Shire Acquires Lotus Tissue Repair, Inc.

Lexington, Massachusetts (ots/PRNewswire) -

- HGT Pipeline Enhanced with Protein Replacement Therapy Being
Investigated for the Treatment of Dystrophic Epidermolysis Bullosa

Shire plc , announces that it has signed an agreement to acquire
Lotus Tissue Repair, Inc. of Cambridge, MA, a privately held
biotechnology company developing the first and only protein
replacement therapy currently being investigated for the treatment of
dystrophic epidermolysis bullosa (DEB). DEB is a devastating orphan
disease for which there is no currently approved treatment option
other than palliative care. Subject to customary government
approvals, Shire will purchase the company for an upfront payment and
certain contingent payments based on the achievement of certain
safety and development milestones.

Epidermolysis Bullosa (EB) is a set of rare, genetic diseases
characterized by the presence of extremely fragile skin and recurrent
blister formation resulting from minor mechanical friction or trauma.
DEB is one of the more severe of the genetic disorders that comprise
EB. Severe cases of DEB may also include internal blistering of the
mouth, esophagus, lower GI tract, upper airway and GU tract.

Shire's Human Genetic Therapies business will undertake the
further development of Lotus Tissue Repair's lead product candidate,
a proprietary recombinant form of human collagen Type VII (rC7), an
intravenous protein replacement therapy for the treatment of DEB. The
product is in late pre-clinical development and has the potential to
be a first-in-class systemic therapy for the treatment of DEB. This
acquisition expands Shire's commitment to finding treatments for EB,
which also includes ABH001, Shire's Regenerative Medicine product
currently being investigated as a dermal substitute therapy for the
treatment of non-healing wounds in patients with EB.

"DEB is one the most devastating orphan diseases, severely
impacting the lives of patients and their families, many of whom have
few or no treatment options other than palliative care," said Dr.
Philip J. Vickers, Global Head of Research and Development, Shire
Human Genetic Therapies. "rC7 protein replacement therapy has the
potential to provide a first-in-class disease-modifying treatment for
these children. We plan to apply our proven ability to develop
protein replacement therapies for rare genetic diseases to progress
rC7 as a possible groundbreaking treatment that offers hope to
patients with DEB."

"This acquisition of Lotus Tissue Repair by Shire further
underscores the potential of this proprietary rC7 technology to
dramatically change the treatment landscape for DEB patients and
their families," said Dr. Mark de Souza, founding Chief Executive
Officer of Lotus Tissue Repair. "We are thrilled that this program
will become part of the innovative pipeline at Shire and the
company's growing commitment to this patient community."

Lotus Tissue Repair is a private company launched in 2011 by a
proven team of biotechnology entrepreneurs, world-leading experts in
rC7 protein replacement therapy for DEB and top-tier life sciences
investor, Third Rock Ventures.

About Type VII Collagen

Type VII collagen (C7) is a collagen found in the skin of humans
and other animals. It is localized in the basement membrane zone
(BMZ) between the epidermis and dermis in large structures called
Anchoring Fibrils (AFs), which are composed of C7. The AFs hold the
epidermis and dermis together. Purified human C7 can be extracted in
very small quantities from human skin, human amnion, and skin cells
such as keratinocytes or dermal fibroblasts. However, large amounts
of C7 cannot be generated from these sources in this manner.

About Recombinant Collagen VII (rC7)

Dr. Mei Chen and Dr. David Woodley, co-founders of Lotus Tissue
Repair Inc., are the co-inventors of recombinant collagen type VII
(rC7) technology and leading experts on its use as protein
replacement therapy. Lotus is developing its rC7 technology as the
first and only protein replacement therapy for dystrophic
epidermolysis bullosa (DEB) [http://lotustissuerepair.com/DEB.php ].
This approach directly addresses a primary driver of the condition:
deficiency or dysfunction of C7. Pre-clinical findings to date are
promising, showing in multiple pre-clinical models that rC7 as a
protein replacement therapy is potent, long-lasting, and is
specifically retained in the skin and other affected tissues after
intravenous injection.

About ABH001

ABH001 is an engineered, human fibroblast-derived dermal
substitute generated by culturing human neonatal dermal fibroblasts
onto a bioresorbable polyglactin mesh scaffold. The PGLLA mesh which
serves as the scaffolding onto which fibroblasts are grown, they
secrete dermal collagen, other extracellular matrix proteins, growth
factors, and cytokines, creating a three-dimensional human tissue
containing metabolically active living cells. The final product
consists of a well-developed dermal matrix and evenly dispersed
neonatal dermal fibroblasts.

About DEB

Epidermolysis bullosa (EB) is a set of rare, genetic diseases
characterized by the presence of extremely fragile skin and recurrent
blister formation resulting from minor mechanical friction or trauma.
Dystrophic epidermolysis bullosa (DEB) is one of the more severe
forms of the genetic disorders that comprise EB and is caused by a
deficiency or dysfunctionality of collagen type VII. The recessive
form of DEB is extremely painful and patients suffer from severe skin
blistering, extremely fragile skin and mucosa of the mouth,
esophagus, and anus, mutilating scarring of the hands and feet, joint
contractures, and strictures of the esophagus. In the second or third
decade of life, these patients develop aggressive squamous cell
carcinomas in chronically wounded areas that often lead to metastasis
and death. There are no adequate treatments available for this
painful, debilitating and costly disease.

Notes to editors

Shire enables people with life-altering conditions to lead better
lives.

Through our deep understanding of patients' needs, we develop and
provide healthcare in the areas of:

- Behavioral Health and Gastro Intestinal conditions
- Rare Diseases
- Regenerative Medicine

as well as other symptomatic conditions treated by specialist
physicians.

We aspire to imagine and lead the future of healthcare, creating
value for patients, physicians, policymakers, payors and our
shareholders.

"SAFE HARBOR" STATEMENT UNDER THE PRIVATE SECURITIES LITIGATION
REFORM ACT OF 1995

Statements included herein that are not historical facts are
forward-looking statements. Such forward-looking statements involve a
number of risks and uncertainties and are subject to change at any
time. In the event such risks or uncertainties materialize, the
Company's results could be materially adversely affected. The risks
and uncertainties include, but are not limited to, risks associated
with: the inherent uncertainty of research, development, approval,
reimbursement, manufacturing and commercialization of the Company's
Specialty Pharmaceuticals, Human Genetic Therapies and Regenerative
Medicine products, as well as the ability to secure new products for
commercialization and/or development; government regulation of the
Company's products; the Company's ability to manufacture its products
in sufficient quantities to meet demand; the impact of competitive
therapies on the Company's products; the Company's ability to
register, maintain and enforce patents and other intellectual
property rights relating to its products; the Company's ability to
obtain and maintain government and other third-party reimbursement
for its products; and other risks and uncertainties detailed from
time to time in the Company's filings with the Securities and
Exchange Commission.

For further information please contact:

Investor Relations
Eric Rojas erojas@shire.com +1-781-482-0999
Sarah Elton-Farr seltonfarr@shire.com +44(0)1256-894157
Media
Jessica Mann (Corporate) jmann@shire.com +44(0)1256-894-280
Jessica Cotrone (Human
Genetic Therapies) jcotrone@shire.com +1-781-482-9538

ots Originaltext: Shire Pharmaceuticals Group Plc
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