Abeona Therapeutics Announces Presentations and Posters at the 13th Annual WORLDSymposium(TM) 2017
(Thomson Reuters ONE) -
Multiple Oral Platform Presentations and Poster Sessions Highlighting Gene
Therapy Programs, Tuesday, February 14(th) through Thursday, February 16(th)
NEW YORK and CLEVELAND, Feb. 07, 2017 (GLOBE NEWSWIRE) -- Abeona Therapeutics
Inc. (NASDAQ:ABEO), a leading clinical-stage biopharmaceutical company focused
on developing therapies for life-threatening rare genetic diseases, today
announced that data on gene therapy programs for Sanfilippo syndrome Type A (MPS
IIIA), Infantile Batten Disease (CLN1) and Juvenile Batten Disease will be
highlighted at the upcoming 13(th) Annual WORLDSymposium(TM) 2017 lysosomal
storage disorders conference, February 13-17, San Diego, CA. Details for the
two oral presentations and three poster sessions are listed below.
Platform Presentations:
ABO-102 Phase 1/2 Clinical trial update - Sanfilippo syndrome Type A (MPS IIIA):
* A Phase 1/2 Clinical trial of systemic gene transfer of scAAV9.U1a.hSGSH for
MPS IIIA: Safety, tolerability, and preliminary evidence of biopotency
- Presenter: Kevin M. Flanigan, M.D., Principal Investigator and Director,
Research Institute of Nationwide Children's Hospital, Columbus, OH
- Thursday, February 16(th), 2017 at 1:30 pm PST
- Location: Seaport Ballroom E
- Clinical data highlights include: CSF and urinary heparan sulfate GAG
reduction, liver and spleen volume reduction, and neurological effects in
pediatric subjects through six months post-injection
ABO-202 - CLN1, Infantile Batten Disease (INCL):
* scAAV9 Gene Therapy is an Effective Treatment for CLN1-deficiency in the
Preclinical INCL Mouse Model
- Presenter: Alejandra J. Rozenberg, Ph.D., University of North Carolina
at Chapel Hill, Chapel Hill, NC.
- Wednesday, February 15(th), 2017 at 11:15 am PST
- Location: Seaport Ballroom E
Poster Sessions:
ABO-102 Phase 1/2 Clinical trial update - Sanfilippo syndrome Type A:
* Systemic gene transfer of scAAV9.U1a.hSGSH for MPS IIIA: tolerability and
preliminary evidence for a biochemical effect - Kevin M. Flanigan, M.D.,
Principal Investigator and Director, Research Institute of Nationwide
Children's Hospital, Columbus, OH
- Tuesday, February 14, 4:30 - 6:30 pm PST
- Poster # 89, Harbor Ballroom
ABO-202 - CLN1, Infantile Batten Disease (INCL):
* Gene therapy extends lifespan and improves quality of life in a mouse model
for Infantile neuronal ceroid lipofuscinosis (INCL) - Alejandra J.
Rozenberg, Ph.D., Steven J. Gray, Ph.D., University of North Carolina at
Chapel Hill, Chapel Hill, NC.
- Wednesday, February 15, 4:30 - 6:30 pm PST
- Poster # 292, Harbor Ballroom
ABO-201 - CLN3, Juvenile Batten Disease (JNCL):
* Comparison of systemic routes of administration for CLN3 delivery via
scAAV9 - S. Kaye Spratt, Ph.D., Abeona Therapeutics Inc.
