Abeona Therapeutics Appoints João Siffert, M.D. Head of Research and Development and Chief Medical Officer

Abeona Therapeutics Inc. (Nasdaq: ABEO), a leading clinical-stage biopharmaceutical company focused on developing novel cell and gene therapies for life-threatening rare genetic diseases, today announced the appointment of João Siffert, M.D. as Head of Research and Development and Chief Medical Officer.

NEW YORK and CLEVELAND, -- Abeona Therapeutics Inc. (Nasdaq: ABEO), a leading clinical-stage biopharmaceutical company focused on developing novel cell and gene therapies for life-threatening rare genetic diseases, today announced the appointment of João Siffert, M.D. as Head of Research and Development and Chief Medical Officer. As a result, former Chief Medical Officer Juan Ruiz, M.D., Ph.D. will assume the role of Head of European Medical Affairs. The Company also announced the appointment of Neena Patil, J.D. as General Counsel and Corporate Secretary. Both Dr. Siffert and Ms. Patil report to Chief Executive Officer, Carsten Thiel, Ph.D.

“I am very pleased to welcome João and Neena to Abeona, and confident in their abilities to make important contributions to the future growth of our company,” said Dr. Thiel. “João has an ideal blend of clinical, scientific, and regulatory experience to reference as we look to advance our clinical and pre-clinical candidates and cultivate future pipeline growth.”

“Neena brings a breadth of legal expertise to Abeona and the leadership required to ensure the organization continues to operate responsibly and in a compliant manner as we evolve from a clinical- to commercial-stage company,” added Dr. Thiel.

Dr. Siffert has 30 years of combined experience in the biopharmaceutical industry, medicine, and academia. He has successfully led multiple drug development programs from pre-clinical to regulatory approvals in the U.S. and Europe, and has held several scientific leadership positions in biopharmaceuticals, including programs in gene therapy. In 2017, Dr. Siffert was appointed to the Board of Directors of gene therapy developer AveXis, which was subsequently acquired by Novartis. He served as Chief Medical Officer for Ceregene from 2007 to 2011, where he was responsible for clinical development of adeno-associated viral (AAV2)-based gene therapies for Parkinson’s and Alzheimer’s diseases. Dr. Siffert also led the R&D and medical organizations at Avanir Pharmaceuticals and Avera Pharmaceuticals before most recently guiding translational research, clinical development, regulatory, and medical affairs as Chief Scientific and Medical Officer for Nestle Health Science.

Dr. Siffert earned an M.D. from the University of São Paulo, Brazil and an M.B.A. from Columbia University. He completed medical residencies in pediatrics at New York University (NYU) School of Medicine and in neurology at Harvard Medical School, followed by a fellowship in neuro-oncology at NYU. The American Board of Neurology and Psychiatry certified Dr. Siffert in 1996.

“I’m excited to be part of the Abeona team and look forward to guiding our cell and gene therapy clinical development programs,” said Dr. Siffert. “The work being done at Abeona has the potential to be transformative for this burgeoning field, and most importantly, for patients.”

Ms. Patil brings nearly 20 years of global biopharmaceutical experience to Abeona. She most recently served as Associate General Counsel and Vice President of Legal Affairs at Novo Nordisk. In this role, Ms. Patil developed legal and compliance strategies for the company and led the team responsible for providing comprehensive legal support to Novo Nordisk’s U.S. business. Ms. Patil has worked extensively in corporate, regulatory, litigation and compliance matters across therapeutic areas, including in rare diseases. Her experience prior to Novo Nordisk includes various positions at other global biopharmaceutical companies including Pfizer and Sanofi.

Ms. Patil earned her J.D. and Master’s in Health Services Administration from the University of Michigan in Ann Arbor and her undergraduate degree from Georgetown University with a Bachelor of Arts in Political Science.

About Abeona

Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing cell and gene therapies for life-threatening rare genetic diseases. Abeona’s lead programs include EB-101 (gene-corrected skin grafts) for recessive dystrophic epidermolysis bullosa (RDEB), ABO-102 (AAV-SGSH), an adeno-associated virus (AAV) based gene therapy for Sanfilippo syndrome type A (MPS IIIA) and ABO-101 (AAV-NAGLU), an adeno-associated virus (AAV) based gene therapy for Sanfilippo syndrome type B (MPS IIIB). Abeona is also developing ABO-201 (AAV-CLN3) gene therapy for CLN3 disease, ABO-202 (AAV-CLN1) for treatment of CLN1 disease, 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 is developing a proprietary vector platform, AIM™, for next generation product candidates. For more information, visit www.abeonatherapeutics.com.

Investor Contact:

Christine Silverstein
SVP, Finance & Investor Relations
Abeona Therapeutics, Inc.
+1 (646) 813-4707
csilverstein@abeonatherapeutics.com

Media Contact:

Scott Santiamo
Director, Corporate Communications
Abeona Therapeutics, Inc.
+1 (646) 813-4719
ssantiamo@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 include statements regarding our pipeline maturing toward commercial stages; our expectation that the broad experience, deep understanding of the healthcare industry, development of high performing teams, effective resource allocation and proven ability to achieve ambitious outcomes by João Siffert and Neena Patil will serve our future growth. We have attempted to identify forward looking statements by such terminology as “may,” will,” “anticipate,” “believe,” “estimate,” “expect,” “intend,” and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, 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 and licenses; 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.

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