Sarepta Therapeutics to Present Results from its Gene Therapy and RNA Platforms at the 2021 Annual MDA Clinical and Scientifi...
March 15 2021 - 8:30AM
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision
genetic medicine for rare diseases, will present at the Muscular
Dystrophy Association (MDA) Annual Clinical and Scientific
Conference, which will take place virtually March 15-18, 2021.
Among the research that will be presented:
- New,
two- and one-year data including expression data from muscle
biopsies taken two years post-treatment, from Study 9003-101 of
SRP-9003, an investigational gene therapy for the treatment of
limb-girdle muscular dystrophy (LGMD) type 2E/R4;
- Data
from Part 1 of Study 9001-102, an ongoing clinical trial of
SRP-9001, Sarepta’s investigational gene therapy for Duchenne
muscular dystrophy and pre-clinical approaches to the challenge of
pre-existing antibodies; and,
- An
analysis of time to loss of ambulation in patients taking
eteplirsen, a phosphorodiamidate morpholino oligomer (PMO) for the
treatment of Duchenne, compared to standard of care.
All posters are available on-demand throughout
the Congress beginning on Monday, March 15, 2021 at 6:00 a.m. ET.
Podium presentations will take place on Thursday, March 18, 2021.
The full MDA 2021 Virtual Congress program is available here:
https://mdaconference.org.
Podium Presentations:
Title |
Program |
Date, Time |
Micro-dystrophin Gene Therapy Delivery and Therapeutic Plasma
Exchange in Non-Human Primates |
SRP-9001 |
Thurs., March 18, 2021 10:30-10:45 AM ET |
A Randomized, Double-Blind, Placebo-Controlled, Gene-Delivery
Clinical Trial of rAAVrh74.MHCK7.micro-dystrophin for Duchenne
Muscular Dystrophy |
SRP-9001 |
Thurs., March 18, 2021 3:30-3:45 PM ET |
Safety, β-sarcoglycan Expression, and Functional Outcomes
from Systemic Gene Transfer of rAAVrh74.MHCK7.SGCB in
Limb Girdle Muscular Dystrophy Type 2E/R4 |
SRP-9003 |
Thurs., March 18, 2021 4:30-4:45 PM ET |
Delay in Duchenne Muscular Dystrophy Progression with Eteplirsen:
Longer Time to Loss of Ambulation Versus Standard of Care |
Eteplirsen |
Thurs., March 18, 2021 5:45-6:00 PM ET |
Poster Presentations:
Poster # |
Title |
6 |
Biological Efficacy of the Peptide-Conjugated Phosphorodiamidate
Morpholino Oligomer SRP-5051 in Preclinical Models of Duchenne
Muscular Dystrophy |
54 |
Casimersen Treatment in Eligible Patients with Duchenne Muscular
Dystrophy: Safety, Tolerability, and Pharmacokinetics Over 144
Weeks of Treatment |
90 |
Patterns of Clinical Progression Among Patients with Autosomal
Recessive Limb-Girdle Muscular Dystrophy (LGMDR): A Systematic
Review |
92 |
Progression to Loss of Ambulation (LOA) Among Patients with
Autosomal Recessive Limb-Girdle Muscular Dystrophy (LGMDR): A
Systematic Review |
104 |
Health-related quality of life (HRQoL) Associated with Duchenne
Muscular Dystrophy (DMD): A Study Using the Health Utilities Index
Mark 3 (HUI3) |
113 |
NorthStar Ambulatory Assessment (NSAA) and Health Utilities Index
(HUI) Scores are Weakly Correlated Among Boys with Duchenne
Muscular Dystrophy (DMD) |
Presentations will be archived on the events and
presentations page in the Investor Relations section of
www.sarepta.com for one year following their presentation at
MDA.
About Sarepta
TherapeuticsAt Sarepta, we are leading a revolution in
precision genetic medicine and every day is an opportunity to
change the lives of people living with rare disease. The Company
has built an impressive position in Duchenne muscular dystrophy
(DMD) and in gene therapies for limb-girdle muscular dystrophies
(LGMDs), mucopolysaccharidosis type IIIA, Charcot-Marie-Tooth
(CMT), and other CNS-related disorders, with more than 40 programs
in various stages of development. The Company’s programs and
research focus span several therapeutic modalities, including RNA,
gene therapy and gene editing. For more information, please
visit www.sarepta.com or follow us on Twitter, LinkedIn,
Instagram and Facebook.
Internet Posting of
Information
We routinely post information that may be
important to investors in the 'For Investors' section of our
website at www.sarepta.com. We encourage investors and
potential investors to consult our website regularly for important
information about us.
Source: Sarepta Therapeutics, Inc.
Sarepta Therapeutics, Inc.
Investors:Ian Estepan, 617-274-4052, iestepan@sarepta.com
Media:Tracy Sorrentino, 617-301-8566, tsorrentino@sarepta.com
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