Sek Kathiresan MD
@skathire
Followers
30K
Following
50K
Media
4K
Statuses
25K
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
Joined July 2012
Best guide on writing a scientific paper (ever?). Useful to read before each attempt to write.
25
1K
3K
Astounding fact I learned today:. Weight of average adult US male. 1960: 166 pounds.2002: 191 pounds . 😬.
234
635
2K
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. .
64
368
2K
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.
25
116
1K
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
49
491
1K
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
50
270
1K
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
41
246
1K
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 .
21
207
934
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.
23
241
855
Wow, some good news from 2016. The development of a vaccine with 100% efficacy against Ebola.
3
358
765
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%).
31
220
794
IMHO, biggest problem in biomedical science literature . 👇🏽. Insane level of focus on "novelty" over reproducibility/"incremental" progress
16
93
580
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. 😳
22
168
610
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.
100
91
600
Humanity. Thank you. 🙏🏽. A San Francisco Doctor Answering the Call to New York -
10
99
486
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.
19
249
490
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"
5
141
482
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
23
77
485
He would have been 50 today. We lost him to a heart attack at 42. Working toward a future with fewer such losses. 🙏🏽
21
17
482
Wow. Every scientist should read. Ten simple rules for structuring papers
2
285
454
1st day, new job 👇🏽. 1st new employer in 22y. Nervous 😬.Super excited 😃.🙏🏽
19
11
423
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 .
15
204
408
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.
16
87
367
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!.
11
66
375
25 years.9,125 days.219,000 hours.13 million minutes.3 loved children.Lots of joy, some sorrow.Much laughter. Happy Anniversary Neela 😘🙏🏽
17
1
346
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!
17
44
356
“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.”
4
80
345
@StevenSalzberg1 Wow @StevenSalzberg1. So much respect for this thoughtful response to the feedback. Simply wonderful example you are setting. 👍🏽👏🏽👌🏽.
2
12
322
9 years ago today (after a heart attack). May he RIP. May we develop a solution to avert similar tragedies. 🙏
23
4
346
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. .
33
89
346
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.
11
134
338
30 min physical activity, at least 5 days a week. How?. Found a parking spot 15 min walk from office. 😊. Happy, healthy 2020 everyone!.
12
23
324
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)
11
78
332
"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
21
100
326
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.
29
34
317
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.
11
83
329
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 🇺🇸
22
72
312
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. 👍🏽.
8
91
299
@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).
11
5
319
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
15
3
312
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
17
100
301
👇🏽 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.
5
121
289
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
15
81
295
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. 👇🏽
7
101
272
Unbelievable. 😱 . The famous ‘Mediterranean diet’ is awesome study seems to have had serious randomization issues affecting >20% enrolled participants. Retracted and republished! .
24
182
285
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).
18
33
268
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.
11
139
271
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. 🙏.
24
17
268
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
12
34
270
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
11
73
263
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
13
81
245
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
3
97
244
What’s the best text a son can get from elderly parents during the pandemic?. 👇🏽👌🏽🙏🏽
10
2
236
👇🏽. Verve Therapeutics Announces Pricing of Initial Public Offering | Business Wire
38
13
251
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.
21
28
244
👇23 years ago today in Tarrytown, NY. Grateful to have found a wonderful partner to share the highs (and lows) of life.
19
0
238
Happy Diwali 2020! 🪔. Diwali is a Hindu holiday which symbolizes the spiritual victory of light over darkness, good over evil, and knowledge over ignorance.
8
24
233
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.
5
95
233
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
7
57
244
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.
29
21
240
👇🏽. 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 -
3
56
243
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."
4
111
237
0.3% of a very large number is a very large number. That’s it.
4
19
221
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
5
87
231
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:
2
60
234
@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.
5
36
214
👇🏽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.
3
26
229
@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
6
23
231
So, I grew up in Tamilnadu, a state in southern India. Imagine my delight when my father sent me (L). @VerveTx shows up in Dinamalar, largest Tamil daily paper 😀. article (L), my father's translation (R). boxed in white is "CRISPR gene editing" in Tamil!.
11
4
233
So, three teenagers in the house as of today 😃😬. Happy 13th Devan. Boy, they grow up fast.
9
4
233
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.
As @LillyPad invests heavily in a GLP-1 based pipeline for #T2D #obesity #NASH Dulaglutide annualizing at 7.3 B USD
11
47
232
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
7
17
223
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)
8
87
234