Full notification fileGeneral informationNotification NumberB/GB/13/R46/01Member State to which the notification was sentUnited KingdomDate of acknowledgement from the Member State Competent Authority17/01/2013Title of the ProjectA Phase 2b, Double-Blind, Placebo-Controlled, Multinational, Multicenter, Randomized Study Evaluating the Safety and Efficacy of Intracoronary Administration of MYDICAR® (AAV1/SERCA2a) in Subjects with Heart Failure and Investigation of the safety and feasibility of AAV1/SERCA2a gene transfer in patients with chronic heart failure and a left ventricular assist deviceProposed period of release:01/01/2013 to 31/12/2015Name of the Institute(s) or Company(ies)Celladon Corporation, ;
3. Is the same GMO release planned elsewhere in the Community?Yes: Belgium; Germany; Denmark; United Kingdom; Netherlands; Sweden; Poland; Has the same GMO been notified elsewhere by the same notifier?YesIf yes, notification number(s): B/BE/12/BVW2; B/SE/12/EU-2012-001700-37; GMO characterizationGMO is a:DNA VirusIdentity of the GMO:Genus: Dependovirus
Species: Adeno-associated virus (vector). The AAV1/SERCA2a vector is a recombinant adeno-associated virus (rAAV) vector. It is a pseudotype of AAV serotype 1(AAV1) and is denoted as rAAV1/2. As such, the capsid proteins are from wt AAV1 and the AAV DNA (two 145 base inverted terminal repeats) is from wtAAV2.Information relating to the recipient or parental organisms from wich the GMO is derived
Final reportEuropean Commission administrative informationConsent given by the Member State Competent Authority:Yes14/04/2013 00:00:00Remarks:Consent issued for a gene therapy trial to assist calcium cycling in the hearts of heart faialure patients. The trial will take place at 3 hospitals in England. The study objectives are to determine the safety and efficacy of a single intracoronary infusion of MYDICAR® added to an optimal heart failure regimen in patients. AAV1/SERCA2a (MYDICAR®) is an experimental gene transfer agent comprised of a vector and a human gene. The vector is derived from adeno-associated virus serotype 1. The human gene is ATP2A2 which codes for the SERCA2a protein a calcium ion pump that is essential for heart muscle cells to complete the contraction and relaxation cycle of a heartbeat.
|AAV1||Dependovirus||Adeno-associated Virus||-||Serotype 1||-|