating diabetic retinopathy (DR) via suprachoroidal delivery. The results showed that RGX-314 was well tolerated in 50 patients across the first three cohorts, with no serious drug-related adverse events. Clinically meaningful improvements were noted, including 54% of patients achieving any DRSS improvement, compared to 20% in the control group. The trial will expand to include a third dose level and new cohorts, with the goal of further evaluating RGX-314's potential.
REGENXBIO Inc. (Nasdaq: RGNX) has announced its participation in two upcoming investor conferences. The first is the Credit Suisse 31st Annual Healthcare Conference on November 8, 2022, located at Terranea Resort, Rancho Palos Verdes, CA. The second is the Barclays Sixth Annual Gene Editing/Therapy Summit on November 14, 2022, where REGENXBIO will discuss AAV Gene Therapy's current status and future direction at 2:50 p.m. ET. The company focuses on advancing AAV Therapeutics with its proprietary NAV Technology Platform, emphasizing its goal of progressing five products to pivotal-stage or commercial status by 2025.
REGENXBIO Inc. (Nasdaq: RGNX) announced it will present data from the Phase II ALTITUDE trial for RGX-314 aimed at treating diabetic retinopathy at the Retina Society Annual Scientific Meeting from November 2-5, 2022, in Pasadena, CA. A conference call is scheduled for November 3, 2022, at 8:30 a.m. ET to discuss Q3 2022 financial results and operational highlights. The presentation will be led by Dr. Lejla Vajzovic of Duke University on November 2, 2022, at 4:41 p.m. PT, and will be accessible on REGENXBIO's website.
REGENXBIO Inc. (Nasdaq: RGNX) announced positive interim data from the Phase II AAVIATE trial of RGX-314 for wet AMD, showing significant reductions in treatment burden. The trial reported an 85% reduction at the third dose level, with 67% of patients in Cohort 4 injection-free. Safety profiles were favorable with no serious drug-related adverse events. Positive long-term data from Phase I/IIa studies showed RGX-314 remains well tolerated and supports a BLA submission expected in 2024. Upcoming data from the ALTITUDE trial for diabetic retinopathy is anticipated at the Retina Society meeting in November.
REGENXBIO Inc. (Nasdaq: RGNX) announced its participation in Chardan's 6th Annual Genetic Medicines Conference on October 4, 2022, at 1:30 p.m. ET in New York, NY. The company will conduct a fireside chat, accessible via a live webcast on its website, with an archived version available for about 30 days post-event. REGENXBIO is recognized for its gene therapy advancements, utilizing its proprietary NAV Technology Platform comprising over 100 AAV vectors. The firm aims to progress five gene therapy programs into pivotal stages or commercial products by 2025.
REGENXBIO Inc. (Nasdaq: RGNX) will present at the American Academy of Ophthalmology 2022 Annual Meeting from September 30 to October 3 in Chicago, IL. Key presentations include updates on RGX-314, an investigational gene therapy for chronic retinal conditions, including wet age-related macular degeneration and diabetic retinopathy. The company will also host a conference call on October 3 at 8:30 a.m. ET to discuss interim data from its ongoing Phase II AAVIATE trial.
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