NEW YORK and CLEVELAND, July 25, 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
today that Juan Ruiz, M.D., Ph.D., MBA has joined the company as Chief Medical Officer. He will be responsible for leading all
clinical development, medical affairs and related functions and will report directly to Timothy J. Miller, Ph.D., President and
CEO.
A photo accompanying this announcement is available at http://www.globenewswire.com/NewsRoom/AttachmentNg/54ff84e1-4ff4-4192-a0eb-92df1dd362c2
Dr. Juan Ruiz is well regarded for his extensive research in molecular biology and gene therapy. He has
previously held global leadership positions at Lykera Biomed and Digna Biotech, where he spent over 13 years leading teams
dedicated to developing gene therapy translational medicine programs. During his tenure as Chief Medical Officer of Digna Biotech,
Dr. Ruiz’s proven track record demonstrated a “start to finish” scope, from proof-of-concept studies and regulatory IND and IMPD
submissions, to manufacturing, clinical trial design and execution, and partnering and licensing activities. In addition, he has
expertise in the manufacturing and quality control of the adenoviral vectors that were tested in his clinical trials.
“Juan is a proven leader in gene therapy clinical development, with both academic and industry experience, which
will be invaluable as we advance our MPS III and RDEB programs into pivotal trials,” said Timothy J. Miller, Ph.D., Abeona
President and CEO. “His training in medicine and ability to translate scientific discoveries from concept to clinical trial will
add depth to an experienced management team and advisors.”
Dr. Ruiz has been instrumental in designing Patient Reported Outcomes (PRO) in the USA to assess skin outcome
measures in patients with Systemic Sclerosis for use as primary endpoints in Phase IIb trials. Additional work includes multiple
regulatory designation achievements and successful Scientific Advice and Protocol Assistance meetings with the FDA and EMA. He is
the author of over forty peer-reviewed journal articles and book chapters and an inventor on multiple issued patents. Dr.
Ruiz holds a M.D. with a specialty in Hepatology and a Ph.D. in Molecular Biology, both from the University of Navarra, and
completed his post-doctoral fellowship at the University of Connecticut. He holds a MBA from IESE Business School, also at the
University of Navarra.
“We stand at a transformative moment in gene therapy and the development of rare disease therapies,” commented
Dr. Ruiz. “Gene therapy has shown the ability to correct the underlying pathology associated with lysosomal storage diseases and
skin disorders, and Abeona is harnessing the power of this technology for the potential to improve patient lives in multiple
ongoing clinical trials. With its robust pipeline, novel AIM™ vector platform and strong gene therapy know-how, Abeona is well
positioned to continue making its innovative mark in the rare disease industry.”
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
csilverstein@abeonatherapeutics.com
Media Contact:
Andre’a Lucca
Vice President, Communications & Operations
Abeona Therapeutics Inc.
+1 (212) 786-6208
alucca@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 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; 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.
