052 – BATTLING for the Top Anti-VEGF Eye Therapy

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

Image credit: https://ir.clearsidebio.com/static-files/c9c4b682-92aa-4f3d-84df-6343468e340c

*This is not investment advice, so please do your own due diligence before investing*


043 – Sarepta keeps the gene therapy throne in DMD! ContraVir submits NASH IND!

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.

Follow me on twitter @matthewlepoire


041 – Is REGENXBIO a buy after Zolgensma approval?

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?

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*this is not investment advice*


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 taken a position and this is not investment advice.
Follow me @matthewlepoire

Relevant links:


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 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*

Relevant links:


Follow me @matthewlepoire
See the blogpost at http://www.breakingbiotech.com


Gene therapy/CAR-T breakdown. Bluebird falls on GBT news

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.

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*This is not trading advice*


Novartis buys Avexis!

-Novartis buys gene therapy company, Avexis for $8.7 billion

-Spark is another gene therapy company I like, involved in hemophilia and eye-related blindness diseases

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