REGENXBIO Announces Presentations of RGX-121 at the 21st Annual WORLDSymposium 2025
REGENXBIO (RGNX) has announced upcoming presentations of data from its RGX-121 (clemidsogene lanparvovec) program at the 21st Annual WORLDSymposium™ 2025 in San Diego, CA, scheduled for February 3-7, 2025. The presentations will feature topline results from the pivotal phase of the Phase I/II/III CAMPSIITE® trial for the treatment of mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome.
Two presentations are scheduled: an audiology assessment of CAMPSIITE® study participants on February 5, and an interim clinical study update on February 6. The presentations will be delivered by Nidal Boulos, Ph.D., Director of Clinical Science at REGENXBIO, and Paul Harmatz, M.D., from UCSF Benioff Children's Hospital, respectively.
REGENXBIO (RGNX) ha annunciato prossime presentazioni dei dati del suo programma RGX-121 (clemidsogene lanparvovec) al 21° Annuale WORLDSymposium™ 2025 a San Diego, CA, previsto per il 3-7 febbraio 2025. Le presentazioni presenteranno i risultati principali della fase cruciale dello studio CAMPSIITE® di fase I/II/III per il trattamento della mucopolisaccaridosi di tipo II (MPS II), nota anche come sindrome di Hunter.
Due presentazioni sono programmate: una valutazione audiologica dei partecipanti allo studio CAMPSIITE® il 5 febbraio e un aggiornamento intermedio dello studio clinico il 6 febbraio. Le presentazioni saranno tenute da Nidal Boulos, Ph.D., Direttore della Scienza Clinica di REGENXBIO, e Paul Harmatz, M.D., dell'UCSF Benioff Children's Hospital, rispettivamente.
REGENXBIO (RGNX) ha anunciado próximas presentaciones de datos de su programa RGX-121 (clemidsogene lanparvovec) en el 21° Simposio Anual WORLDSymposium™ 2025 en San Diego, CA, programado para el 3 al 7 de febrero de 2025. Las presentaciones presentarán los resultados principales de la fase decisiva del ensayo CAMPSIITE® de fase I/II/III para el tratamiento de mucopolisacaridosis tipo II (MPS II), también conocida como síndrome de Hunter.
Se programan dos presentaciones: una evaluación audiológica de los participantes del estudio CAMPSIITE® el 5 de febrero y una actualización intermedia del estudio clínico el 6 de febrero. Las presentaciones serán realizadas por Nidal Boulos, Ph.D., Director de Ciencias Clínicas en REGENXBIO, y Paul Harmatz, M.D., del Hospital Infantil UCSF Benioff, respectivamente.
REGENXBIO (RGNX)는 RGX-121 (clemidsogene lanparvovec) 프로그램의 데이터 발표를 2025년 2월 3일부터 7일까지 샌디에이고, CA에서 열리는 제21회 연례 WORLDSymposium™에 예정하고 있다고 발표했습니다. 발표에서는 뮤코다이소카라이드증 II형 (MPS II), 헌터 증후군으로 알려진 질환에 대한 1/2/3상 CAMPSIITE® 임상 시험의 중요한 단계 결과를 발표할 예정입니다.
두 개의 발표가 예정되어 있습니다: 2월 5일에는 CAMPSIITE® 연구 참가자에 대한 청각 평가가 있으며, 2월 6일에는 임상 연구 중간 업데이트가 있을 것입니다. 발표는 REGENXBIO의 임상 과학 이사인 Nidal Boulos 박사와 UCSF Benioff 아동병원의 Paul Harmatz 박사가 각각 진행할 예정입니다.
REGENXBIO (RGNX) a annoncé ses prochaines présentations de données issues de son programme RGX-121 (clemidsogene lanparvovec) lors du 21e Symposium Annuel WORLDSymposium™ 2025 à San Diego, CA, prévu du 3 au 7 février 2025. Les présentations mettront en lumière les résultats principaux de la phase décisive de l'essai CAMPSIITE® de phase I/II/III pour le traitement de la mucopolysaccharidose de type II (MPS II), également connue sous le nom de syndrome de Hunter.
Deux présentations sont programmées : une évaluation audiologique des participants à l'étude CAMPSIITE® le 5 février et une mise à jour intermédiaire de l'étude clinique le 6 février. Les présentations seront présentées par Nidal Boulos, Ph.D., Directeur de la Science Clinique chez REGENXBIO, et Paul Harmatz, M.D., de l'Hôpital pour Enfants UCSF Benioff, respectivement.
REGENXBIO (RGNX) hat die bevorstehenden Präsentationen von Daten aus seinem RGX-121 (clemidsogene lanparvovec) Programm beim 21. jährlichen WORLDSymposium™ 2025 in San Diego, CA, das für den 3. bis 7. Februar 2025 geplant ist, angekündigt. Die Präsentationen werden die wichtigsten Ergebnisse der entscheidenden Phase der Phase I/II/III CAMPSIITE®-Studie zur Behandlung der Mukopolysaccharidose Typ II (MPS II), auch bekannt als Hunter-Syndrom, präsentieren.
Zwei Präsentationen sind geplant: Eine audiologische Bewertung der CAMPSIITE®-Studienteilnehmer am 5. Februar und ein Zwischenbericht zur klinischen Studie am 6. Februar. Die Präsentationen werden von Nidal Boulos, Ph.D., Direktor der klinischen Wissenschaften bei REGENXBIO, und Paul Harmatz, M.D., vom UCSF Benioff Children's Hospital, gehalten.
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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.
Contacts:
Dana Cormack
Corporate Communications
dcormack@regenxbio.com
Investors:
George E. MacDougall
Investor Relations
IR@regenxbio.com
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SOURCE REGENXBIO Inc.
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