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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 (Nasdaq: RGNX) will host a conference call on November 4, 2020, at 4:30 p.m. ET to discuss its financial results for the third quarter ended September 30, 2020. The company aims to enhance lives through gene therapy using its proprietary NAV Technology Platform. Investors can access the call by phone or through a webcast available for 30 days. The platform includes over 100 novel AAV vectors, showcasing the company's commitment to developing a diverse pipeline of gene therapy candidates.
REGENXBIO Inc. (Nasdaq: RGNX) announced it will receive an $80 million milestone payment from Novartis AG due to Zolgensma achieving $1 billion in net sales. This revenue is expected to be recognized in Q3 2020. Zolgensma, a transformative gene therapy for spinal muscular atrophy, has positively impacted over 700 patients. Since its FDA approval in May 2019, REGENXBIO has received over $140 million from related royalties and milestones.
REGENXBIO Inc. (Nasdaq: RGNX) announced an expansion of its RGX-121 program for Mucopolysaccharidosis Type II (MPS II), aiming to understand the disease's neurodegenerative effects better. RGX-121, a potential gene therapy, delivers the enzyme iduronate-2-sulfatase to the central nervous system. An ongoing Phase I/II trial has dosed six patients with no serious adverse events reported. Additionally, the FDA has cleared a new IND for a trial involving pediatric patients aged 5-18. A natural history study will explore neurocognitive development in young MPS II patients.
REGENXBIO Inc. (Nasdaq: RGNX), a clinical-stage biotechnology company, announced its participation in three upcoming virtual investor conferences. These include the SVB Leerink CybeRx Series on October 1, 2020, the Jefferies Virtual Gene Therapy/Editing Summit on October 2, 2020, and the Chardan Virtual 4th Annual Genetic Medicines Conference on October 6, 2020, featuring a fireside chat at 10:30 a.m. ET. Investors can access a live webcast and a 30-day archived replay on the company's website.
REGENXBIO Inc. (Nasdaq: RGNX) has announced the dosing of its first patient in the AAVIATE trial, a Phase II study evaluating the suprachoroidal delivery of RGX-314 for treating wet age-related macular degeneration (wet AMD). This trial marks a significant milestone as it is the first to assess this delivery method for gene therapy. Interim data is expected by the end of 2020. RGX-314 aims to serve as a one-time treatment targeting the VEGF pathway to inhibit leaky blood vessel growth, addressing a condition affecting millions.
REGENXBIO Inc. (Nasdaq: RGNX), a leader in gene therapy, announced its participation in two September investor conferences. The events will be held virtually, starting with Citi's 15th Annual BioPharma Virtual Conference on September 9, 2020, followed by the Morgan Stanley 18th Annual Global Healthcare Conference on September 15, 2020, including a fireside chat at 8:45 a.m. ET. Interested investors can access a live webcast on REGENXBIO's website, with a replay available for 30 days post-event.
REGENXBIO Inc. (Nasdaq: RGNX) announced the FDA's clearance of its Investigational New Drug (IND) application for RGX-314 to treat diabetic retinopathy (DR). This IND activation allows the company to commence a Phase II trial named ALTITUDE in the second half of 2020, targeting around 40 patients. The primary endpoint is to evaluate patient improvement in DR severity after 48 weeks. This advancement is significant as DR is a major cause of blindness, affecting approximately 8 million people in the U.S.
REGENXBIO Inc. (Nasdaq: RGNX) reported its Q2 2020 financial results, noting significant advancements in its gene therapy programs amid the COVID-19 pandemic. Key highlights include plans to launch a pivotal program for RGX-314 in treating wet AMD by year-end, and the completion of patient dosing for RGX-121 targeting MPS II. Revenues surged to $16.6 million, attributed mainly to increased royalties from Zolgensma sales. However, the company incurred a net loss of $33.8 million. Cash reserves stood at $339.2 million, expected to fund development into 2022.
On August 4, 2020, REGENXBIO announced positive updates for RGX-314, a potential one-time treatment for wet AMD, following the Phase I/IIa trial results. The data showed RGX-314 was well-tolerated in 42 patients, with a significant reduction in anti-VEGF injections—61% and 85% in Cohorts 4 and 5, respectively. The pivotal program for subretinal delivery is set to begin by year-end 2020. Additionally, a Phase II trial (AAVIATE) utilizing suprachoroidal delivery is expected to enroll patients in Q3 2020. Interim data from the AAVIATE trial will be reported by late 2020.
REGENXBIO Inc. (Nasdaq: RGNX), a biotechnology firm focused on gene therapy, will host a conference call on August 6, 2020, at 4:30 p.m. ET, to discuss its financial results for Q2 2020, ending June 30, 2020.
The call will provide insights into the company's operational highlights and financial performance. Investors can participate by calling (800) 708-4539 (domestic) or (800) 619-6396 (international), or by accessing a live or recorded webcast on the REGENXBIO website.
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