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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) will host a conference call on May 3, 2023, at 4:30 p.m. ET to discuss its financial results for the quarter ending March 31, 2023, along with recent operational highlights. Participants can register for the webcast through the provided links. The company is known for its NAV Technology Platform, which includes over 100 novel AAV vectors and is utilized in developing a variety of therapeutic programs. REGENXBIO aims to achieve its "5x'25" strategy, which targets advancing five AAV therapeutics into pivotal or commercial stages by 2025.
REGENXBIO Inc. (Nasdaq: RGNX) announced it will participate in a fireside chat at Chardan's 7th Annual Genetic Medicines and Cell Therapy Manufacturing Summit on April 25, 2023, at 9:30 a.m. ET. The conference will occur in a virtual meeting format, with a live webcast accessible via the Investors section of REGENXBIO's website. An archived replay will be available for about 30 days post-event. REGENXBIO focuses on gene therapy, employing its NAV Technology Platform, which includes exclusive rights to over 100 AAV vectors. The company aims to progress five AAV Therapeutics from internal and licensed programs into pivotal or commercial products by 2025.
REGENXBIO announced that the FDA has granted Fast Track designation for RGX-202, a potential one-time gene therapy targeting Duchenne muscular dystrophy. This designation allows faster development and review for therapies addressing serious conditions. RGX-202 utilizes a novel microdystrophin and REGENXBIO's NAV AAV8 vector, aimed at improving muscle resistance. The Phase I/II AFFINITY DUCHENNE trial is currently active, recruiting pediatric patients, with initial data expected in late 2023. The company is leveraging its cGMP facility for clinical supply, reinforcing its commitment to expedite development. RGX-202 has also received Orphan Drug and Rare Pediatric Disease designations from the FDA, highlighting its potential value in addressing unmet medical needs.
REGENXBIO (Nasdaq: RGNX) announced its participation in two key investor conferences in March 2023. The Barclays Global Healthcare Conference is scheduled for March 15 at 5:00 p.m. ET in Miami, FL, featuring a fireside chat. Additionally, the company will participate in the BMO Biopharma Spotlight Series on March 28 at 9:00 a.m. ET, focusing on novel genetic medicine delivery technologies in a virtual format. Investors can access a live webcast of the Barclays event on the REGENXBIO website, with an archived replay available for 30 days following the presentation.
REGENXBIO (Nasdaq: RGNX) reported Q4 and full-year 2022 results, showing revenues of $31.3 million for Q4 and $112.7 million for the year, a decrease from $398.7 million and $470.3 million in 2021, primarily due to reduced collaboration revenues from AbbVie. The net loss for Q4 was $59.9 million, compared to a net income of $294.0 million in Q4 2021. The cash position stood at $565.2 million, funding operations into 2025. Several pivotal trials, including RGX-314 for wet AMD and diabetic retinopathy, remain ongoing, with the anticipated BLA filing by 2024. The company aims to advance five AAV Therapeutics by 2025 under its '5x'25 strategy.
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