ROCKVILLE, Md., Feb. 1, 2021 /PRNewswire/ -- REGENXBIO Inc.
(Nasdaq: RGNX) today announced that three oral and nine poster
presentations will be presented at the 17th Annual
WORLDSymposium™, taking place virtually from February 8 through 12, 2021. The oral
presentations include interim results from the Phase I/II clinical
trial of RGX-121 for the treatment of mucopolysaccharidosis type II
(MPS II).
The oral presentations will be presented as follows:
Abstract Title: Comparative systemic and neurologic
effectiveness of intravenous and intrathecal AAV9 delivered
individually or combined in a murine model of mucopolysaccharidosis
type I
Presenter: Lalitha Belur,
Ph.D., Assistant Professor Genetics, Cell Bio at University of Minnesota
Date/Time: Monday, February 8,
2021, 10:06 a.m. ET
Abstract Title: Intracisternal administration of AAV9
gene therapies to target the central nervous system
Presenter: Bryan A. Pukenas,
M.D., Assistant Professor of Radiology at Hospital of the
University of Pennsylvania
Date/Time: Tuesday, February 9,
2021, 11:36 a.m. ET
Abstract Title: RGX-121 gene therapy for severe
mucopolysaccharidosis type II (MPS II): Interim results of an
ongoing first in human trial
Presenter: Marie-Laure
Nevoret, M.D., Senior Clinical Development Lead at
REGENXBIO
Date/Time: Thursday, February 11,
2021, 9:42 a.m. ET
The poster presentations will be presented as follows:
Abstract Title: Subretinal injection of RGX-381 to
cynomolgus monkeys leads to supraphysiological levels of TPP1 in
the eye (poster #24)
Presenter: Nicholas Buss, Ph.D., Senior Director,
Preclinical Development at REGENXBIO
Date/Time: Tuesday, February 9,
2021, from 2:30 to 3:30 p.m.
ET
Abstract Title: Intracisternal administration of AAV9
gene therapies to target the central nervous system (poster
#207)
Presenter: Bryan A. Pukenas,
M.D., Assistant Professor of Radiology at Hospital of the
University of Pennsylvania
Date/Time: Tuesday, February 9,
2021, from 2:30 to 3:30 p.m.
ET
Abstract Title: First in-human intracisternal dosing of
RGX-111 (adeno-associated virus 9/human α-Liduronidase) for a
20-month-old child with mucopolysaccharidosis type I (MPS I): 1
year follow-up (poster #264)
Presenter: Raymond Wang, M.D., Biochemical Genetics
Specialist at CHOC Children's Hospital
Date/Time: Tuesday, February 9,
2021, from 2:30 to 3:30 p.m.
ET
Abstract Title: RGX-121 gene therapy for severe
mucopolysaccharidosis type II (MPS II): A clinical program to
address central nervous system manifestations (poster #45)
Presenter: Kirsten Cowley, Director, Clinical
Operations at REGENXBIO Inc.
Date/Time: Wednesday, February 10,
2021, from 2:30 to 3:30 p.m.
ET
Abstract Title: Clinical and numerical presentation of
neurocognitive assessments for MPS II patients using the Bayley
Scales of Infant Development - version 3 (BSID-III) (poster
#34)
Presenter: Yoonjin Cho, Ph.D., Director, Biostatistics
at REGENXBIO Inc.
Date/Time: Thursday, February 11,
2021, from 2:30 to 3:30 p.m.
ET
Abstract Title: RGX-121 gene therapy for severe
mucopolysaccharidosis type II (MPS II): Interim results of an
ongoing first in human trial (poster #171)
Presenter: Marie-Laure Nevoret, M.D., Senior Clinical
Development Lead at REGENXBIO Inc.
Date/Time: Thursday, February 11,
2021, from 2:30 to 3:30 p.m.
ET
Abstract Title: Characterizing expressive language skills
in children with late infantile neuronal ceroid lipofuscinosis type
2 (CLN2): The caregiver perspective (poster #198)
Presenter: Dawn Phillips, Ph.D., Director, Clinical
Scientist, Outcomes Research at REGENXBIO Inc.
Date/Time: Thursday, February 11,
2021, from 2:30 to 3:30 p.m.
ET
Abstract Title: Characterizing visual function in
children with late infantile neuronal ceroid lipofuscinosis type 2
(CLN2): The caregiver perspective (poster #199)
Presenter: Dawn Phillips, Ph.D., Director, Clinical
Scientist, Outcomes Research at REGENXBIO Inc.
Date/Time: Thursday, February 11,
2021, from 2:30 to 3:30 p.m.
ET
Abstract Title: New research examines the evolution of
data sharing practices in natural history studies and patient
registries among patient advocate leaders, industry and academia
(poster #LB-12)
Presenter: Vivian Fernandez, Senior Director of Patient
Advocacy at REGENXBIO Inc.
Date/Time: Friday, February 12,
2021, from 2:30 to 3:30 p.m.
ET
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, 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 in multiple therapeutic areas.
Contacts:
Tricia
Truehart
Investor Relations and Corporate Communications
347-926-7709
ttruehart@regenxbio.com
Investors:
Brendan Burns, 212-600-1902
brendan@argotpartners.com
Media:
David Rosen, 212-600-1902
david.rosen@argotpartners.com
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SOURCE REGENXBIO Inc.