Sek Kathiresan MD
@skathire
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CEO @VerveTx $VERV; Imagine if a one-time treatment led to permanent lowering of LDL cholesterol: that’s what we are developing to treat heart attack
Boston, MA
Joined July 2012
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Best guide on writing a scientific paper (ever?).
Useful to read before each attempt to write.
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Astounding fact I learned today:
Weight of average adult US male
1960: 166 pounds
2002: 191 pounds
😬
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As a cardiology consultant in the hospital, here’s my nomination for the heroes of the pandemic:
1. Nurses in the ER
2. Nurses in the ICU
3. Nurses on the floor
4. Nurses in the dialysis clinic
5. Nurses in the...
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Born in Tirupattur, India
Immigrated to Pittsburgh, PA 1980
Professor Medicine
@harvardmed
CEO
@VerveTx
Assimilated
Most third-world migrants can not assimilate into civil societies.
Prove me wrong.
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Proof-of-concept for an entire new way to treat heart attack:
a gene editing medicine,
administered once in life,
safely turn off a gene -->
permanently lower cholesterol or triglycerides
A ‘Cure for Heart Disease’? A Single Shot Succeeds in Monkeys
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Amazing.
Once weekly injection, 30lbs average weight loss
Phenomenal results for a medicine to treat obesity
Once-Weekly Semaglutide in Adults with Overweight or Obesity | NEJM
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Can we precisely rewrite the genetic code - make a single DNA spelling change - in the liver of a human being for clinical effect?
YES!
Scientific milestone: VERVE-101 provides first proof-of-concept for in vivo DNA base editing in humans
Clinical implication: It’s early…
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Wow, say it with me:
“a functional cure” for sickle cell disease with CRISPR gene editing
All 31 patients with severe SCD, no vaso-occlusive crises (f/u 2 to 32 mos)
Similar durability and efficacy for beta-thal
👏🏽👏🏽👏🏽
@CRISPRTX
@VertexPharma
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Met with postdoc keen on staying in academia & applying for faculty jobs
I asked: "Have you put together a Description of Research Activities?"
👇🏽mine from April 2007 as I was just finishing my research training & applying for research faculty positions
Some thoughts...thread.
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Wow, some good news from 2016. The development of a vaccine with 100% efficacy against Ebola.
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Wow, wow, wow!
In SELECT trial of patients with obesity (mean BMI 33, n>17,000) & prior atherosclerotic cardiovascular disease (but no diabetes),
GLP-1 agonist semaglutide 2.4mg once-weekly
reduced risk of CV death, heart attack, or stroke (RRR 20%)
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IMHO, biggest problem in biomedical science literature
👇🏽
Insane level of focus on "novelty" over reproducibility/"incremental" progress
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A home run!
@intelliatweets
shows for first time in humans that in vivo liver gene editing (CRISPR-Cas9 mRNA + guide RNA packaged in a lipid nanoparticle)
is going to work (REALLY WELL)
At dose of just 0.3 mg/kg in 3 humans,
plasma TTR reductions:
80%, 84%, & 96%
No AEs
😳
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News:
After 22y
@MassGeneralNews
@broadinstitute
understanding risk & resistance to heart attack,
I am moving to new role as CEO
@VerveTx
- a biotech we have founded
to develop gene editing Rx that permanently reduce risk of coronary disease in adults
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Humanity.
Thank you. 🙏🏽
A San Francisco Doctor Answering the Call to New York -
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Want something "unredacted" to read today
See here
Why am I so heavy?
Obesity largely viewed as disease of willpower or poor lifestyle choices
BUT…our research out today shows:
1. Genetics plays a large role in how heavy one gets...
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Best news in months:
@Regeneron
"its scientists have now isolated hundreds of antibodies that neutralize virus, & they’re sorting through them — as well as antibodies isolated from ppl who have recovered from COVID-19 — to find best 2 with which to create a cocktail treatment"
Regeneron's sped up its timeline for human trials of antibody therapy for
#COVID19
by a couple months. Now aims to have doses for testing by early summer. George Yancopoulos: "There are always so many moving parts, but we’re hitting our best timelines"
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He would have been 50 today.
We lost him to a heart attack at 42.
Working toward a future with fewer such losses.
