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Abeona Therapeutics to Present at Multiple Upcoming Conferences

(May 19, 2017)

NEW YORK and CLEVELAND, May 19, 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, announced that members of the management team will present at the following upcoming conferences:

Event: Cell & Gene Exchange

Panelist: President and CEO, Timothy J. Miller, Ph.D.Panel: ?Best Practices for Partnering with Disease Foundations and Patient Groups

Date: Monday, May 22nd 
Time: 3:15 PM ET

Presenter: President and CEO, Timothy J. Miller, Ph.D.
Abeona Company Presentation
Date: Tuesday, May 23rd
Time: 2:30 pm ET
Room: Atrium Ballroom
Location: Ronald Reagan Building, Washington, D.C.
Website: https://ebdgroup.knect365.com/cellgene-exchange/

Event: UBS Global Healthcare Conference
Presenter: Chief Operating Officer, Jeffrey B. Davis
Date: Tuesday, May 23rd
Time: 2:30 PM ET
Room: Ballroom V
Location: Grand Hyatt, New York, NY
Webcast: www.ibb.ubs.com/Conferences

Event: Neurotech Investing and Partnering Conference
Panelist: President and CEO, Timothy J. Miller, Ph.D.
Panel: ?Targeting Orphan Diseases
Date: Thursday, June 1st
Time: 3:45 PM PT
Location: Park Central Hotel, San Francisco, CA
Website: http://www.neurotechpartnering.com/

Abeona Recent Highlights

ABO-102 gene therapy program for Sanfilippo syndrome Type A:
Abeona announced top-line data for Phase 1/2 MPS IIIA gene therapy trial at ASGCT 
--Positive dose response in central nervous system with 60.7% +/- 8.8% reduction of disease-causing heparan sulfate GAG observed in Cohort 2 
--Reduction of disease manifestation observed in decreased liver volume of 14.81% (+/- 1.2%) 
--ABO-102 well-tolerated in six subjects through more than 1,100 days cumulative follow-up with no Serious Adverse Events
--Cohort 1 demonstrated stabilized or improved Leiter Nonverbal IQ scores at six months 
Abeona received regulatory approval to initiate ABO-102 gene therapy clinical trial in Australia for patients with MPS IIIA

EB-101 gene therapy program for severe form of Epidermolysis Bullosa:
Abeona provided an update for Phase 1/2a gene therapy to the Society for Investigative Dermatology Conference
--EB-101 demonstrated significant wound healing (defined as greater than 50% healed) in 100% of treated wounds (36/36) at 3 months; 89% (32/36) at 6 months, 83% (20/24) at 12 months, 88% (21/24) at 24 months and 100% (6/6) at 36 months post-administration
--Clinical endpoints supported by data from Natural History Study observations from 1,436 wounds in 128 patients with Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Abeona was granted Orphan Drug Designation for EB-101 in Epidermolysis Bullosa by the European Medicines Agency (EMA) Committee for Orphan Medicinal Products

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 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 www.abeonatherapeutics.com.

Investor Contact:
Christine Silverstein
Vice President, Investor Relations
Abeona Therapeutics Inc.
+1 (212)-786-6212
[email protected]

Media Contact:
Andrea Lucca
Vice President, Communications & Operations
Abeona Therapeutics Inc.
+1 (212)-786-6208
[email protected]

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 the statement that the ability to develop our products and technologies; 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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