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

1. RGNX to present RGX-121 data at WORLDSymposium 2025. 2. Topline results from pivotal CAMPSIITE trial will be shared. 3. RGX-121 targets MPS II, a rare genetic disorder. 4. REGENXBIO focuses on AAV gene therapy for various diseases.

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Why Bullish?

Positive clinical data presentations can boost investor confidence as seen with other biotech stocks that gained after favorable trial results.

How important is it?

This announcement relates directly to RGNX's pipeline, likely influencing market sentiment about its growth potential.

Why Long Term?

Long-term valuation can improve as more data supports RGX-121's efficacy, potentially leading to FDA approval.

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RGNXABBV
, /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, REGENXBIODate/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 HospitalDate/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. Contacts:Dana CormackCorporate Communications[email protected]  Investors:George E. MacDougallInvestor RelationsIR@regenxbio.com SOURCE REGENXBIO Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In

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