Lots has happened since my last video! Today I talk about the updates from ASH 2018, with a focus on BLUE and GBT. I also talk about FATE, MRNA, and the companies making bispecifics. This is not investment advice, but for information only.
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 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 […]
Today I go through various Q2 earnings reports and see where each company stands relative to its value. I spend most of my time talking about the drop in ONCE and whether or not it is an overreaction. *This is not investment advice*
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 […]
Gene therapy and car-t are leading the way in new potential treatments of blood disorders. I breakdown how these techniques work and which companies are using the technology. Follow me @matthewlepoire *This is not trading advice*
-Novartis buys gene therapy company, Avexis for $8.7 billion http://investors.avexis.com/phoenix.zhtml?c=254285&p=irol-newsArticle&ID=2341482 -Spark is another gene therapy company I like, involved in hemophilia and eye-related blindness diseases http://sparktx.com/ Follow me on twitter @matthewlepoire