-Sangamo Therapeutics recently announced their Phase 1/2 data for the treatment of MPS II/Hunter Syndrome
-The market reacted negatively to the news because of the uncertainty associated with unclear IDS activity and unclear metrics for successful approval by the FDA
I misspoke a couple times:
-Around 2:00 I say the breakdown products can be detected in the blood/urine. The uGAG measurements are not the breakdown products but the actual targets of IDS, which are what is measured in the blood/urine.
-Around 3:30 I talk about the expression of the transgene. The AAV will circulate and infect a variety of cells. The gene editing will take place after albumin promoter of the genome, which is only active in hepatocytes. Hepatocytes will be the only cell type to express the transgene, and the product will circulate in the blood to other tissues.
*This is not investment advice*
Follow me @matthewlepoire
See the blogpost at http://www.breakingbiotech.com