Abeona Therapeutics to Host Gene Therapy R&D Day on October 11, 2017
August 17 2017 - 9:15AM
Abeona Therapeutics Inc. (Nasdaq:ABEO), a leading
clinical-stage biopharmaceutical company focused on developing
novel gene therapies for life-threatening rare diseases, today
announced it will host its inaugural Research and Development
(R&D) Day event for the institutional investment community in
New York City on Wednesday, October 11, 2017. The formal program
and video webcast will begin at 9:00 am and will conclude at 1:00
pm.
“We are excited to celebrate Abeona’s first
R&D day this fall and update our stakeholders on the
significant progress made in our clinical stage gene therapy
programs for ABO-102 and EB-101, as well as provide a review of the
progress made on the IND-enabling studies for our juvenile (CLN3)
and infantile (CLN1) Batten programs,” stated Timothy J. Miller,
Ph.D., President and CEO. “Additionally, we plan to provide details
on our manufacturing strategy for both the EB-101 program and our
multiple AAV gene therapy programs,” he continued.
Presentations by senior management, clinical
investigators and Key Opinion Leaders will cover the following:
--Clinical progress on ABO-102 for the treatment
of Sanfilippo syndrome type A, including an update on enrollment
status at international clinical sites in Australia and Spain, as
well as a review of the clinical data for patients currently
enrolled;
--Program update on the EB-101 gene therapy for
recessive dystrophic epidermolysis bullosa (RDEB) as the Company
prepares to initiate a pivotal Phase 3 clinical trial, including an
update on Abeona’s manufacturing strategy, plans and progress in
the development of its clinical and commercial grade production
facility;
--A detailed discussion on development of
Abeona’s proprietary AIM™ vector system, a next generation platform
of AAV capsids being developed in collaboration with the University
of North Carolina at Chapel Hill; initial studies indicate that
AIM™ vectors can efficiently target multiple tissues with
vector-selective tissue specificity with different routes of
administration relative to first generation vectors, providing
second-generation treatment approaches for patients as a redosing
strategy or for patients that have neutralizing antibodies to
natural AAV serotypes.
“We are pleased with the progress in our ongoing
scientific collaboration with UNC on the next generation AIM™
chimeric AAV vectors, many of which have exhibited improved tissue
tropisms over first generation, naturally occurring AAV serotypes,
together with liver de-targeting capabilities,” stated Steven H.
Rouhandeh, Executive Chairman. “We look forward to developing the
AIM™ chimeric AAV vectors both internally and through strategic
partnering efforts,” he continued.
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), an
adeno-associated virus (AAV) based gene therapy for Sanfilippo
syndrome type A (MPS IIIA) and EB-101 (gene-corrected skin grafts)
for recessive dystrophic epidermolysis bullosa (RDEB). Abeona
is also developing ABO-101 (AAV-NAGLU) for Sanfilippo syndrome type
B (MPS IIIB), ABO-201 (AAV-CLN3) gene therapy for juvenile Batten
disease (JNCL), ABO-202 (AAV-CLN1) for treatment of infantile
Batten disease (INCL), EB-201 for epidermolysis bullosa (EB),
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
proprietary vector platform, AIM™, for next generation product
candidates. For more information, visit
www.abeonatherapeutics.com.
Investor Contact: Christine Silverstein Vice
President, Investor Relations Abeona Therapeutics Inc. +1
(212)786-6212 csilverstein@abeonatherapeutics.com
Media Contact: Lynn Granito Berry
& Company Public Relations
Tel: 212-253-8881 lgranito@berrypr.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 include, without limitation, our
plans for continued development and internationalization of our
clinical programs, that patients will continue to be identified,
enrolled, treated and monitored in the EB-101 clinical trial, and
that studies will continue to indicate that EB-101 is
well-tolerated and may offer significant improvements in wound
healing; the addition of two additional global clinical sites will
accelerate our ability to enroll and evaluate ABO-102 as a
potential treatment for patients with Sanfilippo syndrome type A,
or MPS IIIA. Such 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 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; our belief that initial signals of
biopotency and clinical activity, which suggest that ABO-102
successfully reached target tissues throughout the body, including
the central nervous system and the increased reductions in CNS GAG
support our approach for intravenous delivery for subjects with
Sanfilippo syndromes, 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 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.
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