SM presenting yesterday at HC Wainwright made a couple references to #Fabry study which prompted this thead
- No steroids have been used.
- Best data he's had the privilege to report
investor.sangamo.com/events/event-d…
$SGMO
1/ Fabry disease = abnormal a-Gal enzyme activity leading to Gb-3 and lyso-GB3 accumulation. Prevalence data varies widely given multiple attempts to quantify the impact of under-diagnosis. Kidney and cardiac disease are LT impacts
2/

In recognition of the news that $SGMO has dosed the first human with engineered Tregs for renal transplant patients, here is a thread for anyone interested.
1/ Before we look at some previous $SGMO disclosures it's useful to note that this has become a very active investment sector in the past 2 years. The largest splash was made when $MRK acquired Pandion in Feb 2021 for $1.85b or 3x the market cap. Pandion=Talon effector cells
2/

$SGMO status going into C22
Two synthetic gene transfer programs:
- HemA now run by $PFE P3. Hold for protocol amendment. Expect timing update for full analysis in next couple months.
- #Fabry owned program good data potential first/best in class
No further programs.
1/ $SGMO development focus leveraging ZF technology shows evolution from old four platforms to new integrated tech message. ZF tech facilitates both ex vivo and in vivo genomic medicines. Examples are CAR Tregs cell therapy and ZFP-TF CNS regulation.
2/

The chase for an #HIV cure has been elusive for decades with as many failures as seen with CNS diseases. $SGMO has been dedicated to this effort tho are now supporting sponsors given the cost of the effort. This stream will provide an recap. #Autologous #CellTherapy
1/ $SGMO began this clinical effort in 2009. They have worked with UPenn, City of Hope, Case Western University. NAID and CIRM have helped with funding among others. We should see some critical updates this year.
#HIV
2/

$BEAM 4Q20 results out. Valuation is crazy but they are the best of #Crispr breed in my opinion given execution to date.
#GeneTherapy
1/ $BEAM focus on platform, delivery and manufacturing is enabled by share price and ability to source funds without huge dilution
2/

$SNY looks like they are ready for a Genomic Medicine Unity #GMU coming out year in C21. Many hires. Many open reqs. Building on Ablynx, Bioverativ and Kiadis M&A
#GeneTherapy
1/ $SNY #GMU head of Rare and Neurologic Disease Research is Christian Mueller. Video into is pretty good
Addl heads include Bruno Figueroa (CMC) and Cate O'Riordan (translational unit)

2/

Part II of the $CRSP $SGMO comparison will hopefully be a bit less dry now that Part I updates are in place. This thread will focus more on valuation, outlook and why the valuation cycle should matter to investors.
1/ Let's start with the concept of #Disruptive technologies. The originator of disruptive innovation theory, Clayton Christensen worked with HBR in 2015 to revisit the past 20 years.
2/
hbr.org/2015/12/what-i…

This is the third edition of my tweetstorms on #GeneTherapy #CellTherapy #GeneEditing. This one compares $SGMO to $CRSP and attempts to look at #GenomeMedicine from the perspectives laid out by C.Woods at ARK This is part one of the series. Pt 2 tomorrow AM 1/20 The charts of both are attached and useful context. $CRSP went public in 2016. The weekly chart is attached and notes the pct ownership of $ARKG $ARKK since CY18. Prior the stock was stagnant below the support/resistance levels show. 2/20

Appears Cowen published a useful courtesy $SGMO rept for investors prior to their annual HC conference. Most of the verbiage is an accumulation of prior reports. They have higher model royalties for $PFE and $SNY royalties than mine. Not a SOTP or full blown model Cowen states that though the prelim data on $SNY collab was unimpressive, they are cautiously optimistic. My fcst was pushed out several years consistent with $BLUE issues and probability that more issues will crop up for $CRSP and $SGMO programs.

$ARKG Tweet series
This series of tweets is my opinion only. Cathie Wood has created a group of ETFs that target the disruptive innovation that is both happening and accelerating. These funds have had stellar performance based upon innovation trends and economic theory.
1/ The $ARKG fund description is a good place to start. This has changed recently to a #CRISPR theme but started more with the sequencing cost declines which would drive diagnostics adoption and precision medicine
2/

What's up with $SGMO valuation? This thread will try to show some history and the setup for what I still hope to be an outsized move. First worth noting that news will always trump technicals. First here is a month chart I've been using for the company 1/ This being a 10 year chart, it doesn't show how long $SGMO has been around but it does show virtually no trading volume and a 3 yr ceiling on the price as they were in early development stage while trying to get ill-chosen indications into pre/clinic 2/