🙏🏽
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Wow. Every scientist should read. Ten simple rules for structuring papers
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Thrilled to announce that
@US_FDA
has cleared our
*investigational new drug (IND)* application
for VERVE-101
FDA reviewed our hold response which included:
1. interim clinical data from the ongoing heart-1 trial currently being conducted ex-U.S.
2. pre-clinical data to address
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1st day, new job 👇🏽
1st new employer in 22y
Nervous 😬
Super excited 😃
🙏🏽
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Wow (and a double Wow!).
*52 lb* weight loss (22.5%)
at highest dose of tirzepatide (a dual GLP-1 receptor / GIP receptor agonist from
@LillyPad
)
GLP-1 class truly going to revolutionize the treatment of obesity!!!
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After being told of BRCA1 mutation,
up to 50% women opt for prophylactic mastectomy
Should this be so?
For "monogenic" forms breast ca, colorectal ca, & heart attack, we find polygenic background powerfully modifies risk
New on
@medrxivpreprint
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Quite embarrassing for
@JAMA
to continue to publish these horribly confounded nutritional epidemiology studies.
In this pooled US cohort study, consumption of dietary cholesterol or eggs was associated with higher risk of incident CVD and all-cause mortality in a dose-response manner
#ACC19
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So, you weigh 250lb and want to lose 50lb
Can you do so safely with a once-a-week medicine?
Looks like tirzepatide will do the trick.
Amazing!
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25 years
9,125 days
219,000 hours
13 million minutes
3 loved children
Lots of joy, some sorrow
Much laughter
Happy Anniversary Neela 😘🙏🏽
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“These are stunning results,” says gene editing researcher and cardiologist Kiran Musunuru of the University of Pennsylvania, who was not involved in the trial. “It exceeds all my expectations.”
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Idea: 2016
Company: 2018
NHP data: 2020
1st patient dosed: 2022
1st human data for in vivo liver base editing in patients with heterozygous familial hypercholesterolemia and ASCVD
👇🏽
@American_Heart
@VerveTx
$VERV
Stay tuned!
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@StevenSalzberg1
Wow
@StevenSalzberg1
.
So much respect for this thoughtful response to the feedback.
Simply wonderful example you are setting.
👍🏽👏🏽👌🏽
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9 years ago today (after a heart attack)
May he RIP
May we develop a solution to avert similar tragedies
🙏
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When listening to a new consult presentation, 2 things I would love to hear *first*:
1. reason for consult
2. what brought pt to hospital
What I hear first most often:
82yo with CKD on HD, T2D, AAA s/p repair, s/p CABG, hyperlipidemia, HFrEF, PAF on Xarelto, COPD on home O2...
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Intense hunt by biotech (i.e. Calico) & billionaires to promote lifespan (healthy aging).
Here, a genetic study reveals the secret: avoid a heart attack!
Most gene variants identified for lifespan are just common alleles *protective* for heart attack.
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30 min physical activity, at least 5 days a week
How?
Found a parking spot 15 min walk from office
😊
Happy, healthy 2020 everyone!
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Wowza!
Losing 20lbs with GLP-1 agonist (semaglutide)
averts heart attacks/strokes
and
saves lives
(in patients who are overweight, without diabetes but with pre-existing atherosclerotic cardiovascular disease)
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Grateful to
@American_Heart
for this wonderful honor - the Joseph A. Vita Award.
I had the opportunity to work with Dr. Vita as fellow at the Framingham Heart Study; he was a wonderful man and a terrific scientist.
Harvard scientist gets American Heart Association award for findings of inherited basis for cardiovascular disorders
#AHA18
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New GLP1 Agonist orfoglipron
- once daily
- oral
- non-peptide
- no food or water restriction
2 Phase 2 trials
Weight: up to 14.7% weight loss
T2D: up to 2.1% hemoglobin A1C reduction
Spectacular new option in the works.
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Best news of day (even better than event in DC)
Preliminary study from Sahin of
@BioNTech_Group
@pfizer
@BioNTech_Group
vaccine induced neutralizing antibody response to UK variant similar to wild-type COVID virus
👍🏽
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Tweeps,
Thank you very much for kind words. Grateful to my family, mentors, trainees, institutions, and to this great country.
From immigration 1980 with my brother (L) to
#AHA17
honor of our scientific contributions yesterday (R).
A testament to power of opportunity in 🇺🇸
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Having led company from my basement for the last 9 months 😳,
nice to visit our new
@VerveTx
offices in 500 Tech Square for first time...
