Fibrocell Science Receives Orphan Drug Designation for Genetically-Modified Autologous Human Fibroblasts to Advance Treatment

  Fibrocell Science Receives Orphan Drug Designation for Genetically-Modified
  Autologous Human Fibroblasts to Advance Treatment Development for Recessive
  Dystrophic Epidermolysis Bullosa (RDEB)

 RDEB recognized as most severe form of all epidermolysis bullosa conditions

Business Wire

EXTON, Pa. -- June 13, 2014

Fibrocell Science, Inc., (NYSE MKT:FCSC), an autologous cell therapy company
primarily focused on developing first-in-class treatments for rare and serious
skin and connective tissue diseases with high unmet medical needs, today
announced that the U.S. Food and Drug Administration (FDA) has granted orphan
drug designation to its genetically-modified autologous human fibroblasts to
treat dystrophic epidermolysis bullosa (DEB), a broad category of rare and
severe genetic skin diseases that includes RDEB. Fibrocell is working with
leading medical centers—whose research focuses on all forms of epidermolysis
bullosa, including DEB and RDEB—to advance development of its lead therapeutic
candidate for the most severe form of epidermolysis bullosa, RDEB.

Orphan Drug Designation is granted by the FDA Office of Orphan Drug Products
to drugs intended to treat a rare disease or condition affecting fewer than
200,000 people in the U.S. There are between 2,800 and 5,600 RDEB patients in
the U.S. and typically, they do not live beyond 30 years of age. No cure or
treatment is available for this disease beyond basic wound care.

The genetically-modified fibroblasts combine Fibrocell’s autologous fibroblast
technology with Intrexon Corporation’s (NYSE:XON) UltraVector® synthetic
biology platform to target the fundamental source of this devastatingly
debilitating, genetic disease that causes severe blistering and areas of
missing skin.

“The fibroblast cells of RDEB patients lack the ability to produce functional
collagen VII, the essential protein that forms anchoring fibrils responsible
for attaching the layers of the skin together,” said David Pernock, chairman
and chief executive officer of Fibrocell. “In collaboration with Intrexon, the
gene that encodes functional collagen VII can be inserted into the patient’s
own fibroblast cells and administered as a therapeutic solution. Our goal is
to treat the underlying genetic cause of the disease and relieve the suffering
of these patients. We are working with leading EB centers to complete
proof-of-concept animal work, and look forward to filing our IND in the first
half of 2015 and initiating a Phase 1 trial.”

Suma Krishnan, senior vice president of product development at Intrexon,
added, “We are excited to see continued progress within the Fibrocell
Exclusive Channel Collaboration program. The collaborative effort between the
Intrexon and Fibrocell teams has helped realize thisimportant stage in
developing a cell-based therapy for the unmet need of patients with RDEB. We
look forward to the next steps in advancing the RDEB program to the clinic.”

About Fibrocell Science, Inc.

Fibrocell Science, Inc. (NYSE MKT:FCSC) is an autologous cell therapy company
primarily focused on developing first-in-class treatments for rare and serious
skin and connective tissue diseases with high unmet medical needs. Based on
its proprietary autologous fibroblast technology, Fibrocell is pursuing
breakthrough medical applications of azficel-T for restrictive burn scarring
and vocal cord scarring. The company’s collaboration with Intrexon Corporation
(NYSE:XON), a leader in synthetic biology, includes using genetically-modified
fibroblasts for treating orphan skin diseases for which there are no currently
approved products. For additional information, visit www.fibrocellscience.com.

Forward-Looking Statements

This press release contains, and our officers and representatives may from
time to time make, statements that are “forward-looking statements” within the
meaning of the safe harbor provisions of the U.S. Private Securities
Litigation Reform Act of 1995. Examples of forward-looking statements include,
among others, statements we make regarding our ability to develop breakthrough
therapies for the treatment of skin and connective tissues diseases, and to
expand our proprietary Personalized Biologics platform. These forward-looking
statements rely on a number of assumptions concerning future events and are
subject to a number of risks, uncertainties, and other factors, many of which
are outside of Fibrocell Science’s control. Important factors that could cause
our actual results and financial condition to differ materially from those
indicated in the forward-looking statements include, among others, the
following: (i) uncertainties relating to the initiation and completion of
clinical trials; (ii) whether clinical trial results will validate and support
the safety and efficacy of azficel-T; and (iii) our ability to establish
additional strategic partnerships, as well as those set forth under the
caption “Item 1A. Risk Factors” in Fibrocell Science’s most recent Form 10-K
filing, as updated in “Item 1A. Risk Factors” in Fibrocell Science’s most
recent Form 10-Q filing. Any forward-looking statement made by us in this
press release is based only on information currently available to us and
speaks only as of the date on which it is made. In addition, Fibrocell Science
operates in a highly competitive and rapidly changing environment, and new
risks may arise. Accordingly, you should not place any reliance on
forward-looking statements as a prediction of actual results. Fibrocell
Science disclaims any intention to, and undertakes no obligation to, update or
revise any forward-looking statement. You are also urged to carefully review
and consider the various disclosures in Fibrocell Science’s most recent annual
report on Form 10-K, our most recent Form 10-Q as well as other public filings
with the SEC since the filing of Fibrocell Science’s most recent annual
report.

Contact:

Corporate:
Fibrocell Science, Inc.
Gregory Weaver, +1-484-713-6000
Senior Vice President and Chief Financial Officer
gweaver@fibrocellscience.com
or
Investor Relations:
S.A. Noonan Communications, LLC
Susan Noonan, 212-966-3650
susan@sanoonan.com
 
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