- Pivotal topline results from the CAMPSIITE® trial
of RGX-121 for the treatment of MPS II to be presented
- Conference call Wednesday, February 7, 4:30 p.m.
ET
ROCKVILLE, Md., Jan. 31,
2024 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX)
today announced presentations on its program for the treatment of
mucopolysaccharidosis type II (MPS II), also known as Hunter
syndrome, will be shared at the 20th Annual
WORLDSymposium™ 2024, taking place in San Diego, CA February
3-9, 2024.
The presentations include the topline results from the pivotal
phase of the Phase I/II/III CAMPSIITE® trial of RGX-121
for the treatment of MPS II.
The oral presentations will be presented as follows:
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: Wednesday, February 7, 2024; 8:36 a.m. PT
REGENXBIO will also present at the 3rd Annual Robert
J. Gorlin Symposium on Tuesday, February 6,
2024 to discuss intracisternal administration of
investigational AAV9 gene therapies to target the central nervous
system in pediatric lysosomal disorders.
The poster presentations will be presented as follows:
Abstract Title: Audiology assessment of participants in
CAMPSIITE®, a phase I/II/III study of RGX-121 in
neuronopathic MPS II (abstract 037)
Presenter: Nidal Boulos,
Ph.D., Director, Clinical Science, REGENXBIO
Date/Time: Thursday, February 8, 2024; 3 p.m. PT
Abstract Title: Caregiver Perspectives on their MPS II
Journey (abstract 422)
Presenter: Vivian Fernandez,
Executive Director, Patient Advocacy, REGENXBIO
Date/Time: Thursday, February 8, 2024; 3 p.m. PT
Conference Call
REGENXBIO will host a conference call
Wednesday, February 7 at 4:30 p.m. ET with Dr. Harmatz and Raymond Wang, M.D., Children's Hospital of
Orange County to discuss the
CAMPSIITE trial pivotal phase topline results and the expedited
plan for filing a Biologics License Application using the
accelerated approval pathway in 2024.
Listeners can register for the webcast via this link. Analysts
wishing to participate in the question and answer session should
use this link. A copy of the slides being presented will be
available via the Company's investor website. Those who plan on
participating are advised to join 15 minutes prior to the start
time. A replay of the webcast will also be available via the
Company's investor website approximately two hours after the call's
conclusion.
About REGENXBIO Inc.
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 and AAV9. 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:
Dana Cormack
Corporate Communications
dcormack@regenxbio.com
Investors:
Chris Brinzey
ICR Westwicke
339-970-2843
chris.brinzey@westwicke.com
View original content to download
multimedia:https://www.prnewswire.com/news-releases/regenxbio-announces-presentations-at-the-20th-annual-worldsymposium-2024-302048903.html
SOURCE REGENXBIO Inc.