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 ...

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 ...

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97% reduction in treatment burden at nine months after treatment with ABBV-RGX-314Data consistent with that from multiple previous studies demonstrating favorable safety and efficacy profile Well tolerated with zero cases of intraocular inflammation in a setting of no prophylactic steroids Data highlight the potential of ABBV-RGX-314 to treat both eyes in wet AMD ROCKVILLE, Md., Oct. 21, 2024 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced positive data from the Phase II fellow eye sub-study e ...

ROCKVILLE, Md., Oct. 18, 2024 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that it will present new interim data evaluating subretinal delivery of ABBV-RGX-314 in patients with bilateral wet age-related macular degeneration (wet AMD) at the American Academy of Ophthalmology 2024 annual meeting. ABBV-RGX-314 is an investigational one-time AAV Therapeutic being developed in collaboration with AbbVie for the treatment of wet AMD, diabetic retinopathy and other additional chronic retinal condit ...

ROCKVILLE, Md., Sept. 23, 2024 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced it will participate in Chardan's 8th Annual Genetic Medicines Conference on Monday, September 30, 2024. Chardan's 8th Annual Genetic Medicines Conference Fireside Chat: Monday, September 30, 2024 at 8:30 a.m. ET Location: New York, NY A live webcast of the fireside chat can be accessed in the Investors section of REGENXBIO's website at www.regenxbio.com. An archived replay of the webcast will be available for approx ...