👌🏽
@kendallnow
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@ashishkjha
@bijans
Ashish,
once we get through this wave (let's say end Feb), I do wonder if we need to change our thinking re testing.
For endemic viruses (eg Flu), testing is typically done in people with symptoms (and usually just severe symptoms).
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"healthy", then heart attack
Why?
Cool new study sheds light:
*9500 asymptomatic folks
*Test each with coronary CT angiogram (L)
*follow over time (R)
46% - atleast some heart artery blockages
5% - obstructive & extensive blockages!
Lots heart disease under radar
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2018:
@VerveTx
founded with concept of one-time Rx to lower LDL as low as possible for as long as possible
Today: we are announcing
dosing of *first patient* with VERVE-101,
an in vivo CRISPR base editing medicine
🙏 to patient volunteer
$VERV
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An oral drug which:
1. leads to meaningful weight loss
2. lowers all-cause mortality ~50%
3. lowers CV mortality
4. lowers hgA1C
Wow!
Oral Semaglutide and Cardiovascular Outcomes in Patients with Type 2 Diabetes | NEJM
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👇🏽 what I hope will be our most impactful discovery to date
An enzyme whose loss of function lowers blood cholesterol & *protects* against fatty liver & cirrhosis
Kudos to
@connoremdin
whose clever analysis of public data (
@ukbiobank
) led to observation
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If LDL⬇️for a long time, really hard to get heart attack
Cool new evidence
Some lucky people born with DNA variation that naturally lowers LDL
~50 mg/dl⬇️LDL, lifelong
&
risk for heart attack/death is ~50%⬇️!
So, for every 1 mg/dl⬇️LDL lifelong,
~1%⬇️risk heart attack/death
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Quite an awesome email to wake up to:
About $10M worth of data to be given to you for free
*and*
the data generator is asking for input on file formats
👏🏽👏🏽
@uk_biobank
- emerging as the world standard for human biology research and open data
👇🏽
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Unbelievable. 😱
The famous ‘Mediterranean diet’ is awesome study seems to have had serious randomization issues affecting >20% enrolled participants
Retracted and republished!
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OK.
For all the people who think these trends are because the US has become taller:
🤦
average height US
1960: 5ft 8in (male); 5ft 3in (female)
2002: 5ft 9in (male); 5ft 4in (female)
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What is inherited basis for heart attack?
A new quantitative (genome-wide polygenic score) that captures inherited liability to heart attack
Normal distribution in population
For those in top 8% of scores, 3-4-fold higher risk than all others
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If you develop a once-&-done gene editing med to durably lower LDL cholesterol & Rx heart attack, what do you need?
Capital
Today,
@VerveTx
announces Series B fund raise of $94M led by
@Wellington_Mgmt
& co-led by
@cascapital
, with 9 new investors. 🙏
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Often get asked about "founding story"
@VerveTx
None other than
@American_Heart
"One Brave Idea" competition to cure coronary heart disease
Our submission from April 2016 👇
Idea incubated with
@GVteam
'16-'18
Series A funding August '18
1st patient dosing - planned 2022
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South Asians: about 1/4 of world population
Representation in genetic studies: almost 0
😕
The remarkable and persistent lack of diversity in genomic studies
@NatureMedicine
@SFatumo
@KKuchenbaecker
@tchikowore1
@Ayub1985
@genetisaur
And a plan to fix it
@LHSTM
@UCL
@uclbiosciences
@MRC_Uganda
@H3Africa
@PGCgenetics
@PRSdiversity
@H3ABioNet
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"Pfizer, Moderna, Johnson & Johnson, GlaxoSmithKline, Sanofi and possibly others"
Drugmakers are planning a public pledge to not send any Covid-19 vaccine to the FDA for review without extensive safety and efficacy data, sources say via
@business
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1st patient ever treated by CRISPR gene editing medicine: for sickle cell disease
👏🏽👇🏽🙏🏽
@VertexPharma
@CRISPRTX
CRISPR Gene-Editing Tool: 1st Patient With Genetic Disorder Treated In U.S. : Shots - Health News : NPR
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You thought taking off your shoes was annoying.
How about a quick blood test before flying? 😱
Emirates becomes first airline to conduct on-site rapid COVID-19 tests for passengers
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What’s the best text a son can get from elderly parents during the pandemic?
