NEW YORK and CLEVELAND, Aug. 17, 2017 (GLOBE NEWSWIRE) -- 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.
