Welcome to our dedicated page for Regenxbio news (Ticker: RGNX), a resource for investors and traders seeking the latest updates and insights on Regenxbio stock.
Regenxbio Inc (RGNX) is a clinical-stage biotechnology leader advancing novel AAV gene therapies through its proprietary NAV® technology platform. This page provides investors and stakeholders with centralized access to the company’s latest press releases, clinical trial updates, and strategic developments.
Track critical updates across Regenxbio’s pipeline, including progress on RGX-202 for Duchenne muscular dystrophy, RGX-121 for Hunter syndrome, and ABBV-RGX-314 for retinal diseases. Stay informed about regulatory milestones, manufacturing advancements, and partnership announcements with entities like AbbVie.
All content is sourced directly from Regenxbio’s official communications, ensuring accuracy and timeliness. Bookmark this page for streamlined access to essential updates on one-time gene therapies targeting rare genetic disorders.
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.
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.
