087 – Sangamo Therapeutics Leverages Pharma Partnerships for Success!

Sangamo Therapeutics is clinical-stage biotech company looking to commercialize a number of assets for the treatment of disease. Their furthest along program is a gene therapy treatment for Hemophilia A. The sector has seen some recent upsets with the FDA issuing a CRL to Biomarin for their HemA gene therapy, as well as some safety concerns with Uniqure and Bluebird Bio. Sangamo also has a number Phase 1/2 programs that will have readouts in late 2021 as well as a large preclinical pipeline. They have been able to leverage a number of partnerships with Pharmaceutical companies that has led to significant cash payments and equity purchases.

In this episode, I share an email exchange I had with the SVP, Head of Business Development at Sangamo, Melita Sun Jung.

I also discuss updates from BioXcel and Bluebird Bio.

Check out our sponsor at Gallant.com and use promo code: BIO to save when you enroll your pet for stem cell banking.

Presentation slides: https://drive.google.com/drive/folders/1MENe3kFzFosR6-ALPoY9ovpXXcERH_j3?usp=sharing

Help out the show (or join the discord) by becoming a patron at: https://www.patreon.com/breakingbiotech

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http://www.breakingbiotech.com #breakingbiotech

Disclaimer: All opinions expressed by Matt in this podcast are solely his opinions. You should not treat any opinion expressed by Matt in this podcast as a specific inducement to make a particular investment or follow a particular strategy, but only as an expression of his opinion. Matt’s opinions are based upon information he considers reliable, but Matt cannot warrant its completeness or accuracy, and it should not be relied upon as such. Matt is not under any obligation to update or correct any information provided in this podcast. Past performance is not indicative of future results. Matt does not guarantee any specific outcome or profit. You should be aware of the real risk of loss in following any strategy or investment discussed in this podcast. #biotech

 

086 – Will Selecta Biosciences be the Next Top Platform Biotech? Annovis Shows Positive PD Data

Selecta Biosciences is developing a platform to promote immuno-tolerance. Many treatment regiments have unwanted immune responses that can reduce the therapy’s efficacy or cause immunity against the vector in a gene therapy. They are also seeking indications in autoimmune diseases. If successful, SELB’s platform would see massive adoption. They recently had negative data in a Phase 2 trial of Gout, but because upcoming catalysts are for other programs, I see an opportunity.

I also discuss new data from Eli Lilly, Annovis and Marker Therapeutics.

Check out our sponsor at Gallant.com and use promo code: BIO to save when you enroll your pet for stem cell banking.

If you want to help out the show (or join the discord), become a patron at: https://www.patreon.com/breakingbiotech

Follow me on twitter @matthewlepoire

Send me an email matthewlepoire@gmail.com

http://www.breakingbiotech.com #breakingbiotech

Disclaimer: All opinions expressed by Matt in this podcast are solely his opinions. You should not treat any opinion expressed by Matt in this podcast as a specific inducement to make a particular investment or follow a particular strategy, but only as an expression of his opinion. Matt’s opinions are based upon information he considers reliable, but Matt cannot warrant its completeness or accuracy, and it should not be relied upon as such. Matt is not under any obligation to update or correct any information provided in this podcast. Past performance is not indicative of future results. Matt does not guarantee any specific outcome or profit. You should be aware of the real risk of loss in following any strategy or investment discussed in this podcast. #biotech

 

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.

http://www.breakingbiotech.com
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?

Follow me @matthewlepoire
http://www.breakingbiotech.com

*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
http://www.breakingbiotech.com

Relevant links:
https://www.sarepta.com/our-pipeline
https://www.sec.gov/Archives/edgar/data/873303/000119312518292068/d616670dex992.htm

 

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:
https://www.sangamo.com/
https://seekingalpha.com/article/4205472-depth-analysis-sbminus-913-early-clinical-readout-mps-ii
https://seekingalpha.com/article/4205037-sangamo-work
https://doi.org/10.1016/j.ymthe.2018.03.002

https://www.ncbi.nlm.nih.gov/pubmed/23837440

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.

Follow me @matthewlepoire

 

*This is not trading advice*

 

Novartis buys Avexis!

-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

 
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