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AskBio Partners with TPG and Vida Ventures

Leading gene therapy company announces $235 million investment to
accelerate growth

RESEARCH TRIANGLE PARK, N.C.& FORT WORTH, Texas & SAN FRANCISCO & BOSTON–(BUSINESS WIRE)–Asklepios BioPharmaceutical, Inc (AskBio), a developer and manufacturer
of Adeno-Associated Virus (AAV) gene therapy therapeutics for
underserved patient populations with rare and generally untreatable
genetic disorders, announced today $235 million in funding to augment
and accelerate its business. TPG Capital and Vida Ventures are investing
$225 million for a minority stake in the company. As part of the
transaction, AskBio’s founders and board members are co-investing $10
million. The investment provides AskBio with additional capital to help
advance and expand clinical trials, enhance its manufacturing
capabilities and capacity, and drive long-term growth. Additional terms
of the transaction were not disclosed.

AskBio was founded in 2001 by Dr. Jude Samulski, Ph.D., the first
scientist to clone AAV, along with Dr. Xiao Xiao, who was the first to
develop a miniaturized dystrophin gene enabling the advancement of gene
therapy for Duchenne Muscular Dystrophy, and company CEO Sheila Mikhail,
an accomplished life sciences executive. Formed in the Gene Therapy
Center at the University of North Carolina at Chapel Hill, AskBio offers
proprietary AAV technology, best-in-class manufacturing capabilities,
and a robust pipeline of in-house clinical programs. An early innovator
in the space, the company holds over 500 patents in areas such as AAV
production, chimeric vectors, and self-complementary DNA. AskBio’s
engineered viral vectors are able to target particular tissues,
de-target other tissues, and minimize the effects of neutralizing
antibodies.

“It’s humbling to imagine that my Ph.D. project several decades ago has
ushered in a new generation of transformative therapies that will impact
so many lives,” said Dr. Jude Samulski, Ph.D., President and Chief
Scientific Officer of AskBio. “I am excited to partner with TPG and Vida
to catalyze the next generation of gene therapy innovation and translate
discovery into life changing therapeutics.”

“With the funding from TPG and Vida, we will be able to accelerate our
development of a broad range of transformative therapies for those
affected by serious and oftentimes incurable genetic diseases,” said
Sheila Mikhail, CEO and Co-Founder of AskBio. “We look forward to
advancing our approaches for repeat administration and avoidance of
neutralizing antibodies into the clinic to maximize the number of
patients who benefit from AAV therapies.”

Gene therapy has been one of the most active areas of pharmaceutical
R&D, and in recent years, AAV has emerged as an efficient, safe, and
versatile vehicle for delivering corrective genes inside of the body.
AskBio has successfully partnered with companies on two of its programs:
Bamboo Therapeutics, a therapy targeting Duchenne Muscular Dystrophy and
other rare diseases, and Chatham Therapeutics, which focuses on
Hemophilia A and B. The platforms were sold to Pfizer and Baxter
(programs now owned by Takeda), respectively.

“We are thrilled to partner with Jude, Sheila, and the entire AskBio
team to support the development of therapies for patients suffering from
historically untreatable diseases,” said John Schilling, M.D., Partner
at TPG Capital. “AskBio is a very special company, operating in one of
the most innovative and strategically important sectors of the
pharmaceutical industry today. In addition to its leading clinical
programs in several therapeutic areas, AskBio has a broad base of
technologies that we believe can support quantum leaps in therapies for
many partners in the market. We are honored to have the opportunity to
work with AskBio in the next leg of this journey.”

AskBio recently advanced a therapeutic for Pompe Disease into the clinic
based on the work by Dwight Koeberl, M.D., Ph.D., and Priya Kishnani,
M.D., M.B.B.S., at Duke University. The company is also developing
therapeutics for heart failure, Limb Girdle 2i, Myotonic Dystrophy, and
several other muscular and neurological disorders. AskBio’s AAV
manufacturing process, Pro10, currently yields 1018 in
200-liter bioreactors and is recognized within the industry as the gold
standard in scale manufacturing. Pro10 is used by Pfizer, Takeda, and
Viralgen Vector Core SA. Recently, AskBio formed a partnership with
Touchlight Genetics Ltd. for the commercialization of a safer, cheaper,
and more efficient alternative to plasmid DNA.

“At Vida, we are driven to find innovative companies with disruptive
technologies that have the potential to transform how we combat
disease,” said Fred Cohen, M.D., Ph.D., Co-Founder and Senior Managing
Director at Vida Ventures. “Jude’s acclaimed research in AAV and
scientific vision for AskBio, along with the leadership of Sheila and
the research expertise of the larger team, exemplify what’s possible
when passionate scientists and advocates demand more than conventional
therapies.”

Some of TPG Capital’s past and current investments in the pharmaceutical
industry include Par Pharmaceutical (now known as Endo International),
Adare Pharmaceuticals, Amneal Pharmaceuticals, Aptalis, EnvisionRx,
Quintiles, and IMS (together, now known as IQVIA). Last year, TPG
Capital and Vida partnered to invest in Allogene Therapeutics, Inc
(NASDAQ: ALLO). Vida’s recent biotechnology investments include Kronos
Bio, Praxis Precision Medicines, Oyster Point Pharma, and Pionyr
Immunotherapeutics.

Piper Jaffray served as financial advisor to AskBio in connection with
this transaction.

About AskBio

Asklepios BioPharmaceutical, Inc. (AskBio) is a privately held, gene
therapy platform company dedicated to improving the lives of children
and adults with rare genetic disorders. AskBio’s gene therapy platform
includes an industry-leading proprietary cell line manufacturing process
known as Pro10 and an extensive AAV capsid library. The company has
generated hundreds of proprietary third generation gene vectors that
have entered clinical testing, and maintains a portfolio of clinical
programs across a range of indications including Pompe, Limb Girdle
Muscular Dystrophy, Cystic Fibrosis, Myotonic Muscular Dystrophy,
Huntington’s, Hemophilia (Chatham Therapeutic/Shire) and Duchenne
Muscular Dystrophy (Bamboo Therapeutics/Pfizer). For more information,
visit www.askbio.com.

About TPG

TPG is a leading global alternative asset firm founded in 1992 with more
than $104 billion of assets under management and offices in Austin,
Beijing, Boston, Dallas, Fort Worth, Hong Kong, Houston, London,
Luxembourg, Melbourne, Moscow, Mumbai, New York, San Francisco, Seoul,
and Singapore. TPG’s investment platforms are across a wide range of
asset classes, including private equity, growth venture, real estate,
credit, and public equity. TPG aims to build dynamic products and
options for its investors while also instituting discipline and
operational excellence across the investment strategy and performance of
its portfolio. For more information, visit www.tpg.com

About Vida Ventures

Vida Ventures is a next-generation life sciences investment firm founded
in 2017 by a group of scientists, physicians, entrepreneurs and
investors passionate about building and funding breakthroughs in
biomedicine. Together they form an independent, bold investment group
bound together by a simple word – life. Its mission is to bring science
to life and advance transformative biomedical innovations that have the
potential to make a meaningful difference for patients. Vida has a
bicoastal presence and currently manages approximately $355 million. For
more information on Vida Ventures, please visit www.vidaventures.com,
on LinkedIn or follow on Twitter @Vida_Ventures.

Contacts

Media Contacts
TPG Capital
Luke Barrett
415-743-1550
media@tpg.com

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