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Ophthotech Obtains Exclusive Global License to AAV Gene Therapy Program for BEST1 Related Retinal Diseases

NEW YORK–(BUSINESS WIRE)–Ophthotech
Corporation
(NASDAQ: OPHT) announced today that the Company has
converted its option and entered into an exclusive global license
agreement with the University of Pennsylvania (Penn), including the
Perelman School of Medicine at the University of Pennsylvania and the
University of Pennsylvania School of Veterinary Medicine, and the
University of Florida Research Foundation (UFRF) for rights to develop
and commercialize novel adeno-associated virus (AAV) gene therapy
product candidates for the treatment of Best vitelliform macular
dystrophy, also known as Best disease and other bestrophinopathies,
which are diseases related to mutations to the BEST1 gene.

Best disease, which generally affects individuals in both eyes, is an
orphan inherited retinal disease. In October 2018, Ophthotech entered
into an exclusive option agreement with Penn and UFRF for rights to
negotiate to acquire a license for novel AAV serotype 2 based gene
therapy product candidates for the treatment of Best disease. Since that
time, Ophthotech has sponsored research at Penn, facilitated by the Penn
Center for Innovation (PCI), to conduct preclinical and natural history
studies for Best disease and commenced IND-enabling activities. Based on
current timelines and subject to regulatory review, the Company expects
to initiate a Phase 1/2 clinical trial in the first half of 2021.

Ophthotech estimates that approximately 10,000 individuals in the United
States and the five major European markets on a combined basis have Best
disease. Patients with Best disease develop an egg yolk-like vitelliform
lesion in their macular region, which over time leads to macular atrophy
and permanent loss of central vision. There is currently no FDA or EMA
approved therapy to treat this orphan inherited degenerative retinal
disease.

Proof-of-concept studies demonstrating that the AAV-BEST1 gene
therapy product candidate restored the anatomy between photoreceptors
and retinal pigment epithelial (RPE) cells in the naturally occurring
canine disease model with distinct phenotypic similarities to human
bestrophinopathies were published in March 2018 in the journal Proceedings
of the National Academy of Sciences
(PNAS), titled: “BEST1
gene therapy corrects a diffuse retina-wide microdetachment modulated by
light exposure
.

About Ophthotech Corporation

Ophthotech is a biopharmaceutical company specializing in the
development of transformative gene therapies and novel therapeutics to
treat retinal diseases, with a focus on orphan and age related
indications. For more information, please visit www.ophthotech.com.

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 negotiation and consummation
of in-license and/or acquisition transactions 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.

OPHT-G

Contacts

Investors
Kathy Galante
Ophthotech Corporation
Vice
President, Investor Relations and Corporate Communications
212-845-8231
[email protected]

Media
Alex
Van Rees, 973-442-1555 ext. 111
SmithSolve LLC on behalf
of Ophthotech Corporation
[email protected]

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