Full Press Release Details
Targeted Genetics Corporation
TARGETED GENETICS AND CELLADON ANNOUNCE A COLLABORATION TO
DEVELOP AAV-BASED GENE THERAPIES FOR
CONGESTIVE HEART FAILURE
-Deal terms include $6 million equity investment in Targeted Genetics by Celladon s lead
Seattle, WA and La Jolla, CA January 4, 2005 Targeted Genetics Corporation (Nasdaq: TGEN) and
Celladon Corporation today announced a collaboration agreement to develop adeno associated virus
(AAV)-based gene therapies for the treatment of congestive heart failure. Simultaneous with the
initiation of this collaboration, Enterprise Partners and Venrock Associates, venture capital funds
that have invested in Celladon, made a $6 million common stock investment in Targeted Genetics.
The collaboration combines Targeted Genetics expertise in the development, manufacture and
clinical evaluation of AAV-based therapies with Celladon s portfolio of genes with potential in the
treatment of congestive heart failure. Under the terms of the agreement, Targeted Genetics and
Celladon will work together to develop AAV vectors for the treatment of heart failure.
Celladon was founded with the goal of becoming the leader in developing molecular therapies for
congestive heart failure, said Drew Senyei, M.D., Chairman at Celladon and Managing Director at
Enterprise Partners Venture Capital. An extensive evaluation of available gene delivery
technologies has led us to believe that AAV vectors have the greatest potential to address the
medical and commercial needs of a chronic disease such as congestive heart failure. We believe
that Targeted Genetics is the partner of choice for developing AAV-based products due to their
exceptional expertise in the development, manufacture and clinical evaluation of gene based
Our collaboration with Celladon broadens our leadership position and development of AAV-based
therapies and presents an exciting opportunity to expand our capabilities into the area of
congestive heart failure, said H. Stewart Parker, President and Chief Executive Officer of
Targeted Genetics. The ability of AAV vectors to deliver genes to cardiac muscle cells combined
with the long-term gene expression properties of these vectors makes them particularly exciting in
the treatment of chronic cardiac diseases such as congestive heart failure. Five million people in
the United States today are living with congestive heart failure, and our efforts to innovate new
therapies in this area are consistent with our mission to address diseases with significant unmet
Celladon s therapeutic candidates target the SERCA2a pathway, an important regulator of myocardial
contractility. Malfunction in this pathway can lead to progressive heart failure, said Krisztina
Zsebo, President of Celladon Corporation. Pre-clinical studies by Celladon s co-founders, Dr.
Kenneth R. Chien M.D., Ph.D. and Dr. Roger J. Hajjar, M.D., prominent researchers in the field of
cardiovascular medicine, indicate that AAV agents targeting this pathway can reverse the
progression of congestive heart failure in animal models. Gene delivery may provide a new approach
to unlocking the therapeutic potential of this pathway, whereas previous traditional small molecule
attempts have not been successful.
Under the terms of the collaboration agreement, Targeted Genetics agreed to commit $2 million
towards the development, manufacture and preclinical development of AAV vectors containing the
SERCA2a gene and phospholamban gene mutations. Celladon agreed to cover all additional
development, manufacture and preclinical development costs. Targeted Genetics will receive
milestone payments upon the completion of pre-specified product development milestones, and also
will receive significant royalties on any sales of potential products commercialized under the
collaboration. Simultaneously with the signing of the agreement with Celladon, Celladon investors
Enterprise Partners and Venrock Associates purchased $6 million of Targeted Genetics stock in a
directed public offering at $1.52 per share.
We are pleased that these investors have decided to increase their investment in our company
beyond the amount of funding committed to the Celladon collaboration. In addition to funding
development activities under the collaboration, this investment will also help to support our other
therapeutic product development programs, said Todd E. Simpson, Chief Financial Officer of
ABOUT CONGESTIVE HEART FAILURE
Congestive heart failure (CHF) is a serious condition in which the heart loses its ability to pump
blood efficiently. According to the National Heart, Lung and Blood Institute, about 5 million
people in the United States alone have heart failure, and another 550,000 new cases are diagnosed
each year. CHF contributes to or causes about 300,000 deaths annually. The disease is most common
in people aged 65 or older, women and African Americans. The most common symptoms of heart failure
are shortness of breath, feeling tired, and swelling in the ankles, feet, legs, and sometimes the
abdomen. There is no cure for CHF.
ABOUT TARGETED GENETICS
Targeted Genetics Corporation develops gene-based products for preventing and treating acquired and
inherited diseases. The Company has three clinical product development programs, targeting cystic
fibrosis, AIDS prophylaxis and rheumatoid arthritis. The Company also has a promising pipeline of
product candidates focused on hemophilia and cancer, and a broad platform of gene delivery
technologies for application in nucleic acid-based drug development. For more information about
Targeted Genetics, visit its website at
Celladon, Inc., a privately held company based in La Jolla, California, was founded by Dr. Kenneth
R. Chien M.D., Ph.D. and Dr. Roger J. Hajjar, M.D. The company is focused on developing novel
molecular therapies for congestive heart failure. The company s first product targets the
treatment of congestive heart failure by enhancing calcium cycling in the heart through a gene
based therapy. Founding investors include Enterprise Partners Venture Capital and Venrock
Associates. For more information about Celladon, visit its website at www.celladon.net.
Safe Harbor Statement under the Private Securities Litigation Reform Act of 1995:
Targeted Genetics Corporation has included in this press release certain forward-looking
statements within the meaning of the Private Securities Litigation Reform Act of 1995 concerning
Targeted Genetics business , collaboration with Celladon, projected financial resources,
intellectual property, clinical trials and regulatory filings and anticipated data from Targeted
Genetics clinical and preclinical programs . The words or phrases can be, expects,
may affect, anticipates, may depend, believes, estimates, plans, projects and similar
words and phrases are intended to identify such forward-looking statements. These
forward-looking 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. Actual results could
differ materially from expectations for a number of reasons, including failure of our partners to
provide funding, our failure or our partner s failure to make progress with our clinical trials,
our failure or our partner s failure to obtain positive results from our preclinical programs, our
failure or our partner s failure to obtain or maintain regulatory approvals, our failure or our
partner s failure to maintain or protect our intellectual property and the other risks described in
the section entitled Factors Affecting Our Operating Results, Our Business and Our Stock Price in
our Quarterly Report on Form 10-Q for the quarter ended September 30, 2004 and Risk Factors in
our Prospectus Supplement, dated December 31, 2004 and Base Prospectuses filed with the Securities
and Exchange Commission on January 4, 2005. You should not rely unduly on these forward-looking
statements, which apply only as of the date of this release. We undertake no duty to publicly
announce or report revisions to these statements as new information becomes available that may
change our expectations.