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 YORK and CLEVELAND, 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 22^nd at 3:35 pm EST 
Location: The Lotte/Palace Hotel, New York City, NY 
Room: Kennedy II, 4^th Floor 
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 22^nd at 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 Sanfilippo type A program at WORLDSymposium (TM) lysosomal 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(TM) (alpha-1 protease inhibitor) for inherited COPD, using its proprietary SDF(TM) (Salt Diafiltration) ethanol-free process. For more information, visit www.abeonatherapeutics.com.