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Pfizer Aims to Become Industry Leader in Gene Therapy with Acquisition of Bamboo Therapeutics, Inc.

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Monday, August 1st 2016 at 2:00pm UTC

Acquisition combines Bamboo’s gene therapy portfolio, advanced vector
design, and production technologies and capabilities with Pfizer’s
global scale, research, development and commercialization experience

NEW YORK–(BUSINESS WIRE)– Pfizer Inc. (NYSE:PFE) today announced that it has acquired Bamboo
Therapeutics, Inc., a privately held biotechnology company based in
Chapel Hill, N.C., focused on developing gene therapies for the
potential treatment of patients with certain rare diseases related to
neuromuscular conditions and those affecting the central nervous system.
This acquisition significantly expands Pfizer’s expertise in gene
therapy by providing Pfizer with a clinical and several pre-clinical
assets that complement the company’s rare disease portfolio, an advanced
recombinant Adeno-Associated Virus (rAAV) vector design and production
technology, and a fully functional Phase I/II gene therapy manufacturing
facility that Bamboo acquired from the University of North Carolina
earlier this year.

Gene therapy is an emerging area of medical research focused on highly
specialized, one-time, transformative treatments addressing the root
cause of diseases caused by genetic mutation. Gene therapy is a
promising investigational technology, especially for patients with rare
diseases, many of which are caused by a single genetic mutation. The
technology involves introducing genetic material into the body to
deliver a corrected copy of a gene to a patient’s cells to compensate
for a defective one. The genetic material can be delivered to the cells
by a variety of means, most frequently using a viral vector such as
rAAV. There have been no gene therapy products approved in the U.S. to
date.

“The field of gene therapy research has made tremendous strides in
recent years, and we are pleased to be able to further enhance our
leadership position in this area through this transaction with Bamboo,”
said Mikael Dolsten, President, Pfizer Worldwide Research & Development.
“We believe that gene therapy may hold the promise of bringing true
disease modification for patients suffering from devastating diseases,
and we hope to see this promise come to fruition – through new and
existing in-house capabilities and potential partnership opportunities –
in the years to come.”

Bamboo’s portfolio includes potential best-in-class rAAV-based gene
therapies that will complement Pfizer’s rare disease and gene therapy
portfolios in two priority areas: neuromuscular, with a pre-clinical
asset for Duchenne Muscular Dystrophy (DMD); and central nervous system,
with pre-clinical assets for Friedreich’s Ataxia and Canavan disease,
and a Phase I asset for Giant Axonal Neuropathy.

Bamboo’s approximately 11,000-square foot, fully staffed and operational
manufacturing facility has experience producing Phase I/II materials
using a superior suspension, cell-based production platform that
increases scalability, efficiency and purity. This helps enable the DMD
program and other projects requiring large amounts of rAAV. The
facility, previously known as the University of North Carolina Vector
Core facility, has served as a qualified supplier of rAAV vectors for
several healthcare companies and academic institutions.

“We believe Bamboo’s industry leading capabilities in rAAV vector design
and manufacturing complement Pfizer’s rare disease strategy and help
advance Pfizer’s mission to deliver life-changing innovation to patients
with the greatest needs,” said Gregory LaRosa, Chief Scientific Officer,
Rare Disease Research Unit, Pfizer. “Bringing together Pfizer and Bamboo
colleagues’ deep scientific understanding of both rAAV biology and
complex biologic manufacturing will help position us for success in this
area. We are pleased to welcome Bamboo colleagues to Pfizer and look
forward to working together on transformative gene therapies for
patients in areas of high unmet medical need.”

Jude Samulski, Chief Scientific Officer and Executive Chairman of Bamboo
and a leading expert in the field of rAAV vectors with more than 25
years of experience, will be joining Pfizer. Dr. Samulski, together with
the Bamboo team, will play a key role in helping to develop and
accelerate Pfizer’s capabilities in gene therapy.

“We are pleased to begin working with Pfizer, as this represents a
significant step toward bringing Bamboo’s portfolio into the clinic and
ultimately potential new medicines to patients,” Dr. Samulski said.

Pfizer previously acquired approximately 22 percent of Bamboo’s fully
diluted equity during the first quarter of 2016 for a payment of
approximately $43 million. Under the terms of this transaction, Pfizer
acquired all of Bamboo’s remaining equity for an upfront payment of $150
million, and Bamboo’s selling shareholders will be eligible for
potential milestone payments of up to $495 million contingent upon the
progression of key assets through development, regulatory approval and
commercialization. Following the acquisition, Bamboo is now a
wholly-owned subsidiary of Pfizer.

Kaye Scholer LLP acted as Pfizer’s legal advisor for the transaction and
Ice Miller LLP served as Bamboo’s legal advisor.

Pfizer’s Investments in Gene Therapy

Pfizer is committed to becoming an industry leader in the field of gene
therapy, which holds tremendous promise to deliver highly specialized,
one-time, transformative therapies to patients in areas of high unmet
medical need, particularly in rare, monogenic diseases with loss of
function. To support this commitment, the company has been making
investments in gene therapy for the past several years and has sought to
bring together the foremost expertise in rAAV vector design and
development with partnerships, in-house knowledge of disease biology,
manufacturing and analytical capabilities.

In 2014, Pfizer established within the company’s Rare Disease Research
Unit the Genetic Medicines Institute (GMI) in London, UK, which is a
dedicated gene therapy research group under the direction of leading
gene therapy researcher Michael Linden. Dr. Linden and the GMI are
charged with identifying potential gene therapy projects and supporting
them through scientific discovery, process development and translational
advancement.

