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Third Rock Ventures Launches Voyager Therapeutics with $45 Million Series A to Develop Life-Changing Gene Therapies for CNS

  Third Rock Ventures Launches Voyager Therapeutics with $45 Million Series A
  to Develop Life-Changing Gene Therapies for CNS Disorders

Company Assembles Leading Clinical and Scientific Founders, Multiple Clinical
  and Preclinical Product Programs, and Key Strategic Technology Agreements

Business Wire

CAMBRIDGE, Mass. -- February 12, 2014

Third Rock Ventures, LLC today announced the formation of Voyager
Therapeutics, a gene therapy company developing life-changing treatments for
fatal and debilitating diseases of the central nervous system (CNS), with a
$45 million Series A financing. Voyager’s adeno-associated virus (AAV)
approach to gene therapy has the potential to transform treatment for a wide
range of CNS diseases with one-time therapies that may dramatically improve
patients’ lives. The company is committed to advancing the field of AAV gene
therapy by innovating and investing in areas such as vector optimization and
engineering, dosing techniques, as well as process development and production.

“We believe the time is right for gene therapy, and we have assembled the
expertise, technology and strategies to convert the promise of AAV gene
therapy into meaningful treatments for patients,” said Mark Levin, Interim
Chief Executive Officer of Voyager and Partner at Third Rock. “Our world-class
founders have driven significant advances in their respective fields, and the
combination of their deep clinical and scientific knowledge, our experienced
management team and Third Rock’s investment positions Voyager to deliver
breakthrough therapies to patients suffering from devastating CNS disorders.”

Voyager was founded by world leaders in the fields of AAV gene therapy,
expressed RNA interference (RNAi) and neuroscience:

  *Krystof Bankiewicz, M.D., Ph.D., is a translational neurosurgeon and
    leader in AAV gene therapy who has brought multiple new AAV therapies to
    the clinic; he is the Kinetics Foundation Chair in Translational Research
    and a Professor in Residence of Neurological Surgery and Neurology at the
    University of California, San Francisco (UCSF).
  *Guangping Gao, Ph.D., is a leading AAV researcher who has played a key
    role in the discovery and characterization of new AAV serotypes; he is
    Director of theUniversity of Massachusetts Medical School (UMMS) Gene
    Therapy Center& Vector Core, Scientific Director of the UMMS-China
    Program Office and Professor of Microbiology and Physiology Systems and
    the Penelope Booth Rockwell Professor in Biomedical Research at UMMS.
  *Mark Kay, M.D., Ph.D., is a leading researcher in the fields of gene
    therapy and AAV biology, including the identification of new AAV capsids;
    he is the Head of the Division of Human Gene Therapy and Professor in the
    Departments of Pediatrics and Genetics at Stanford University School of
    Medicine; he is one of the founders of the American Society of Gene and
    Cell Therapy and is currently the Editor of Human Gene Therapy.
  *Phillip Zamore, Ph.D., is a leader and innovator in the understanding of
    RNAi and development of related therapeutic approaches, including
    expressed RNAi; he is a Howard Hughes Medical Institute Investigator, the
    Gretchen Stone Cook Chair of Biomedical Sciences at UMMS, Professor of
    Biochemistry and Molecular Pharmacology and Co-Director of the RNA
    Therapeutics Institute at UMMS.

Voyager is launching with multiple clinical and preclinical product programs
for CNS diseases in dire need of effective new therapies, including a
Parkinson’s disease program in an ongoing Phase 1b study with our
collaborators at UCSF, as well as preclinical programs for a monogenic form of
amyotrophic lateral sclerosis (ALS) and Friedreich’s ataxia. Depending on the
disease, the company’s gene therapy products will use either gene replacement
or gene knockdown techniques. By significantly increasing or decreasing
production of relevant proteins at targeted sites within the CNS, the goal is
to address the underlying biology of the disease and make a meaningful
difference for patients.

“CNS disorders are one of the largest areas of unmet medical need,” said
Philip Reilly, M.D., J.D., Interim Chief Medical Officer of Voyager and
Venture Partner at Third Rock. “By expressing the right amount of protein in
the right cell at the right time or knocking down a gene to decrease levels of
a disease-causing protein, we believe our gene therapy approach is uniquely
suited to targeting these devastating diseases and has the potential to
fundamentally change the face of treatment for many patients.”

