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Abeona Therapeutics Inc

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Abeona Therapeutics to Present at the 2017 RBC Capital Markets Global Healthcare Conference and 3rd Annual CRISPR Congress

Company CEO and COO Presentations to be Held on Wednesday, February 22nd

NEW YORKandCLEVELAND, Feb. 21, 2017 (GLOBE NEWSWIRE) -- Abeona Therapeutics Inc. (NASDAQ:ABEO) a leading clinical-stage biopharmaceutical company focused on developing therapies for life-threatening rare genetic diseases, today announced that members of management will present at the following upcoming investor and industry conferences:

Investor Conference:2017 RBC Capital Markets Global Healthcare Conference
Presenter: Jeffrey B. Davis, Chief Operating Officer
Date/Time:Wednesday, February 22ndat 3:35 pm EST
Location: The Lotte/Palace Hotel, New York City, NY
Room: Kennedy II, 4thFloor
Live Webcast Link:http://www.veracast.com/webcasts/rbc/healthcare2017/58114482008.cfm

Industry Conference: 3rd Annual CRISPR & Precision Genome Editing Congress 2017
Presenter: Timothy J. Miller, Ph.D., Chief Executive Officer
Presentation Title: Application of CRISPR Technology in the Development of Therapeutics for Blood Disorders
Date/Time: Wednesday, February 22ndat 11:05 am EST
Location: Sheraton Boston Hotel, Boston, MA
Website:http://crispr-congress.com/

Abeona Recent Highlights:

  • February 17, 2017: Provided update from ABO-102 Sanfilippotype A program at WORLDSymposiumlysosomal storage disorders conference

-- 63% +/- 0.5% reduction in the disease-causing sugar (heparan sulfate GAG) in the central nervous system 6 months post-injection (N=2)
-- Continued evidence of biopotency: reduced liver and spleen volumes, decreased urinary GAGs
-- 6-month timepoint (n=2) showed evidence for stabilization or improvement (average 60%) in several Mullen subdomains
-- Adaptive behavior ratings on the Vineland stabilized
-- Subjects showed improved ability to complete individual items on the Leiter-R non-verbal IQ assessment resulting in improved raw scores
-- ABO-102 gene therapy well-tolerated in 4 subjects (N=3 low dose, N=1 high dose) through 650 days follow up with no Serious Adverse Events

  • February 1, 2017: Enrolled first high dose subject in ongoing Phase 1/2 gene therapy clinical trial for Sanfilippo syndrome Type A (MPS IIIA)
  • January 19, 2017: Received Orphan Drug Designation in the European Union for ABO-101 gene therapy in Sanfilippo syndrome Type B (MPS IIIB)
  • January 3, 2017: Received Orphan Drug Designation in the European Union for ABO-201 gene therapy program in juvenile Batten disease (JNCL)

About Abeona:Abeona Therapeutics Inc. is a leading 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, visitwww.abeonatherapeutics.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 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; 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; our belief that the data demonstrate an early and robust systemic delivery of ABO-102, 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 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.

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

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

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