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Abeona Therapeutics Enrolls First High Dose Subject in Ongoing Phase 1/2 Gene Therapy Clinical Trial in Sanfilippo Syndrome Type A

Abeona Therapeutics Inc
Press release

Abeona Therapeutics Enrolls First High Dose Subject in Ongoing Phase 1/2 Gene
Therapy Clinical Trial in Sanfilippo Syndrome Type A

-- ABO-102, the leading clinical gene therapy program for Sanfilippo syndrome
type A patients, has demonstrated central nervous system (CNS) and
peripheral organ disease biopotency
-- First high dose cohort patient is enrolled, and all patients (n = 4) have
cumulative 644 days post-injection with no Serious Adverse Events (SAEs)
reported to date
-- Global ABO-102 enrollments in Europe and Australia commencing in the second

NEW YORK and CLEVELAND, Feb. 01, 2017 (GLOBE NEWSWIRE) -- Abeona Therapeutics
Inc. (Nasdaq:ABEO), a clinical-stage biopharmaceutical company focused on
developing therapies for life-threatening rare genetic diseases, announced
today that the first high-dose subject was enrolled in the ongoing Phase 1/2
trial for ABO-102 (AAV-SGSH). The first-in-man clinical trial utilizes a single
intravenous injection of AAV gene therapy for patients with MPS IIIA
(Sanfilippo syndrome type A), a devastating lysosomal storage disease that
affects every cell and organ resulting in neurocognitive decline, speech loss,
loss of mobility, and premature death.

“The encouraging clinical data from the low-dose patients continue to support a
whole-body treatment approach using an intravenously delivered AAV to deliver
and drive expression of the SGSH enzyme in all organs of the body, particularly
the brain,” stated Kevin M. Flanigan, MD, principal investigator of the
clinical trial, Director of the Center for Gene Therapy and Neuromuscular
Disorders Program at Nationwide Children’s Hospital and Professor of Pediatrics
and Neurology at The Ohio State University College of Medicine. “Additionally,
we are encouraged that the 6-month biopotency in two initial subjects are
suggestive of sustained disease modification, and we look forward to presenting
additional data at the WORLDSymposium™ lysosomal storage conference later this

The ongoing Phase 1/2 clinical trial, which has received FastTrack designation
by the FDA, is designed to evaluate safety and preliminary indications of
efficacy of ABO-102 in patients suffering from MPS IIIA. Per the design of the
trial, subjects in the low-dose and high-dose cohorts received a single,
intravenous injection of ABO-102 to deliver the AAV viral vector systematically
throughout the body to introduce a corrective copy of the gene that underlies
the cause of the MPS IIIA disease, particularly the CNS. Subjects are evaluated
at multiple time points over the initial 6-months post-injection for safety
assessments and initial signals of biopotency.

Previously announced 30-day post-injection data for the low dose cohort

-- 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%

“The combination of CSF and urinary heparan sulfate GAG reduction, liver and
spleen volume reduction, and neurological effects support our world leading
gene therapy treatment paradigms for patients with MPS III,” stated Timothy J.
Miller, Ph.D., President and CEO of Abeona Therapeutics. “We look forward to
Dr. Flanigan’s presentation of the biopotency and neurological data at the
upcoming WORLDSymposium.”

Abeona’s MPS IIIA program, ABO-102, has been granted Orphan Product Designation
in the USA and Europe, and has also received the Rare Pediatric Disease
Designation in the United States.

About ABO-102 (AAV-SGSH): ABO-102 is an adeno-associated viral (AAV)-based gene
therapy for MPS IIIA (Sanfilippo syndrome), which involves a one-time delivery
of a normal copy of the defective gene to cells of the central nervous system
(CNS) with the aim of reversing the effects of the genetic errors that cause
the disease. ABO-102 has been well tolerated in initial subjects of the
low-dose cohort with no safety or tolerability concerns identified through 30
day post-injection in patients suffering from MPS IIIA, or Sanfilippo syndrome
Type A, a rare autosomal recessive disease caused by genetic mutations that
result in a deficiency of SGSH enzyme activity, leading to abnormal
accumulation of GAG (specifically, heparan sulfate) in the CNS and systemic
tissues and organs. This accumulation of heparan sulfate results in
neurocognitive decline, speech loss, loss of mobility, and premature death.
Encouraging signs of early biopotency have been observed in urinary and CSF GAG
(glycosaminoglycan, specifically, heparan sulfate) measurements, as well as
potential disease-modifying effects in the liver and spleen of the initial
subjects enrolled and treated in the trial. The clinical study is supported by
neurocognitive evaluations, biochemical assessments and MRI data generated in a
25-subject MPS III Natural History Study, also conducted at Nationwide
Children's Hospital, where patients continued through one-year of follow up

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™ (alpha-1 protease inhibitor) for
inherited COPD, using its proprietary SDF™ (Salt Diafiltration) ethanol-free
process. For more information, visit

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

Investor Contact:
Christine Berni-Silverstein
Vice President, Investor Relations
Abeona Therapeutics Inc
+1 (212)-786-6212

Media Contact:
Andre'a Lucca
Vice President, Communications & Operations
Abeona Therapeutics Inc
+1 (212)-786-6208

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