REGENXBIO Announces Presentations at the Society for the Study of Inborn Errors of Metabolism Annual Symposium 2023
Presentations:
Title: RGX-121: An investigational gene therapy for the treatment of neuronopathic mucopolysaccharidosis type II (MPS II), an interim analysis of data from the first-in-human study
Presenter:
Date/Time:
Title: RGX-111: An investigational gene therapy for the treatment of severe mucopolysaccharidosis type I (MPS I): Interim analysis data from first-in-human study
Presenter: Raymond Wang, M.D.,
Date/Time: Wednesday, August 30, 2023,
Posters:
Title: First-in-human intracisternal dosing of RGX-181 (adeno-associated virus 9 / human tripeptidyl peptidase 1) for a 5-year-old child with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2): 6-month follow up
Authors: Carolina Fischinger De Souza, M.D., Ph.D., Hospital de Clinicas de
Date/Time:
Title: Daily living skills on the Vineland Adaptive Behavioral Scale version 2 (VABS-II) in neuronopathic mucopolysaccharidosis type II (MPS II)
Authors: Michelle Wood,
Date/Time: Wednesday, August 30, 2023,
About
REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. REGENXBIO's NAV Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO and its third-party NAV Technology Platform Licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates, including late-stage and commercial programs, in multiple therapeutic areas. REGENXBIO is committed to a "5x'25" strategy to progress five AAV Therapeutics from our internal pipeline and licensed programs into pivotal-stage or commercial products by 2025.
Contacts:
Investors:
ICR Westwicke
339-970-2843
chris.brinzey@westwicke.com
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