Celladon Announces Successful Preclinical Trials in Congestive
Heart Failure
- Celladon's Collaborators Present Large Animal Trial Results at the American
Society of Gene Therapy Conference in St. Louis, MO -
LA JOLLA, Calif., June 2 /PRNewswire/ -- Celladon Corporation announced today
that its collaborators presented successful results of heart failure trials
conducted in large animal models. Drs. Roger J. Hajjar, M.D. and David Kaye,
MD, Ph.D. presented data on treatment of advanced heart failure in preclinical
models using Celladon's therapeutic agents targeting the Sarcoplasmic Reticulum
ATPase2a (SERCA2a) pump. The SERCA2a pump is a central control point for
progression of heart failure, and treatments targeting this pathway essentially
reversed the deterioration in cardiac function in experimental models.
Furthermore, the therapeutic agents were delivered using delivery methodologies
easily translated into clinical procedures for human heart failure, which are
similar to how coronary angioplasty is performed.
Dr. Kaye presented data from an ovine model of pacing induced heart failure. The pacing model induces moderate to severe heart failure, with animals that
have left ventricular ejection fractions of around 25%, which is typical in
humans of the NYHA Class III group of heart failure patients. Animals treated
with the SERCA2a gene restored left ventricular ejection fractions to normal
levels. Other hemodynamic parameters mirrored the effects on ejection
fraction. The agents were delivered using a novel device developed by
V-Kardia, a cardiovascular device company of The Baker Heart Research
Institute, the largest and leading cardiovascular research institute in
Australia.
Dr. Hajjar presented data from a porcine model of mitral regurgitation induced
heart failure. The porcine model generates a progressive heart failure model
similar to Class III patients. Animals treated with the SERCA2a gene induced
not only short term improvement but by using adeno-associated vectors (AAV)
targeted to the heart, Dr Hajjar also showed that there was long-term
improvement in ventricular function indices following gene transfer of SERCA2a.
"Celladon's therapeutic candidate targets the SERCA2a pathway, an important
regulator of myocardial contractility. Malfunction in this pathway is
associated with progressive heart failure," said Krisztina Zsebo, Ph.D.,
President of Celladon Corporation. "These large animal studies by Celladon's
collaborators, indicate that AAV agents targeting this pathway can reverse the
progression of congestive heart failure. 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. These
results will pave the way for the first clinical trial of gene therapy for
heart failure using AAV vectors." Celladon plans to initiate human trials with
this agent in 2006.
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.
About Celladon 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. In December 2004, Celladon Corporation entered into a collaboration agreement
with Targeted Genetics Corporation (NASDAQ:TGEN) to develop adeno-associated
virus (AAV)-based gene therapies for the treatment of congestive heart failure. Founding investors include Enterprise Partners Venture Capital and Venrock
Associates. http://www.celladon.net/. DATASOURCE: Celladon Corporation CONTACT: Moya Gollaher of Enterprise Partners Venture Capital, +1-858-731-0244, for Celladon Corporation Web site: http://www.celladon.net/
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