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Celladon Provides MYDICAR R Programme Renovate Of First-In-Human Evaluation For Contemporary Heart Failure At American Kingdom Of Gene Therapy Annual Conclave

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Celladon Corporation presented today Chapter 1 data from the Calcium Up-Regulation by Percutaneous Governance of Gene Therapy in Cardiac Disease (CUPID), a First-in-Human Folio 1/2 Clinical Trial, in a scientific symposium at the 12th Annual American Society of Gene Therapy Meeting.
The Period 1 data showed that MYDICAR(R) had an acceptable safety profile in twelve patients, as determined by study investigators and an independent safety committee. In addition, improvements from baseline to six months were observed across a character of decisive efficacy parameters essential in assessing feelings failure status. Efficacy was defined as the mean improvement in at least 2 of 5 domains without any worsening in the remaining domains, including a functional six-minute pace test, o2 consumption, excellence of lifetime questionnaire, biomarker exercise and left ventricular magnitude and function.
"We are encouraged by the essential improvements in cardiac function and overall case of patients, findings that we have exhibit the go back toward habitual intracellular calcium cycling and contractility in some of the heart muscle cells of these very ill patients," said Krisztina M. Zsebo, Ph.D., president and manager executive officer. "Our lingering preclinical and clinical controversy to understand the molecular basis of myocardial dysfunction, together with the evolution of safe and efficient gene transfer technology, has placed gene-based therapy for heart failure within distance and yielded valuable insights for the abundant earth of study."
The Sheet 1 open-label, sequential potion escalation, multi-center leaf of the test was designed to investigate safety and biological part of restoring SERCA2a enzyme continuance in affection muscle cells. The enzyme levels are decreased in late stages of emotions failure, and lenghty research shows loss of SERCA2a levels represents a usual pathway resulting in a defect in the adeptness of the heart to business agreement properly. Replacing the enzyme may restore supply and reverse passion failure.
The Phase 2 randomized, double-blind, placebo-controlled, parallel-group, dose ranging piece of the announce is designed to evaluate the use of MYDICAR at two or three dose levels compared to placebo in 37 patients. CUPID is currently enrolling patients with advanced love failure at 17 U.S. medical centers.
Zsebo adds, "We anticipate completion of Stage 2 enrolment late this summer and way to announcement results in the beginning half of 2010. In addition, we obtain adequate MYDICAR product manufactured to the works Page 3 and recently acquired exclusive licence to utilize Adeno-Associated Viral (AAV) vector technology in heart failure, which bodes flourishing for commercial product course of MYDICAR and leading to viable strategic partners. "
Celladon scientists, led by company co-founder Roger J. Hajjar, M.D., Director of the Cardiovascular Test Center at Mount Sinai School of Medicine, New York, developed MYDICAR for restoring the SERCA2a calcium transporter in heart failure and validated the overall beneficial factor on cardiac function. MYDICAR is a recombinant adeno-associated viral (rAAV) vector that transfers the SERCA2a gene into heart muscle cells. MYDICAR is delivered in a unmarried dose directly to the heart muscle during a short outpatient procedure, performed in a standard cardiac catheterization laboratory via a small section in the upper leg.
Of the twelve patients treated, two with low levels of pre-existing antibodies to the AAV vector did not pageantry improvement in these parameters. The information are consistent with safety established for other rAAV vectors, which has been demonstrated in clinical studies of bounteous than 500 patients. AAV vectors are the product of decades of check focused on the safety of gene transfer agents, and are derived from components of a non-replicating, non-pathogenic, commonly occurring human virus. AAV vectors do not integrate into the chromosome and are considered non-mutagenic. In addition, they gain not been associated with the types of inflammatory reactions observed in trials involving adenoviral vectors, which are known to induce acute inflammation of tissues due to activation of the body's immune system.
Source: Celladon Gathering
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