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Targeted Genetics Corporation
TARGETED GENETICS NAMED AS SUBCONTRACTOR TO NIAID CONTRACT TO EXPAND
AAV-BASED HIV VACCINE PROGRAM
funding to total approximately $22 Million
Contract provides funding mechanism to develop AAV-based vaccines against HIV
strains most prevalent in North America and Europe
Seattle, WA November 28, 2005 Targeted Genetics Corporation (Nasdaq: TGEN) and its scientific
collaborators at The Children s Hospital of Philadelphia and Columbus Children s Research Institute
(CCRI) today announced that the National Institute of Allergy and Infectious Disease (NIAID) has
awarded a $21.75 million contract to expand AAV-based HIV vaccine development. This contract is
being supported by the HIV Vaccine Design and Development Teams (HVDDT) program from the NIAID
Division of AIDS. Under the terms of this agreement, Targeted Genetics as a subcontractor is
eligible to receive up to $18 million of the total over 5 years to be used to advance AAV-based
vaccines against HIV.
This program underscores the commitment and collaboration within the HIV vaccine community toward
the potential for AAV-based vaccines, said H. Stewart Parker, President and Chief Executive
Officer of Targeted Genetics. Funding provided by this subcontract will enable us to expand our
HIV vaccine program beyond the scope of our important collaboration with the International AIDS
Vaccine Initiative (IAVI), which is focused on developing an AAV-based vaccine for use in
developing nations. We believe that a safe and effective vaccine against HIV is the best way to
slow the growth of the AIDS pandemic. The focus of the program funded by this contract is to
develop an AAV-based HIV vaccine for worldwide use, and may represent a significant future product
Our research to date in animal models of HIV infection suggest that AAV-based vaccines can
stimulate robust B-cell and T-cell responses, reduce viral load, and ultimately improve survival in
vaccinated animals subsequently challenged with a virulent strain of SIV, said Philip R. Johnson
Jr., M.D., Chief Scientific Officer at Children s Hospital of Philadelphia and the
Principal Investigator for the contract. Johnson noted that the HVDDT will test a prime-boost
vaccination protocol using rAAV vectors based on two different serotypes of the AAV capsid. If we
are successful, this strategy can easily be applied to HIV vaccines representing all clades of this
genetically diverse human pathogen, Johnson added.
We are pleased that the NIAID recognizes the potential of this coordinated approach to HIV
vaccine, prevention, and treatment research and are looking forward to continuing to advance the
development of AAV-based vaccines against HIV, said K. Reed Clark, Ph.D., Director, Viral Vector
Core Facility and investigator in the Center for Gene Therapy at Columbus Children s Research
Institute (CCRI) and Associate Professor of Pediatrics at The Ohio State University College of
Medicine and Public Health.
The contract is designed to provide funding to optimize the manufacturing process for AAV-based
vaccines and support preclinical and clinical development of a vaccine against HIV. Clinical
trials of a candidate vaccine will be coordinated through the NIH-sponsored HIV/AIDS Vaccine Trials
Federal funding will be provided for this project by the National Institute of Allergy and
Infectious Diseases, National Institutes of Health, Department of Health and Human Services, under
Contract No. HHSN266200500008C, ADB No. N01-AI-50008. The project will be 100% federally funded in
the amount of $21,752,051.
The Targeted Genetics-IAVI-CCRI-Children s Hospital of Philadelphia collaboration has an ongoing
Phase I HIV Vaccine clinical study in Belgium, Germany, and India. The study is designed to assess
safety and immune responses following immunization with tgAAC09, its AAV-based HIV/AIDS vaccine, in
healthy volunteers who are uninfected with HIV. In early 2005, the collaborative group reported
preliminary Phase I results demonstrating that the vaccine candidate met the safety endpoint and
In November, 2005, the collaborative group announced the start of a Phase II trial in South Africa
to test the safety and immunogenicity of tgAAC09, a preventive HIV vaccine candidate that is based
on HIV subtype C, the subtype of the virus most prevalent in southern and eastern Africa. This is
the first Phase II HIV vaccine trial to be held in South Africa. The trial is being conducted in
three sites in South Africa, anticipated to take about 18 months to complete and designed to enroll
78 volunteers who are in good health. tgAAC09 is designed to elicit two different types of immune
responses, an antibody response and a cell mediated response, which is thought to be critical for
an effective vaccine to prevent AIDS.
Targeted Genetics Corporation
Targeted Genetics Corporation is a biotechnology company committed to the development and
commercialization of innovative targeted molecular therapies for the prevention and treatment of
inflammatory arthritis, HIV/AIDS and other acquired and inherited diseases with significant unmet
medical need. Targeted Genetics uses its considerable knowledge and capabilities in the development
and manufacturing of gene delivery technologies to advance a diverse product development pipeline.
Its product development efforts target inflammatory arthritis, HIV/AIDS, congestive heart failure,
Huntington s disease, and hyperlipidemia. To learn more about Targeted Genetics, visit its website
Contact: Stacie D. Byars, 206-521-7392
About The Children s Hospital of Philadelphia
The Children s Hospital of Philadelphia was founded in 1855 as the nation s first pediatric
hospital. Through its long-standing commitment to providing exceptional patient care, training new
generations of pediatric healthcare professionals, and pioneering major research initiatives, The
Children s Hospital of Philadelphia has fostered many discoveries that have benefited children
worldwide. Its pediatric research program is among the largest in the country, ranking second in
National Institutes of Health funding. In addition, its unique family-centered care and public
service programs have brought the 430-bed hospital recognition as a leading advocate for children
and adolescents. For more information about The Children s Hospital of Philadelphia, visit
Contact: Peggy Flynn, 267-426-6080
Columbus Children s Research Institute
Columbus Children s Hospital ranks among the top 10 in National Institutes of Health research
awards and grants to freestanding children s hospitals in the country and houses the Department of
Pediatrics of The Ohio State University College of Medicine and Public Health. The Columbus
Children s Research Institute (CCRI) conducts more than 400 research projects annually and is the
home of Centers of Emphasis encompassing gene therapy; molecular and human genetics; vaccines and
immunity; childhood cancer; cell and vascular biology; developmental pharmacology and toxicology;
injury research and policy; microbial pathogenesis; cardiovascular medicine; and biobehavioral
health. Pediatric Clinical Trials International (PCTI), a site management organization affiliated
with the hospital, also coordinates more than 50 clinical trials annually. More information about
Children s Hospital of Columbus or CCRI is available by calling (614) 722-KIDS or through the
hospital s web site at www.columbuschildrens.org.
Contact: Pam Barber, 614-722-4595
Harbor Statement under the Private Securities Litigation Reform Act
This release contains forward-looking statements regarding Targeted Genetics anticipated revenues,
regulatory filings, research programs, clinical trials, product development and potential related
to Targeted Genetics AAV based vaccine initiatives. These statements, involve current
expectations, forecasts of future events and other statements that are not historical facts.
Inaccurate assumptions and known and unknown risks and uncertainties can affect the accuracy of
forward-looking statements. Factors that could affect its actual results include, but are not
limited to, Targeted Genetics ability to recruit and enroll suitable trial participants, the
timing, nature and results of research and clinical trials, Targeted Genetics ability to raise