UPDATE 11/29/2019: Check out this image from Clearside’s investor presentation about the different ‘back of eye’ delivery methods
Happy Thanksgiving! In this video, I talk about all things related to eye diseases caused by over-proliferating endothelial cells. These diseases are currently treated by monthly intravitreal injections but there are new therapies in clinical trials that are likely to unseat them. I touch on #RGNX, #ADVM and #KOD as well as some others that have made headlines in the last couple weeks.
Relevant publication: Ding et al. AAV8-vectored Suprachoroidal Gene Transfer Produces Widespread Ocular Transgene Expression. JCI. 2019
Hey everybody, hope you’re all doing well! In this video, I talk about Pfizer’s DMD gene therapy update that indicates continued Sarepta hegemony in the space. I also talk about ContraVir and their NASH drug, CRV431.
I must have drank too much coffee before recording because I made more than a couple errors this time around. So, if you’re an audio-only listener and hear something that doesn’t sound right, double check the video version, which corrects the mistakes via captions or has it written properly even though I say it wrong (i.e. I say 3E16 when 3E13 is clearly written on the slide).
Thanks to everyone for listening and do your own due diligence if you are considering investing, this is not a substitute for investment advice.
Today, I talk about REGENXBIO and their in-house gene therapy candidates. Their most important asset in early clinical trials is RGX-314, indicated for wet AMD (and hopefuily Diabetic Retinopathy). This gene therapy could replace blockbuster drugs that treat millions patients, but is it a buy today?
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.
-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.
-What does $SGMO need to accomplish for investigators on the trial to feel comfortable switching off ERT? Is there a GAG reduction sufficient for this? If so, is urine GAG really the right assay to determine whether a patient is benefiting?