$SGMO status going into C22
Two synthetic gene transfer programs:
- HemA now run by $PFE P3. Hold for protocol amendment. Expect timing update for full analysis in next couple months.
- #Fabry owned program good data potential first/best in class
No further programs.
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$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/
Cell therapy pipeline:
-Sickle cell collab with $SNY lead indication (see next)
-$GILD collab programs on hold. No update for a while but likely tied to durability issues seen at $ALLO $CRSP $DTIL
-Steadfast study is #POC for CAR Tregs with significant pipeline to follow 3/
$SNY concluded that their preliminary POC efficacy and safety results confirm the potential tx value. Appears headed for registrational study in next 12-18 months. $SGMO 4/
Reminder to look at previous tweet figure 6 showing number of #SCD vaso-occlusive crises (VOC) dropped from 44 to 1. 5/
ZFP-TF initial in vivo. Partners progressing but appear delivery hurdle remains
$TAK -686 clinical trial being planned for huntington’s
ALS subject of breakthrough article on $PFE website
$NVS 2 VPs joined Sangamo
$BIIB working on 4 selected programs. Waiting for updates 6/
Gene regulation pipeline:
- Owned #Prion and one undisclosed preclinic.
- $BIIB has selected four programs (2 preclinic) w/ 8 optional targets which $SGMO likely working on
- $NVS has three active programs
- $PFE ALS ZFP-TF selected and in animal testing
- $TAK see prior 7/
Key for all bios in this kind of market is cash runway IMO. $SGMO relies on collaboration partners to fund opex thru reimbursed research and #milestones. Impact of HemA hold on forecast is key. No change yet to prior fcst: will update once we get more clarity. 8/
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
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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/
ERT is the SOC for patients with #Fabry. The LT follow up studies for patients on ERT show that it does not stop disease progression and imposes a high treatment burden. 3/
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.
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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/
OrcaBio announced a raise of $192m in 2020
GentiBio received $157m in Aug 2021
Quell raised $156m in Nov 2021 after they received CTA approval for a liver transplant treatment similar to $SGMO 3/
The chase for an #HIV cure has been elusive for decades with as many failures as seen with CNS diseases. $SGMO has been dedicated to this effort tho are now supporting sponsors given the cost of the effort. This stream will provide an recap. #Autologous#CellTherapy
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$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/
The first trial (sponsored by UPenn) SB-728 began in 2009 focused on HAART treated patients and was focused on safety and comparison/persistence of CD4/CD8 T cells after ZFN modifed autologous T cells were infused. #HIV#CellTherapy 3/
$SNY looks like they are ready for a Genomic Medicine Unity #GMU coming out year in C21. Many hires. Many open reqs. Building on Ablynx, Bioverativ and Kiadis M&A #GeneTherapy
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$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)
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$SNY #GMU Principal Scientist Robert Jackson profile says he is working on: Huntingtin's, MLD, PKU, Neurophathies, T1Diabetes Hypertension, Nephrophaty, vascular disease, Rheumatoid Arthritis and sensorineural hearing loss
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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.
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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…
#GenomicMedicine is disrupting Big Pharma who has begun to respond by spinning off old product lines and jumping into #GeneTherapy. 3/