👇🏽👌🏽🙏🏽
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Nice reminder today:
Getting LDL cholesterol
as low as possible for as long as possible
is the best way to treat or prevent heart attack
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👇🏽
Verve Therapeutics Announces Pricing of Initial Public Offering | Business Wire
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Happy Diwali 2020! 🪔
Diwali is a Hindu holiday which symbolizes the spiritual victory of light over darkness, good over evil, and knowledge over ignorance.
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👇23 years ago today in Tarrytown, NY
Grateful to have found a wonderful partner to share the highs (and lows) of life.
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A new era of CRISPR-based medicines with single treatments that offer curative potential!
Kudos to
@VertexPharma
and
@CRISPRTX
FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease | FDA
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Amazing!
“After the CRISPR therapy, Gray’s total hemoglobin reached normal levels; of which 47% was fetal hemoglobin. It’s thought that a fetal hemoglobin level of 25%-30% is sufficient to “cure” a patient with sickle cell disease.“
New era of single, transformative therapies
$VRTX $CRSP news! —> First CRISPR treatment for blood diseases shows early benefits in two patients via
@sxbegle
and me.
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Grateful to announce👇🏽
Thanks to
@GVteam
(lead),
@rtnarch
,
@FPrimeCapital
, Biomatics, Partners Innovation Fund
and 2 new investors Wellington &
@cascapital
Verve raises another $63m to develop gene-editing treatment for heart disease via
@BostonGlobe
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👇🏽
First CRISPR-based medicine approved
Amazing new option for patients with sickle-cell disease & b-thal
MHRA authorises world-first gene therapy that aims to cure sickle-cell disease and transfusion-dependent β-thalassemia -
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Terrific must-read
@TheLancet
on COVID-19 epidemiology
Did you know:
"A simple calculation gives fraction likely to be infected without mitigation. This fraction is roughly
1–1/R0. With R0 values for COVID-19 in China around 2·5...~60% of population would become
infected."
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Cool new study from
@Regeneron
Identification loss-of-function variants in GPR75 gene that protect against obesity
Het LoF carriers about 12lb lighter.
Sequencing of 640,000 exomes identifies GPR75 variants associated with protection from obesity
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0.3% of a very large number is a very large number
That’s it.
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👇🏽a cure for sickle cell seems to be coming. Wow.
Vertex and CRISPR Therapeutics showed their experimental therapy kept two sickle patients from experiencing painful incidences and kept patients with another blood disorder from requiring transfusions.
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@ScottGottliebMD
key contributor to the sentinel surveillance of COVID-19 in Seattle was the Seattle Flu Study, a collaborative effort by the Brotman Baty Institute, UW Medicine, Fred Hutch, and Seattle Children’s
cc:
@JShendure
head Brotman Baty
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@VerveTx
is thrilled to announce
*Clearance* of first VERVE-101 clinical trial application (CTA)
🎉🎉
Expect to treat 1st patient with HetFH in mid-2022
Expect clinical data in 2023
As part of planned global regulatory strategy,
CTA in UK & IND in US are up next
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In
@CircAHA
, we publish today pre-clinical data for VERVE-101
Of note,
@American_Heart
tells us that for Scientific Sessions '22, this abstract the highest ranked one of all submitted from U.S.
#AHA22
Press release:
Paper:
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So, three teenagers in the house as of today 😃😬
Happy 13th Devan
Boy, they grow up fast.
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GLP-1 agonists do seem to be the *miracle* drug for the 21st century obesity epidemic:
Lose weight, lower glucose, lower triglycerides, lower BP
*AND*
Lower risk of heart attack
Oh, also, potential benefits re NASH and sleep apnea
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We are thrilled to gain
@EliLillyandCo
as a new partner for
@VerveTx
PCSK9 & ANGPTL3 programs👇
Lilly has purchased from
@BeamTx
all of their previously held product rights in PCSK9 & ANGPTL3 programs for a $600M total potential deal value.
For the PCSK9 & ANGPTL3 product…
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In first test for base editing, Verve’s one-time treatment lowers cholesterol levels in patients via
@statnews
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2 powerful relationships in medicine:
(L) For every 39 mg/dL pharmacologic LDL lowering ~5y,
⬇️21% in CV events
(regardless starting LDL)
Now, we have:
(R) For every 5 mmHg pharmacologic BP lowering ~4y,
⬇️10% in CV events
(regardless starting BP)
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