Abeona Therapeutics Inc: Abeona Enrolls First Subject in Spain in Ongoing Phase 1/2 Clinical Trial in MPS IIIA

-- Company Opens European Subsidiary in Spain to Support Global Clinical
     Development
  -- Nine subjects at global clinical sites enrolled to date with more than
     2,000 days cumulative follow up assessed

NEW YORK and CLEVELAND, Nov. 09, 2017 (GLOBE NEWSWIRE) -- Abeona Therapeutics
Inc. (Nasdaq:ABEO), a leading clinical-stage biopharmaceutical company focused
on developing novel cell and gene therapies for life-threatening rare diseases,
announced today that the first patient was enrolled in the Company's ABO-102
Phase 1/2 clinical trial at the Hospital Clinico Universitario of Santiago de
Compostela, Spain.  In conjunction with the initiation of the Spain clinical
site, Abeona has established a local subsidiary to manage clinical trial and
regulatory development efforts in Europe. 

"We are pleased to initiate enrollment at our Spain clinical site for ABO-102. 
We remain encouraged by the improvements observed in clinically relevant
biomarkers post-dosing of the gene therapy in the patients in Cohort 3 and the
ongoing safety profile ABO-102 demonstrates," stated Juan Ruiz, M.D., Ph.D.,
Chief Medical Officer of Abeona Therapeutics. "Developing a local company
presence in Spain allows us to exercise closer supervision and further advance
Abeona's gene therapy programs in Europe, as well as advance our relationships
with the patient community. We are grateful to the many patient foundations and
parents who have supported the research needed to advance a potential treatment
for this devastating unmet medical need. We are also proud to collaborate with
one of the leading clinical centers in Spain dealing with MPS IIIA patients." 

Pursuant to the design of the ongoing clinical trial, subjects receive a
single, intravenous injection of ABO-102 to deliver the AAV viral vector
systemically throughout the body to introduce a corrective copy of the gene
that underlies the cause of the MPS IIIA disease. Subjects are evaluated at
multiple time points  post-injection for safety assessments and signals of
biopotency and clinical activity, which to date have demonstrated that ABO-102
successfully reaches target tissues throughout the body, including the central
nervous system. 

"MPS IIIA is a profound and deadly lysosomal storage disease with no approved
treatments available,"  stated Dr. Maria Luz Couce, Director of the Unit of
Diagnosis and Treatment of Congenital Metabolic Diseases, Hospital Clinico
Universitario of Santiago de Compostela in Spain, "We are excited to initiate
enrollment and are encouraged by the durable and significant reduction in
central nervous system heparan sulfate as a key biomarker of disease pathology
and look forward to enrolling more patients in this gene therapy trial." 

A total of nine subjects have been infused in the ongoing global MPS IIIA
dose-escalation clinical trial, which has been well-tolerated through more than
2,000 cumulative follow-up days.  Safety and efficacy data has been reviewed
and enrolliment in Cohort 3 continues at all three active clinical sites (US,
Spain, and Australia). 

"We are grateful to see the fruition of our collaboration with Abeona, which
has opened a promising new hope in Sanfilippo treatments. That the first
patient in Spain has been treated with this therapy is great news for our
community, and we look forward to supporting additional enrollments in this
clinical trial," stated  Emilio Lopez, President of Fundación Stop Sanfilippo. 

About Abeona: Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical
company developing cell and 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 is developing 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.
[phone]
[e-mail]

Media Contact:
Lynn Granito
Berry & Company Public Relations
[phone]
[e-mail]

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 obtain any necessary intellectual property to commercialize any
of our products; the ability to achieve or obtain necessary regulatory
approvals and licenses; 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.