Goldman Sachs downgraded Regenxbio Inc RGNX, citing gene therapy’s appeal for wet AMD and diabetic retinopathy is uncertain, as most patients are elderly and newer anti-VEGF treatments offer longer-lasting effects at lower costs.Analyst Paul Choi writes that AbbVie Inc partnered ABBV-RGX-314 faces growing competition, with its invasive delivery method making it less favorable than rival treatments.Data from the ATMOSPHERE and ASCENT pivotal trials evaluating the safety and efficacy of the subretinal deliver ...

Shares of Regenxbio (RGNX) have gained 9.3% over the past four weeks to close the last trading session at $8.21, but there could still be a solid upside left in the stock if short-term price targets of Wall Street analysts are any indication. Going by the price targets, the mean estimate of $35.55 indicates a potential upside of 333%.The average comprises 11 short-term price targets ranging from a low of $22 to a high of $52, with a standard deviation of $10.42. While the lowest estimate indicates an increa ...

ROCKVILLE, Md., Jan. 30, 2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced data from its RGX-121 (clemidsogene lanparvovec) program for the treatment of mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome, will be shared at the 21st Annual WORLDSymposium™ 2025, taking place in San Diego, CA February 3-7, 2025. The following presentations include an encore of the topline results from the pivotal phase of the Phase I/II/III CAMPSIITE® trial of RGX-121. Abstract Title: Audiol ...

Regenxbio (RGNX) closed the last trading session at $7.95, gaining 1% over the past four weeks, but there could be plenty of upside left in the stock if short-term price targets set by Wall Street analysts are any guide. The mean price target of $36.36 indicates a 357.4% upside potential.The average comprises 11 short-term price targets ranging from a low of $22 to a high of $52, with a standard deviation of $9.66. While the lowest estimate indicates an increase of 176.7% from the current price level, the m ...

REGENXBIO to receive $110 million upfront, potential milestone payments of up to $700 million and meaningful double-digit royalties on net sales, and lead manufacturing Nippon Shinyaku to lead commercialization of first potential gene therapies for Mucopolysaccharidosis II (MPS II) and Mucopolysaccharidosis I (MPS I) in U.S. and Asia REGENXBIO retains rights to RGX-121 Priority Review Voucher (PRV) with potential accelerated approval expected in 2025; rolling BLA submission underway ROCKVILLE, Md., Jan. 1 ...

Core Insights - REGENXBIO Inc. has achieved alignment with the FDA on the AFFINITY DUCHENNE® pivotal program for RGX-202, a gene therapy for Duchenne muscular dystrophy, with a Biologics License Application (BLA) expected in 2026 [1][2] - The pivotal trial has commenced, enrolling ambulatory patients aged 1 and above, with the first patient already dosed [1][2] - Positive Phase I/II data indicate that RGX-202 recipients are exceeding external natural history benchmarks for clinical outcomes, showing functional improvements at both dose levels [1][3][4] Functional Data - Positive functional results were reported from the first five participants in the Phase I/II trial, with 12-month data from three patients at dose level 1 and nine-month data from two patients at dose level 2 [3][4] - All five participants demonstrated stable or improved function on the North Star Ambulatory Assessment (NSAA) and timed function tests, outperforming external natural history controls [4][5] Pivotal Dose Functional Data - Participants at the pivotal dose level showed a mean NSAA score improvement of 5.5 points at nine months, exceeding external natural history controls [5] - Dose level 1 participants also demonstrated improved performance, surpassing minimal clinically important difference (MCID) benchmarks at 12 months [5] Biomarker Data - New biomarker data confirms high expression and localization of RGX-202 microdystrophin in muscle tissue, with levels reported as the highest across approved or investigational gene therapies [6][7] - RGX-202 microdystrophin expression results in ambulatory patients aged 8 and above show significant levels, indicating effective targeting of muscle [6][7] Safety and Tolerability Data - As of November 1, 2024, RGX-202 has shown a favorable safety profile with no serious adverse events or adverse events of special interest reported [7][8] - Common drug-related adverse events included nausea, vomiting, and fatigue, all of which resolved and are typical for gene therapy administration [7][8] Pivotal Study Design - The pivotal trial is designed to support a BLA submission using the accelerated approval pathway, with a primary endpoint focused on microdystrophin expression and secondary endpoints on functional tests [11][12] - The trial will evaluate approximately 30 ambulatory patients aged 1 and older, with specific assessments for younger patients [11][12]

-       Event will feature Aravindhan Veerapandiyan, M.D., principal investigator of the AFFINITY DUCHENNE® trial and Michael Kelly, PhD, Chief Scientific Officer of CureDuchenneROCKVILLE, Md., Nov. 14, 2024 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that it will host a webcast to discuss the AFFINITY DUCHENNE® pivotal program and new clinical data, including the first functional data from the ongoing Phase I/II study of RGX-202, the company's next-generation gene therapy for the treatmen ...

Call Start: 16:30 January 1, 0000 5:24 PM ET REGENXBIO Inc. (NASDAQ:RGNX) Q3 2024 Earnings Call November 6, 2024 16:30 ET Company Participants Patrick Christmas - Chief Legal Officer Curran Simpson - President and Chief Executive Officer Mitchell Chan - Chief Financial Officer Steve Pakola - Chief Medical Officer Conference Call Participants Gospel Enyindah-Asonye - Morgan Stanley Mani Foroohar - Leerink Partners Jack Padovano - Stifel Luca Issi - RBC Capital Markets Paul Choi - Goldman Sachs Daniil Gatauli ...

Regenxbio (RGNX) came out with a quarterly loss of $1.17 per share versus the Zacks Consensus Estimate of a loss of $1.16. This compares to loss of $1.41 per share a year ago. These figures are adjusted for non-recurring items.This quarterly report represents an earnings surprise of -0.86%. A quarter ago, it was expected that this biotechnology company would post a loss of $1.29 per share when it actually produced a loss of $1.05, delivering a surprise of 18.60%.Over the last four quarters, the company has ...

Table of Contents Securities registered pursuant to Section 12(b) of the Act: UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended September 30, 2024 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from ___ to ___ Commission File Number 001-37553 REGENXBIO Inc. (Exact Name of Re ...