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Re: poorgradstudent post# 189775

Friday, 04/10/2015 6:07:15 AM

Friday, April 10, 2015 6:07:15 AM

Post# of 257273
CLDN: thanks PGS for the replay. Hearing at Roger Hajjar, He seems confident with the use of AAV vector as it has been chosen because of his long therm expression and safety profile. Also being a small vector, after intracoronary infusion, this vector is able to infect the myocardium. The biopsy made to some of the patients in CUPID1, also supports this as most of the patients that went trough biopsy and had the larger dose, has the gene transfected, compared to NONE in the placebo group. I believe this is an evidence that the vector is doing his job. Also in the clinical trial, before enrolling patients, they undergo a test to see if they have neutralizing Abs and in that case they are excluded from the trial. Also int herms of safety, no T cell response has been seen to date either in Cupid 1 or 2. I believe the company knows what they are doing in therms of type of vectors and transcriptional controls.

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