Celladon Heart-Failure Study Looms Large as Next Big Test for Gene Therapy

NEW YORK (TheStreet) -- The next investor referendum on the resurgent gene-therapy field will arrive next month whenCelladon (CLDN)is expected to announce results from a mid-stage study of a gene therapy aimed atimproving the heart's pumping ability in patients suffering fromthe organ's advanced failure.

Gene therapy uses engineered viruses to replace defective, disease-causing genes. Celladon's lead therapy, Mydicar, is a virus engineered to insert a working gene capable of producing a protein called SERCA2a into heart-failure patients. SERCA2a is responsible for helping heart muscles contract and pump blood more efficiently. Heart-failure patients have low levels of SERCA2a and hearts that do a poor job pumping blood around the body. Celladon believes infusing Mydicar should lead to higher SERCA2a levels and improved heart function.

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Celladon went public in January 2014 at $8 per share. The stock was tradingat $24.10, down 1.8%, on Wednesday morning, after rising by more than 30% in March as investors anticipate the Mydicar study results.

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Celladon Heart-Failure Study Looms Large as Next Big Test for Gene Therapy