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REGENXBIO Announces Presentations of RGX-121 at the 21st Annual WORLDSymposium 2025

Thursday, January 30, 2025 BiopharmaWatch Research 2 min read
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Key Takeaway: REGENXBIO Inc. announced that it will present data on RGX-121, a gene therapy for mucopolysaccharidosis type II (MPS II), at the 21st Annual WORLDSymposium 2025 in San Diego. Presentations will include topline results from the pivotal phase of the CAMPSIITE trial, showcasing ongoing advancements in the treatment of this rare genetic disorder. The scheduled presentation times and the involvement of experts underline the significance of the findings in pursuing innovative treatments for MPS II.
Price reaction · baseline $7.52 (2025-01-29 close) · hit after-hours · clean, no other RGNX news in the window
day 0 close
+5.1%
day 1
+7.3%
day 3 · peak
+12.1%

Market Sentiment Analysis

POSITIVE FACTORS

  • REGENXBIO is presenting pivotal results from its RGX-121 trial at a significant conference.
  • The participation at the WORLDSymposium highlights the company's advancements in gene therapy for rare diseases.
  • RGX-121 shows promise for treating MPS II, a serious genetic disorder.

BiopharmaWatch Analysis

From our catalyst data and publicly available data · not financial advice
Best trade, last catalyst
+101%
120-day peak, hindsight
Typical move
8.4%
average across 5 past catalysts
Lead asset
RGX-202
Phase 2 · Duchenne Muscular Dystrophy

Full Press Release Details

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: Audiology assessment of participants in CAMPSIITE®, a phase I/II/III study of RGX-121 in neuronopathic MPS II (poster 35)
Presenter: Nidal Boulos, Ph.D., Director, Clinical Science, REGENXBIO
Date/Time: Wednesday, February 5, 2025; 3:30 p.m. PT
Abstract Title: CAMPSIITE® Phase I/II/III: An interim clinical study update of RGX121, an investigational gene therapy for the treatment of neuronopathic mucopolysaccharidosis type II (MPS II)
Presenter: Paul Harmatz, M.D., UCSF Benioff Children's Hospital
Date/Time: Thursday, February 6, 2025; 8:30 a.m. PT
ABOUT REGENXBIO Inc.
REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. Since its founding in 2009, REGENXBIO has pioneered the development of AAV Therapeutics, an innovative class of gene therapy medicines. REGENXBIO is advancing a pipeline of AAV Therapeutics for rare and retinal diseases, including RGX-202 for the treatment of Duchenne, ABBV-RGX-314 for the treatment of wet AMD and diabetic retinopathy, being developed in collaboration with AbbVie, and RGX 121 for the treatment of MPS II. Thousands of patients have been treated with REGENXBIO's AAV Therapeutic platform, including Novartis' ZOLGENSMA for children with spinal muscular atrophy. Designed to be one-time treatments, AAV Therapeutics have the potential to change the way healthcare is delivered for millions of people. For more information, please visit www.regenxbio.com.
Corporate Communications
George E. MacDougall

Frequently Asked Questions

What is RGX-121 used for?

RGX-121 is an investigational gene therapy for treating mucopolysaccharidosis type II.

When will RGX-121 data be presented?

Data on RGX-121 will be shared at the WORLDSymposium™ from February 3-7, 2025.

Who is presenting RGX-121 data?

Nidal Boulos, Ph.D., and Paul Harmatz, M.D., will present RGX-121 data.

What is REGENXBIO's focus?

REGENXBIO focuses on developing gene therapies for rare and retinal diseases.

What types of diseases does REGENXBIO target?

REGENXBIO targets rare and retinal diseases with its AAV Therapeutics platform.

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Tags: RGNX Viatris Investor Conferences Healthcare REGENXBIO RGX-121 WORLDSymposium MPS II gene therapy
Last updated: Jan 30, 2025

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$106M liquidity · $0M/mo burn
Low dilution risk
Lead asset
RGX-202Phase 2
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