Voyager has entered intolicenseand other agreements with UMMS, UCSF and
Stanford University to access relevant technology and data, including certain
rights to intellectual property related to expressed RNAi technology
discovered at UMMS, clinical data related to the Parkinson’s disease program
from UCSF, and certain rights to AAV intellectual property developed at
Stanford. In order to accelerate its research and development efforts, Voyager
has also entered into a broad strategic collaboration with UMMS, a leader in
AAV research and translation.

Over the past 10 years, AAV has emerged as a highly promising and attractive
approach to gene therapy. In clinical trials, AAV has been shown to be a safe
and effective gene therapy delivery vehicle. Advances in AAV vector
development and related dosing techniques that enable widespread gene delivery
in the brain and spinal cord have made AAV particularly well-suited for the
treatment of CNS diseases. In addition, improvements in related production
technology and approaches have made AAV production more easily scalable and
cost effective to meet commercial requirements.

Voyager’s founding management team has deep expertise and a track record of
building exceptional life science companies. In addition to Mr. Levin, who has
more than 30 years of experience building and operating leading biotech
companies, and Dr. Reilly, a highly respected clinical geneticist who leads
Third Rock’s efforts in rare diseases, the team includes: Steven Paul, M.D.,
Interim President of R&D and Venture Partner at Third Rock, who spent 17 years
at Eli Lilly in leadership roles of increasing responsibility, including Vice
President of Discovery Research and Neuroscience Research, and President of
the Lilly Research Laboratories, Dr. Paul also served as Scientific Director
of the National Institute of Mental Health having served as a Branch Chief at
the National Institutes of Health (NIH) for 10 years prior to joining Eli
Lilly; Jim Geraghty, Interim Chief Business Officer and
Entrepreneur-in-Residence at Third Rock, who spent 20 years at Genzyme in
leadership roles across a variety of functions, including as a Senior Vice
President and Officer; Dinah Sah, Ph.D., Interim Senior Vice President of
Neuroscience and former Vice President of Research at Alnylam; Jeff Goater,
Vice President of Business Development and former Managing Director at
Evercore Partners; Rob Kotin, Ph.D., Vice President of Production and former
Senior Investigator at the NIH, where he was the co-inventor of the
baculovirus-based AAV production system; and James McLaughlin, Head of
Operations and former Associate at Third Rock.

A core part of our strategy is to work closely with and learn from patient
advocacy groups, and we have developed strong relationships with leading
patient education and research foundations such as the Friedreich’s Ataxia
Research Alliance (FARA) and The Michael J. Fox Foundation for Parkinson’s
Research, which partly funds Dr. Bankiewicz’s work with AAV gene therapy.

About Voyager Therapeutics

Voyager Therapeutics is a gene therapy company developing life-changing
treatments for fatal and debilitating diseases of the central nervous system
(CNS). Voyager is committed to advancing the field of AAV (adeno-associated
virus) gene therapy through innovation and investment in vector optimization
and engineering, dosing techniques, as well as process development and
production. The company’s initial pipeline is focused on CNS diseases in dire
need of effective new therapies, including Parkinson’s disease, a monogenic
form of amyotrophic lateral sclerosis (ALS), and Friedreich’s ataxia. Founded
by scientific and clinical leaders in the fields of AAV gene therapy,
expressed RNA interference and neuroscience, Voyager Therapeutics was launched
in 2014 with funding from leading life sciences investor Third Rock Ventures
and is headquartered in Cambridge, Mass. For more information, please visit
www.voyagertherapeutics.com.

About Third Rock Ventures

Third Rock Ventures is a leading healthcare venture firm focused on investing
and launching companies that make a difference in people’s lives. The Third
Rock team has a unique vision for ideating and building transformative
healthcare companies. Working closely with our strategic partners and
entrepreneurs, Third Rock has an extensive track record for managing the value
creation path to deliver exceptional performance. For more information, please
visit the firm’s website at www.thirdrockventures.com

Contact:

Pure Communications, Inc.
Katie Wilson, 910-509-3977
 
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