Abeona Therapeutics to Present Data for ABO-401 in Cystic Fibrosis and Retinal Disorders at the American Society of Gene and ...
April 15 2019 - 02:41PM
Abeona Therapeutics Inc. (Nasdaq: ABEO), a fully-integrated leader
in gene and cell therapy, today announced that new data
demonstrating the capabilities of the AIM™ capsid library in cystic
fibrosis and retinal disorders will be presented at the American
Society of Gene and Cell Therapy 22nd Annual Meeting, being held
April 29 – May 2, 2019 in Washington, D.C. Additional preclinical
data for ABO-202, the Company’s AAV9-based gene therapy for the
treatment of patients with CLN1 disease, will also be presented.
“The data to be presented at the ASGCT meeting will highlight
study results on the delivery and expression of hCFTR gene in
cystic fibrosis and the potential of the AIM™ AAV vector platform
in the treatment of multiple retinal disorders. New data from
IND-enabling studies of our program in CLN1 disease, expected to
enter the clinic later this year, will also be presented,” said
João Siffert, M.D., Chief Executive Officer.
“The AIM capsid library has demonstrated significant versatility
with the ability to deliver genes to target tissues with enhanced
specificity,” added Timothy J. Miller, Ph.D., President and Chief
Scientific Officer. “This next-generation AAV capsid platform has
the potential to transform how we can target pulmonary, retinal and
neurological disorders with gene therapy.”
Identification of AAV Developed for Cystic Fibrosis (CF)
Gene Therapy That Restores CFTR Function in Human CF Patient
CellsPresenter: Paul T. Wille, Ph.D., Case Western Reserve
University, Cleveland, OH; Abeona Therapeutics Inc.Date/Time:
Tuesday April 30, 5:00 – 6:00 p.m. ETPoster Board Number: 92Session
title: Cardiovascular and Pulmonary DiseasesRoom: Columbia
HallAbstract number: 528
Novel AAV Capsids Demonstrate Strong Retinal Expression
in Non-Human Primates After Intravitreal
AdministrationPresenter: Brian Kevany, Ph.D., Abeona
Therapeutics Inc.Date/Time: Monday April 29, 5:00 – 6:00 p.m.
ETSession title: Neurologic DiseasesRoom: Columbia HallAbstract
number: 225
Intrathecal and Intravenous Combination Gene Therapy in
the Mouse Model of Infantile Neuronal Ceroid Lipofuscinosis Extends
Lifespan and Improves Behavioral Outcomes in Moderately Affected
MicePresenter: Erik A. Lykken, Ph.D., University of Texas
Southwestern Medical Center, Dallas, TXDate/Time: Monday April 29,
11:45 a.m. – 12:00 p.m. ETSession title: Tools, Delivery and Neuro
CapsidsRoom: Monroe
About Abeona Therapeutics Abeona Therapeutics
Inc. is a clinical-stage biopharmaceutical company developing gene
and cell therapies for serious diseases. The Company’s clinical
programs include EB-101, its autologous, gene-corrected cell
therapy for recessive dystrophic epidermolysis bullosa, as well as
ABO-102 and ABO-101, novel AAV9-based gene therapies for Sanfilippo
syndrome types A and B (MPS IIIA and MPS IIIB). The Company’s
portfolio of AAV9-based gene therapies also features ABO-202 and
ABO-201 for CLN1 disease and CLN3 disease, respectively. Its
preclinical assets include ABO-401, which uses the novel AIM™ AAV
vector platform to address all mutations of cystic fibrosis. Abeona
has received numerous regulatory designations from the FDA and EMA
for its pipeline candidates and is the only company with
Regenerative Medicine Advanced Therapy designation for two
investigational therapies (EB-101 and ABO-102). For more
information, visit www.abeonatherapeutics.com.
Forward Looking StatementThis
press release contains certain statements that are forward-looking
within the meaning of Section 27A of the Securities Act of 1933, as
amended, and Section 21E of the Securities Exchange Act of 1934, as
amended, and that involve risks and uncertainties. These
statements include statements regarding our pipeline including the
potential for the AIM™ vector platform in the treatment of multiple
disorders including cystic fibrosis and retinal diseases, and the
potential for ABO-202 for CLN-1, including the initiation of
clinical studies, the nature of feedback from regulatory agencies,
and the company’s goals and objectives. We have attempted to
identify forward looking statements by such terminology as “may,”
“will,” “anticipate,” “believe,” “estimate,” “expect,” “intend,”
and similar expressions.
Actual results may differ materially from those indicated by
such forward-looking statements as a result of various important
factors, numerous risks and uncertainties, including but not
limited to: continued interest in our rare disease portfolio, our
ability to initiate and enroll patients in clinical trials, the
impact of competition, the ability to secure licenses for any
technology that may be necessary to commercialize our products, the
ability to achieve or obtain necessary regulatory approvals, the
impact of changes in the financial markets and global economic
conditions, risks associated with data analysis and reporting, and
other risks as may be detailed from time to time in the Company’s
annual reports on Form 10-K and quarterly reports on Form 10-Q and
other reports filed by the Company with the Securities and Exchange
Commission. The Company undertakes no obligation to revise
the forward-looking statements or update them to reflect events or
circumstances occurring after the date of this presentation,
whether as a result of new information, future developments or
otherwise, except as required by the federal securities laws.
Investor Contact: Sofia Warner Senior Director,
Investor Relations Abeona Therapeutics +1 (646) 813-4710
swarner@abeonatherapeutics.com
Media Contact: Scott Santiamo Director,
Corporate Communications Abeona Therapeutics +1 (718) 344-5843
ssantiamo@abeonatherapeutics.com
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