Abeona Therapeutics to Present at Jefferies Gene Editing &

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News Desk 2018
Abeona Therapeutics to Present at Jefferies Gene Editing & Gene Therapy Summit

NEW YORK, NY and CLEVELAND, OH --(Marketwired - October 04, 2016) - Abeona Therapeutics Inc. ( NASDAQ : ABEO ) a clinical-stage biopharmaceutical company focused on developing therapies for life-threatening rare genetic diseases, today announced that CEO & President, Timothy J. Miller, Ph.D., will be presenting for The Company at Jefferies Gene Editing & Gene Therapy Summit, October 11 th at 11 am ET in New York City.

The following are the specific details regarding Abeona Therapeutics Presentation:

Event: Jefferies Gene Editing & Gene Therapy Summit Date: Tuesday, October 11 th , 2016 Time: 11:00 am ET Location: New York City, NY

Abeona Recent Highlights:

  • September 26 th , 2016, Abeona Therapeutics Enrolls First Patient in Phase 2 for EB-101 Gene Therapy Clinical Trial for Epidermolysis Bullosa
  • September 21 st , 2016, Abeona announced the exclusive worldwide license of a next generation gene therapy AAV capsid portfolio from University of North Carolina at Chapel Hill
  • September 8 th , 2016, Abeona Enrolls 5 th Patient in Phase 1/2 Gene Therapy Clinical Trial for Epidermolysis Bullosa
  • August 29, 2016, Abeona completes Enrollment of Low-Dose Cohort for ABO-102 in Phase 1/2 Clinical Trial for MPS IIIA Patients
  • August 9, 2016, Abeona announced a collaboration with the EB Research Partnership, EB Medical Research Foundation and Stanford University for the development of treatments for recessive dystrophic epidermolysis bullosa (RDEB)
  • August 4, 2016, Abeona announced it had received European regulatory approval by the Agencia Espanola de Medicamentos y Productos Sanitarios for its Phase 1/2 trial for ABO-102 (AAV-SGSH) to be conducted at Cruces University Hospital (Bilbao, Spain)
  • August 2, 2016, Abeona provided an update on the initial subjects enrolled in this trial, stating that ABO-102 had been well tolerated with no safety or tolerability concerns identified through 30-days post-injection, and that encouraging signs of early biopotency had been observed in urinary and CSF GAG (heparan sulfate) measurements as well as potential disease-modifying effects in the liver and spleen

About Abeona: Abeona Therapeutics, Inc. is a clinical stage company developing gene and plasma-based therapies for life-threatening rare genetic diseases. Abeona's lead programs are 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), 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 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 our proprietary SDF™ (Salt Diafiltration) ethanol-free process. For more information, visit www.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; 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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