In addition to the London-based GMI investment, Pfizer and
Philadelphia-based Spark Therapeutics established a collaboration on a
clinical program for SPK-9001, which is being investigated as a
one-time treatment for hemophilia B that incorporates a bio-engineered
rAAV vector. Initial data from an ongoing Phase I/II trial for this
treatment has shown promising early results, and SPK-9001 has
received breakthrough therapy designation by the U.S. Food and Drug
Administration.

Pfizer has research agreements with several leading academic
institutions, including an agreement with King’s College London for the
development of a series of rAAV gene therapy vectors, and an agreement
with the University of Iowa Research Foundation for the development of a
potential gene therapy for cystic fibrosis through the University of
Iowa laboratories.

Pfizer also entered into a collaboration and license agreement with
Emeryville, Calif.-based 4D Molecular Therapeutics (4DMT) to discover
and develop targeted next-generation rAAV vectors for cardiac disease.
In addition, Pfizer made an equity investment in 4DMT in October 2015.

About Pfizer and Rare Diseases

Rare diseases are among the most serious of all illnesses and impact 350
million patients worldwide, often children. Although there are over
7,000 known rare diseases, only five percent have an approved
medication. For rare disease patients and their loved ones, better
treatment options cannot come soon enough. At Pfizer, we share their
urgency and passionately dedicate our resources, expertise and global
reach to bring them the transformative medicines they need. The Pfizer
focus on rare diseases builds on more than two decades of experience, a
pipeline of more than 20 compounds and a global portfolio of more than
20 medicines approved worldwide that treat rare diseases in the areas of
hematology, neuroscience, inherited metabolic disorders, and
pulmonology. Pfizer Rare Disease is inspired by patients, born from
science and powered by the passion of the hundreds of colleagues in
Pfizer who dedicate their work to helping patients with rare diseases.

Pfizer Inc.: Working together for a healthier world®

At Pfizer, we apply science and our global resources to bring therapies
to people that extend and significantly improve their lives. We strive
to set the standard for quality, safety and value in the discovery,
development and manufacture of healthcare products. Our global portfolio
includes medicines and vaccines as well as many of the world’s
best-known consumer healthcare products. Every day, Pfizer colleagues
work across developed and emerging markets to advance wellness,
prevention, treatments and cures that challenge the most feared diseases
of our time. Consistent with our responsibility as one of the world’s
premier innovative biopharmaceutical companies, we collaborate with
health care providers, governments and local communities to support and
expand access to reliable, affordable health care around the world. For
more than 150 years, Pfizer has worked to make a difference for all who
rely on us. For more information, please visit us at www.pfizer.com.
In addition, to learn more, follow us on Twitter at @Pfizer and
@Pfizer_News, LinkedIn, YouTube and like us on Facebook at Facebook.com/Pfizer.

PFIZER DISCLOSURE NOTICE

The information contained in this release is as of August 1, 2016.
Pfizer assumes no obligation to update forward-looking statements
contained in this release as the result of new information or future
events or developments.

This release contains forward-looking information related to Pfizer,
Bamboo and the acquisition of Bamboo by Pfizer that involves substantial
risks and uncertainties that could cause actual results to differ
materially from those expressed or implied by such statements.
Forward-looking statements in this release include, among other things,
statements about the potential benefits of the acquisition, Pfizer’s and
Bamboo’s plans, objectives, expectations and intentions, the business of
Pfizer and Bamboo, and Pfizer’s and Bamboo’s rare disease or gene
therapy portfolio, technologies and manufacturing capabilities. Risks
and uncertainties include, among other things, risks related to the
ability to realize the anticipated benefits of the acquisition,
including the possibility that the expected benefits from the
acquisition will not be realized or will not be realized within the
expected time period; the risk that the businesses will not be
integrated successfully; unknown liabilities; other business effects,
including the effects of industry, market, economic, political or
regulatory conditions; future exchange and interest rates; changes in
tax and other laws, regulations, rates and policies; the uncertainties
inherent in research and development, including the ability to meet
anticipated pre-clinical and clinical study commencement and completion
dates as well as the possibility of unfavorable study results, including
unfavorable new data and additional analyses of existing data; risks
associated with initial data; whether and when any applications may be
filed with regulatory authorities for any potential rare disease or gene
therapy product; whether and when

regulatory authorities may approve any such applications, which will
depend on the assessment by such regulatory authorities of the
benefit-risk profile suggested by the totality of the efficacy and
safety information submitted; decisions by regulatory authorities that
could affect the availability or commercial potential of any such rare
disease or gene therapy product; and competitive developments.

A further description of risks and uncertainties can be found in
Pfizer’s Annual Report on Form 10-K for the fiscal year ended December
31, 2015 and in its subsequent reports on Form 10-Q, including in the
sections thereof captioned “Risk Factors” and “Forward-Looking
Information and Factors That May Affect Future Results”, as well as in
its subsequent reports on Form 8-K, all of which are filed with the U.S.
Securities and Exchange Commission and available at 
www.sec.gov and www.pfizer.com.

Contacts

Pfizer Inc.
Media:
Dean Mastrojohn, 212-733-6944
dean.mastrojohn@pfizer.com
or
Investors:
Ryan
Crowe, 212-733-8160
ryan.crowe@pfizer.com

Source: Pfizer Inc.

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Acquisition of Bamboo Therapeutics, Inc.
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