Marty Chilberg Profile picture
Dec 26, 2020 23 tweets 15 min read Read on X
$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.
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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
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The economic driver of the Genomic Revolution is a multiplier. Not only sequencing cost declines but the impact of moving from #ZFN to #TALENs to #CRISPR is key. As the editing cost has dropped CRISPR is seeing a dominant increase in publications.
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Table shows the economic cost advantage follows. #ZFN in 2003 to #TALENs to the current cost of #CRISPR has declined in a similar fashion to what has been seen with genomic sequencing declines. The ease of CRISPR use has led to exponential increases in volume driving cost
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Pharma has been re-engineering their companies to return to a biopharma model primarily driven by this trend. I've written a couple articles on this lately. One example looked at $NVS. They see the opportunity and risk as seen in this chart
seekingalpha.com/article/439364…
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The early focus in the clinic has been #oncology. #Kymriah #Yescarta are a couple examples of #CRISPR ex vivo autologous #Celltherapy approvals and the cost-effectiveness of the approach.
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The combination of clinical initiations and covid-19 disruption has led to what Peter Atwater in Moods and Markets said: "as always, nothing exceeds like success". Money is flowing in which is creating a multiplier effect.
Sep $ARKG up 90% YTD. Dec 24th incr to 203%
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The issue of concern for investors is not the premise but the conclusion. Investors assume that $CRSP $EDIT and $NTLA are the winners. $ARKG is seeing the underlying technology as the winner. Consider the change in fund weight and accum in $TAK $NVS $RHHBY
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$CRSP $EDIT $NTLA are platform advantages that will eventually be acquired or cos will need to rent the platform to third parties. Hundreds of INDs means hundreds of competitors. Smartly $ARKG has reduced weighting for $EDIT from 5.2% to 1.9%. $NTLA from 5.3% to 1.7%
One limiting factor for #CRISPR is the IP landscape though $ARKG believes eventually this will be consolidated in a way that facilitates the growth
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The Genomics Revolution is real but the ability to monetize is driven by appr meds. The $ARKG premise is driven by econ more than lab results but the econ adv are real. Don't be surprised if at least one of these three get acq'd in 2021 but be careful with valuations
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I'm invested in $NVS $RHHBY $PFE primarily because they are bigpharma leaders in this new era. They all have been investing heavily + have been acquiring component tech. I'm building a position in $BIIB for the same reason along with their lead in neuroscience = largest TAM
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I'm invested in $SGMO for combination of platform and pipeline. Far and away superior today. Eventually #CRISRP will overtake them so they need to stay ahead which is why the advancement of ZFP-TF and CAR Tregs is so key. Next Mitochondria which again puts them in lead.
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I've been in $CRSP $EDIT and $NTLA in past but now feel they are all over valued due to "success excess" while acknowledging that there today are only 3 leading platforms with a lot of potential interest, but M&A doesn't drive my investment decisions. Waiting for pullback
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Just as an aside, I started writing about genomic disruptive innovation back in 2013 with #NIPT and later with #Liquidbiopsy. Way too early just like $ARKG if you look at their NAV chart.
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seekingalpha.com/article/147814…
Also wrote about #disruption driven by #Cloud #IoT and #Digitaltransformation. One example from 2017. Point is that this is a loooong lead and longer tail. The cloud/iot TAM was estimated in the #trillions per $MSFT at which point I stopped tracking.
seekingalpha.com/article/407897…
Woods $ARKG interview comments worth noting:
- #Genomics the most inefficiently priced segment. Healthcare the most misunderstood.
- cost declines leading H/C into golden era similar Genentech emerging in 1980s w/20 yrs of rising returns.
bloomberg.com/opinion/articl…
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$ARKG has been accumulating: $NVS $RHHBY $TAK now at 2-3% weight. Don't follow Takeda but other two are leading the transition of big pharma to #GeneTherapy #biopharma.
Top 4 holdings of portfolio: $PACB $TDOC $CRSP $TWST
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The $ARKG investment case was updated in Sept. Mostly new but expect to see more about #liquidbiopsy going forward as cost effectiveness drives reimbursement catalyst
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$ARKG white paper last year also quantified the impacts of #GenomicAge:
- Create trillions in market cap
- Significant decrease in clinical failure rates
- #Diagnostic tests generate tens of billions in C24 rev
- medical #IoT $200b spending C24
- Timeline shows #ZFN contr
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$ARKK is on the top 100 list of #RobinhoodTraders stocks
fool.com/investing/2021…
$ARKG returns thru 2019
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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
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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.
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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
Apr 12, 2021
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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Read 13 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

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