- Wednesday, February 15, 4:30 - 6:30 pm PST
- Poster # 323, Harbor Ballroom
Abeona Recent ABO-102 Program Highlights:
* Enrolled and dosed the first patient in the high-dose cohort for ABO-102 in
subjects with Sanfilippo syndrome type A
* Received FastTrack designation by the FDA
* Received Orphan Drug Designation in the European Union
* Reported top-line 30-day post-injection data of the low-dose cohort (n=3)
- ABO-102 reduced GAG (heparan sulfate) in urine 57.6% +/- 8.2%
- ABO-102 reduced GAG (heparan sulfate) in the CSF 25.6% +/- 0.8%
- Reduction in liver volume of 17.7% +/- 1.9%
- Reduction in spleen volume of 17.6% +/- 7.1%
* Data to be presented at the WORLDSymposium will include CSF and urinary
heparan sulfate GAG reduction, liver and spleen volume reduction, and
neurological effects in pediatric subjects through six months post-injection
with ABO-102
About WORLDSymposium(TM): The goal of WORLDSymposium(TM) is to provide an
interdisciplinary forum to explore and discuss specific areas of interest,
research and clinical applicability related to lysosomal diseases. Each year,
WORLDSymposium(TM) hosts a scientific meeting presenting the latest information
from basic science, translational research, and clinical trials for lysosomal
diseases. This symposium is designed to help researchers and clinicians to
better manage and understand diagnostic options for patients with lysosomal
diseases, identify areas requiring additional basic and clinical research,
public policy and regulatory attention, and identify the latest findings in the
natural history of lysosomal diseases. For more information,
visit http://www.worldsymposia.org/
About Abeona: Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical
company developing gene therapies for life-threatening rare genetic diseases.
Abeona's lead programs include ABO-102 (AAV-SGSH) and ABO-101 (AAV-NAGLU),
adeno-associated virus (AAV) based gene therapies for Sanfilippo syndrome (MPS
IIIA and IIIB, respectively). Abeona is also developing EB-101 (gene-corrected
skin grafts) for recessive dystrophic epidermolysis bullosa (RDEB), EB-201 for
epidermolysis bullosa (EB), ABO-201 (AAV-CLN3) gene therapy for juvenile Batten
disease (JNCL), ABO-202 (AAV-CLN1) gene therapy for treatment of infantile
Batten disease (INCL), and ABO-301 (AAV-FANCC) for Fanconi anemia (FA) disorder
and ABO-302 using a novel CRISPR/Cas9-based gene editing approach to gene
therapy for rare blood diseases. In addition, Abeona has a plasma-based protein
therapy pipeline, including SDF Alpha(TM) (alpha-1 protease inhibitor) for
inherited COPD, using its proprietary SDF(TM) (Salt Diafiltration) ethanol-free
process. For more information, visit www.abeonatherapeutics.com.
This press release contains certain statements that are forward-looking within
the meaning of Section 27a of the Securities Act of 1933, as amended, and that
involve risks and uncertainties. These statements are subject to numerous risks
and uncertainties, including but not limited to continued interest in our rare
disease portfolio, our ability to enroll patients in clinical trials, the impact
of competition; the ability to develop our products and technologies; the
ability to achieve or obtain necessary regulatory approvals; the impact of
changes in the financial markets and global economic conditions; and other risks
as may be detailed from time to time in the Company's Annual Reports on Form 10-
K and other reports filed by the Company with the Securities and Exchange
Commission. The Company undertakes no obligations to make any revisions to the
forward-looking statements contained in this release or to update them to
reflect events or circumstances occurring after the date of this release,
whether as a result of new information, future developments or otherwise.
Investor Contact:
Christine Berni-Silverstein
Vice President, Investor Relations
Abeona Therapeutics Inc.
+1 (212)-786-6212
csilverstein(at)abeonatherapeutics.com
Media Contact:
Andre'a Lucca
Vice President, Communications & Operations
Abeona Therapeutics Inc.
+1 (212)-786-6208
alucca(at)abeonatherapeutics.com
This announcement is distributed by Nasdaq Corporate Solutions on behalf of Nasdaq Corporate Solutions clients.
The issuer of this announcement warrants that they are solely responsible for the content, accuracy and originality of the information contained therein.
Source: Abeona Therapeutics Inc via GlobeNewswire
Unternehmensinformation / Kurzprofil:
Datum: 07.02.2017 - 13:45 Uhr
Sprache: Deutsch
News-ID 522484
Anzahl Zeichen: 8463
contact information:
Town:
Dallas
Kategorie:
Business News
Diese Pressemitteilung wurde bisher 184 mal aufgerufen.
Die Pressemitteilung mit dem Titel:
"Abeona Therapeutics Announces Presentations and Posters at the 13th Annual WORLDSymposium(TM) 2017"
steht unter der journalistisch-redaktionellen Verantwortung von
Abeona Therapeutics Inc (Nachricht senden)
Beachten Sie bitte die weiteren Informationen zum Haftungsauschluß (gemäß TMG - TeleMedianGesetz) und dem Datenschutz (gemäß der DSGVO).
