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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 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.
REGENXBIO Inc. (Nasdaq: RGNX) has announced its participation in two key investor conferences:
- Morgan Stanley 20th Annual Global Healthcare Conference on September 14, 2022, at 2:55 p.m. ET in New York.
- UBS Biotech South Beach Conference on September 29, 2022, in Miami.
A live webcast of the Morgan Stanley conference will be available on REGENXBIO's website with a 30-day archived replay. The company is committed to advancing gene therapy through its proprietary NAV Technology Platform and aims to progress five AAV Therapeutics by 2025.
REGENXBIO (Nasdaq: RGNX) announced positive interim data for RGX-121, a gene therapy for Mucopolysaccharidosis Type II (MPS II), at the SSIEM Annual Symposium. The Phase I/II/III CAMPSIITE trial revealed significant median reductions in cerebrospinal fluid (CSF) glycosaminoglycans (GAGs) at 48 weeks, demonstrating a favorable safety profile with no serious drug-related adverse events. The company plans to file a Biologics License Application in 2024 via the accelerated approval pathway, aiming to address significant unmet needs in MPS II treatment.
REGENXBIO Inc. (Nasdaq: RGNX) announced key presentations at the Society for the Study of Inborn Errors of Metabolism Annual Symposium from August 30 to September 2, 2022, in Freiburg, Germany. The focus will be on new data from the Phase I/II/III CAMPSIITE™ trial of RGX-121, a gene therapy for Mucopolysaccharidosis Type II, and RGX-111 for Mucopolysaccharidosis Type I. Notable presentations include interim analyses by Dr. Roberto Giugliani and Dr. Raymond Wang, as well as a discussion on neurodevelopment in MPS II by Dr. Maria Escolar.
REGENXBIO Inc. (Nasdaq: RGNX) will present at the H.C. Wainwright 2nd Annual Ophthalmology Virtual Conference on August 17, 2022, at 11:00 a.m. ET. The panel will focus on Unique Delivery Technology Platforms for Better Compliance and Efficacy. A live webcast will be accessible on the company's website, followed by an archived replay for 30 days. REGENXBIO is a clinical-stage biotechnology company harnessing gene therapy through its proprietary NAV Technology Platform, which includes over 100 exclusive AAV vectors, supporting its goal of advancing five AAV Therapeutics by 2025.
REGENXBIO announced its Q2 2022 financial results, reporting revenues of $32.6 million, driven by Zolgensma royalty revenues, up from $22.0 million in Q2 2021. The net loss increased to $68.2 million, or $1.58 per share, compared to $57.6 million a year ago. Key developments include progress in the RGX-314 program for wet AMD and diabetic retinopathy, with BLA filings anticipated in 2024. The company holds $682 million in cash, providing operational runway into 2025, supporting its '5x'25 strategy to have five gene therapies on the market or in late-stage development by 2025.
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