Sarepta Declines on Good Micro-Dystrophin Data

Sarepta took a hit from their latest Micro-Dystrophin gene therapy trial update despite showing robust expression in all 4 patients, confirmed by protein analyses and behavioral improvements. In this video, I go through the details of muscular dystrophy, the current treatment options and how this new therapy could significantly improve patients outcomes. I have not […]

Sangamo Therapeutics drops after SB-913 update

-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 […]

I was wrong about Zogenix! FDA updates guidelines for gene therapy.

Note: I meant to say that sub-1% factor activity is SEVERE hemophilia. Normal is from 50% to 150%. Today I talk again about Zogenix because I was wrong about Study 2 being priced in! Congrats to the ZGNX holders. I also talk about the new FDA guidelines on gene therapy for hemophilia and its implications […]