Marty Chilberg Profile picture
Apr 12, 2021 13 tweets 10 min read Read on X
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
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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
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$SGMO ran SB-728-0902 to find out whether #ZFN modified CD4+ T cells are safe and how these cells affect #HIV. #Autologous #CellTherapy
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$SGMO ran a second trial SB-728-1002 beginning in 2010 which continued the effort of determining the effect of escalating #ZFN modified T cells post HAART interruption.
#HIV #Autologous #CellTherapy
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$SGMO continued to invest in this effort with the next trial SB-728-1101 in C11 which upped the dosage to 5-to-30b #ZFN modified T cells. This trial was to determine the impact of escalating doses of cyclophosphamide as a pretreatment regimen.
#HIV #Autologous #Celltherapy
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SB-728-mR-1401 was initiated by $SGMO in C14 to better understand treatment impact of 2 vs 3 doses of #ZFN modified T cells increasing the dosage up to 40b cells supported by safety data from earlier trials.
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It is pretty remarkable how much $$$ $SGMO invested in this effort even tho they knew how many years the process would take given the step function of the trials to date. In 2015 UPenn with funding from NIAID sponsored the first trial focused on SB-728mR. Note conclusion
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The City of Hope with Paula Cannon and CIRM funding sponsored SB-728mR-HSPC. This trial had an estimated primary completion of last month. It includes the impact of busulfan levels with the #ZFN edited T cells. Safety and dosing have led to treatment w/preconditioning.
#HIV
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Case (Sekaly, Rodriguez) began their next trial in Jun 2019 focusing on replication-competent HIV reservoir. This trial is still recruiting. 2 yrs of data are required before primary completion date of Jan 2024. #ZFN $SGMO
#HIV #Trailblazer #Reservoir
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Pablo Tegas is the investigator in the next UPenn sponsored trial that was initiated in Jul 2019. This trial compares treatment interruption after $SGMO #ZFN infusion. The primary completion data is at the end of this year.
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$SGMO is also running a LT followup study for patients treated with SB-728-T or SB-728mR cells. I found the Oct 2020 Cowen report interesting as they have concluded that the cum data provides POC for the #CellTherapy platform technology.
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More from @mchilberg

Sep 14, 2022
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/
Read 14 tweets
Mar 29, 2022
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
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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
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Read 25 tweets
Dec 19, 2021
$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.
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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
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Read 9 tweets
Mar 16, 2021
$BEAM 4Q20 results out. Valuation is crazy but they are the best of #Crispr breed in my opinion given execution to date.
#GeneTherapy
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$BEAM focus on platform, delivery and manufacturing is enabled by share price and ability to source funds without huge dilution
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$BEAM focus on based editing of transition mutations.
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#dreaminase ImageImageImage
Read 6 tweets
Mar 15, 2021
$SNY looks like they are ready for a Genomic Medicine Unity #GMU coming out year in C21. Many hires. Many open reqs. Building on Ablynx, Bioverativ and Kiadis M&A
#GeneTherapy
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$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)

2/
$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
3/
Read 14 tweets
Feb 25, 2021
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.
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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.
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Read 24 tweets

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