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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) announced a proposed underwritten public offering of $175 million in common stock, subject to market conditions. The offering may include an additional 15% of shares at the underwriters' option. BofA Securities, Morgan Stanley, and Barclays are managing the offering. This action follows a shelf registration statement filed with the SEC in August 2018. The funds raised will likely support REGENXBIO's gene therapy initiatives based on its proprietary NAV® Technology Platform, which includes over 100 novel AAV vectors.
REGENXBIO Inc. (Nasdaq: RGNX) has announced a significant advancement in its gene therapy programs. The pivotal program for RGX-314, aimed at treating wet age-related macular degeneration (wet AMD), is active with ongoing patient screening. Two randomized trials will enroll approximately 700 patients, targeting a Biologics License Application (BLA) submission in 2024. Additionally, REGENXBIO introduced RGX-202, a promising one-time gene therapy for Duchenne Muscular Dystrophy (DMD), with plans to submit an Investigational New Drug (IND) application in mid-2021.
REGENXBIO Inc. (Nasdaq: RGNX) announced an agreement to sell a portion of its royalty rights from Novartis Gene Therapies' Zolgensma for $200 million. This transaction provides non-dilutive capital to enhance REGENXBIO's gene therapy pipeline and internal manufacturing capabilities. The agreement includes an upfront payment with future royalty potential, capped at 1.3 times the purchase price until November 2024. Zolgensma is approved in multiple countries for Spinal Muscular Atrophy (SMA) treatment, marking a significant milestone in gene therapy.
REGENXBIO (Nasdaq: RGNX) announced that the first patient has been dosed in its ALTITUDE Phase II trial for RGX-314, aimed at treating diabetic retinopathy (DR) via suprachoroidal delivery. This trial evaluates RGX-314's efficacy and safety, potentially providing a one-time treatment option to replace frequent anti-VEGF injections. The study plans to enroll about 40 patients, comparing RGX-314 with an observational control. Initial data is expected in 2021. DR, a leading cause of vision loss in adults, affects approximately 8 million in the U.S.
REGENXBIO Inc. (Nasdaq: RGNX) reported positive interim data from Cohorts 1 and 2 of its Phase I/II trial for RGX-121, a gene therapy targeting Mucopolysaccharidosis Type II (MPS II) in patients up to 5 years old. As of November 13, 2020, RGX-121 showed a good safety profile with no serious adverse events in 8 patients. Notable reductions in heparan sulfate levels in CSF were observed, and neurocognitive development continued in Cohort 1 patients. REGENXBIO plans to initiate Cohort 3 with a higher dose in Q1 2021, with more data to be presented at the WORLD Symposium in early 2021.
REGENXBIO (Nasdaq: RGNX) announced the dosing of the first patient in the Phase I/II trial for RGX-111, targeting Mucopolysaccharidosis Type I (MPS I). This investigational gene therapy aims to deliver the IDUA gene to the central nervous system using the NAV AAV9 vector. MPS I is a rare genetic disorder affecting 1 in 100,000 births, leading to severe neurological symptoms. The trial, focused on safety and pharmacodynamics, will enroll up to five patients, marking a significant step in REGENXBIO's commitment to treating rare diseases, following its previous candidate RGX-121.
REGENXBIO Inc. (Nasdaq: RGNX) announced participation in two upcoming virtual investor conferences in December 2020. The company will host a pre-recorded fireside chat at the Piper Sandler 32nd Annual Virtual Healthcare Conference and a live webcast at the Evercore ISI 3rd Annual HealthCONx Conference on December 3, 2020, at 11:45 a.m. ET. The pre-recorded chat is now available on REGENXBIO's website for 30 days. REGENXBIO focuses on gene therapy through its proprietary NAV Technology Platform, featuring over 100 AAV vectors for various therapeutic applications.
REGENXBIO Inc. (Nasdaq: RGNX) announced two oral presentations at the AAO 2020 Annual Meeting (Nov 13-15) focused on their gene therapy for wet AMD. The first presentation will highlight the RGX-314 Phase I/II trial results, presented by Dr. Dante Pieramici. The second presentation, by Dr. Peter Campochiaro, will cover preclinical studies on suprachoroidal delivery of RGX-314. REGENXBIO aims to leverage its NAV Technology Platform for advancing gene therapy solutions across various therapeutic areas.
REGENXBIO Inc. (Nasdaq: RGNX) announces participation in upcoming virtual investor conferences. Key events include the 29th Annual Credit Suisse Virtual Healthcare Conference on November 12, 2020, at 8:45 a.m. ET, and the Barclays Gene Editing & Gene Therapy Summit on November 16, 2020, at 2:00 p.m. ET. Live webcasts will be available on REGENXBIO's website, with archived replays accessible for approximately 30 days post-presentation. The company focuses on gene therapy and has a broad pipeline utilizing its proprietary NAV Technology Platform.
REGENXBIO Inc. (RGNX) announced its Q3 2020 financial results, highlighting a significant revenue increase to $98.9 million, driven by Zolgensma royalties and an $80 million milestone payment. The company reported a net income of $8.8 million, reversing a loss from the previous year. Operational achievements include the initiation of Phase II trials for RGX-314 targeting wet AMD and diabetic retinopathy. Additionally, RGX-121's program expansion for MPS II patients was noted. Cash and equivalents stood at $289.8 million, ensuring funding through mid-2022.
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