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Scientific Data for Novel Adeno-Associated Virus Gene Therapy in Rhodopsin-Mediated Autosomal Dominant Retinitis Pigmentosa Published in Proceedings of the National Academy of Sciences


- Preclinical Proof-of-Concept Results in a Large Animal Model
Published Online Ahead of Print in PNAS -

Ophthotech Corporation (NASDAQ:OPHT) announced today that scientists at
the University of Pennsylvania (Penn) and University of Florida
published proof-of-concept study results on an adeno-associated virus
(AAV) gene therapy product candidate for the treatment of
rhodopsin-mediated autosomal dominant retinitis pigmentosa (RHO-adRP)
licensed by Ophthotech. Their work conducted in a naturally occurring
canine model of the disease was published online ahead of print in the
journal Proceedings of the National Academy of Sciences of the USA (PNAS).
In June 2018, Ophthotech announced that it entered into an exclusive
global license agreement with the University of Florida Research
Foundation and Penn for rights to develop and commercialize this novel
AAV gene therapy product candidate for the treatment of RHO-adRP. In
addition to the exclusive license agreement, Ophthotech and Penn have
also entered into a master sponsored research agreement, facilitated by
the Penn Center for Innovation, pursuant to which Ophthotech and Penn
plan to conduct additional preclinical studies of the RHO-adRP product
candidate and a natural history study in RHO-adRP patients.

This publication is entitled: "Mutation-independent Rhodopsin Gene
Therapy by Knockdown and Replacement with a Single AAV vector" by Artur
V. Cideciyan, Raghavi Sudharsan, Valérie L. Dufour, Michael T.
Massengill, Simone Iwabe, Malgorzata Swider, Brianna Lisi, Alexander
Sumaroka, Luis Felipe Marinho, Tatyana Appelbaum, Brian Rossmiller,
William W. Hauswirth, Samuel G. Jacobson, Alfred S. Lewin, Gustavo D.
Aguirre, and William A. Beltran. PNAS 2018.

RHO-adRP is an orphan monogenic inherited retinal disease that is
characterized by progressive and severe loss of vision, and is caused by
more than 150 different mutations in the RHO gene. The construct
for the RHO-adRP product candidate combines a transgene expressing a
highly efficient, novel short hairpin RNA (shRNA) designed to target and
knock-down endogenous rhodopsin (RHO) in a mutation-independent
manner with a human RHO replacement transgene made resistant to
RNA interference, in a single AAV2/5 vector. This construct was tested
in a naturally-occurring canine disease model of RHO-adRP by
investigators at Penn, resulting in a complete suppression of the
endogenous RHO RNA while the human RHO replacement
transgene resulted in up to 30% of normal RHO protein levels. Long term
(over 8 months) anatomic and functional preservation was demonstrated
with retinal imaging and electrophysiology. Ophthotech believes these
results further confirm the therapeutic benefit of a similar knock-down
and replacement approach that was tested in mice by investigators at the
University of Florida, the results of which were previously published in Human
Gene Therapy
in 2012.

"Our prior research indicated that the canine animal model may provide
useful predictive data when developing treatment strategies for patients
with degenerative retinal diseases," stated Professor William A.
Beltran, DVM, PhD, Director of the Division of Experimental Retinal
Therapies, Department of Clinical Sciences & Advanced Medicine, School
of Veterinary Medicine, University of Pennsylvania. "We are honored to
have our preclinical research in AAV therapy for RHO-adRP published in PNAS."

"We are excited to collaborate with prominent scientists in the field of
degenerative retinal diseases at the University of Pennsylvania and
University of Florida and congratulate them on the strength and elegance
of this publication," stated Kourous A. Rezaei, M.D., Chief Medical
Officer of Ophthotech. "We are intrigued by these proof-of-concept
results in the canine disease model which further support the potential
therapeutic impact of this AAV gene therapy product in patients with
RHO-adRP. We are commencing IND-enabling activities, and based on
current timelines and subject to regulatory review, we expect to
initiate a Phase 1/2 clinical trial for the treatment of RHO-adRP in

About RHO-adRP

RHO-adRP is an orphan monogenic inherited retinal disease that is
characterized by progressive and severe loss of vision. Ophthotech
estimates that there are approximately 11,000 individuals in the United
States and the five major European markets with RHO-adRP. There is
currently no U.S. Food and Drug Administration or European Medicines
Agency approved therapy to treat this orphan inherited retinal disease.

About Ophthotech Corporation

Ophthotech is a science-driven biopharmaceutical company specializing in
the development of novel therapies to treat ophthalmic diseases, with a
focus on age-related and orphan retinal diseases. For more information,
please visit

Forward-looking Statements

Any statements in this press release about Ophthotech's future
expectations, plans and prospects constitute forward-looking statements
for purposes of the safe harbor provisions under the Private Securities
Litigation Reform Act of 1995. Forward-looking statements include any
statements about Ophthotech's strategy, future operations and future
expectations and plans and prospects for Ophthotech, and any other
statements containing the words "anticipate," "believe," "estimate,"
"expect," "intend", "goal," "may", "might," "plan," "predict,"
"project," "target," "potential," "will," "would," "could," "should,"
"continue," and similar expressions. In this press release, Ophthotech's
forward-looking statements include statements about the implementation
of its strategic plan, the timing, progress and results of clinical
trials and other research and development activities and the potential
utility of its product candidates. Such forward-looking statements
involve substantial risks and uncertainties that could cause
Ophthotech's preclinical and clinical development programs, future
results, performance or achievements to differ significantly from those
expressed or implied by the forward-looking statements. Such risks and
uncertainties include, among others, those related to the initiation and
the conduct and design of research programs and clinical trials,
availability of data from these programs, expectations for regulatory
matters, need for additional financing and other factors discussed in
the "Risk Factors" section contained in the quarterly and annual reports
that Ophthotech files with the Securities and Exchange Commission. Any
forward-looking statements represent Ophthotech's views only as of the
date of this press release. Ophthotech anticipates that subsequent
events and developments will cause its views to change. While Ophthotech
may elect to update these forward-looking statements at some point in
the future, Ophthotech specifically disclaims any obligation to do so
except as required by law.


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