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Newsletter April 2014

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News: FDA Grants Breakthrough Status to Gene Therapy for First Time
Celladon today became the first company to receive breakthrough status for a gene therapy treatment in development. The treatment, MYDICAR, is intended to reduce the risk of heart failure in patients with a deficiency of the enzyme SERCA2a. The FDA made its decision to grant breakthrough status to MYDICAR on the basis of Celladon's Phase 1 trial of 39 systolic dysfunction patients. The study reported that subjects receiving high doses of MYDICAR experienced fewer heart failure events than subjects given a placebo, by a factor of more than 80%, and that this reduction was sustained for three years after treatment. No safety issues were reported in this initial trial. Breakthrough status, which is reserved for treatments targeting life-threatening diseases that show significant improvements over the standard of care, allows closer communication with the FDA and potentially an accelerated approval process.

News: Stem cell gene therapy: Chances and risks
Physicians and scientists from Munich and Heidelberg have now proven the long-term effectiveness of stem cell gene therapy, based on a study of patients from the first clinical trial worldwide using gene therapy to treat Wiskott-Aldrich syndrome. Yet several years after the therapy, the researchers also observed an increased incidence of acute leukemia among the patients.

- 23 - 26 April 2014, International Society for Cellular Therapy (ISCT) 20th Annual Meeting, Paris, France
- 21 – 24 May 2014, American Society of Gene and Cell Therapy (ASGCT) 17th Annual Meeting, Washington, DC
- 2 – 3 June 2014, 3. Laupheimer Zelltage - Processes for Virus-based Biologics, Laupheim, Germany
- 6 – 8 August 2014, The 20th Annual Meeting of the Japan Society of Gene Therapy (JSGT), Tokio, Japan
- 25 – 27 September 2014, 14th Annual Meeting of the International Society for Cell & Gene Therapy of Cancer, Amsterdam, The Netherlands
- 23 – 26 October 2014, XXII Congress of the European Society of Gene and Cell Therapy (ESGCT), The Hague, The Netherlands
- 27 – 29 October 2014, 3rd International Conference and Exhibition on Cell & Gene Therapy, Las Vegas, NV
- November 2014, Formulation and Drug Delivery Congress, London, UK
- November 2014, Cell Therapy Congress, London, UK


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