Scott Vafai, MD (@scottvafai) 's Twitter Profile
Scott Vafai, MD

@scottvafai

SVP of Clinical Development @VerveTx. Formerly @sanofi, @broadinstitute, @MassGeneralNews, @BrighamWomens. All opinions are my own.

ID: 716339346501197824

calendar_today02-04-2016 18:59:43

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Sek Kathiresan MD (@skathire) 's Twitter Profile Photo

2018: Verve Therapeutics 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 globenewswire.com/news-release/2…

Antonio Regalado (@antonioregalado) 's Twitter Profile Photo

Someone got their liver gene edited for PCSK9 in New Zealand. Could lower bad blood cholesterol by 60%. First genetic therapy with a prevention angle. In the future, maybe, one-and-done editing at age 30 for all of us. End of clogged arteries? technologyreview.com/2022/07/12/105…

Sek Kathiresan MD (@skathire) 's Twitter Profile Photo

Excited to announce today: development candidate for 2nd program (VERVE-201 targeting the ANGPTL3 gene) to treat homozygous FH as well as ASCVD data to be presented by Amit V. Khera European Society of Cardiology 2022 Press release: ir.vervetx.com/news-releases/… Data: ir.vervetx.com/static-files/3… $VERV

Excited to announce today: 

development candidate for 2nd program 

(VERVE-201 targeting the ANGPTL3 gene) 

to treat homozygous FH as well as ASCVD

data to be presented by <a href="/amitvkhera/">Amit V. Khera</a> <a href="/escardio/">European Society of Cardiology</a> 2022

Press release: ir.vervetx.com/news-releases/…

Data: ir.vervetx.com/static-files/3…

$VERV
Sek Kathiresan MD (@skathire) 's Twitter Profile Photo

Delighted to announce a Clinical Trial Authorization (CTA) clearance by UK MHRA (Medicines and Healthcare products Regulatory Agency) for VERVE-101 as investigational Rx for patients with heterozygous familial hypercholesterolemia ir.vervetx.com/news-releases/… $VERV Verve Therapeutics

Delighted to announce a

 Clinical Trial Authorization (CTA) clearance 

by UK MHRA (Medicines and Healthcare products Regulatory Agency) 

for VERVE-101

as investigational Rx for patients with heterozygous familial hypercholesterolemia 

ir.vervetx.com/news-releases/…

$VERV <a href="/VerveTx/">Verve Therapeutics</a>
Josh Knowles (@joshuawknowles) 's Twitter Profile Photo

FH is the MOST COMMON morbid genetic condition and affects about 1 in 200 worldwide. It leads to lifelong high LDL-cholesterol and greatly increases the risk of #HeartDisease. #KnowFH

FH is the MOST COMMON morbid genetic condition and affects about 1 in 200 worldwide. It leads to lifelong high LDL-cholesterol and greatly increases the risk of #HeartDisease. #KnowFH
Vamsi Mootha (@vamsimootha) 's Twitter Profile Photo

Today, we and ⁦⁦Rebecca Ganetzky⁩ report ⁦NEJM⁩ a remarkable case of twin boys with euthyroid hyper-metabolism (low body weight despite high caloric intake), due to a de novo mutation in mito CV that uncouples respiration from ATP synthesis. nejm.org/doi/full/10.10…

Sek Kathiresan MD (@skathire) 's Twitter Profile Photo

For @VerveTx targets, 2 criteria: human genetics & human pharmacology VERVE-201 targets ANGPTL3 Human genetics (L): complete deficiency ANGPTL3 = low blood lipids, no liver fat New human pharmacology (R): siRNA ArrowheadPharma, mimics human genetics! researchsquare.com/article/rs-209…

For @VerveTx targets, 2 criteria:
human genetics &amp; human pharmacology

VERVE-201 targets ANGPTL3

Human genetics (L): complete deficiency ANGPTL3 = low blood lipids, no liver fat

New human pharmacology (R): siRNA <a href="/ArrowheadPharma/">ArrowheadPharma</a>, mimics human genetics!

researchsquare.com/article/rs-209…
Sek Kathiresan MD (@skathire) 's Twitter Profile Photo

In Circulation, we publish today pre-clinical data for VERVE-101 Of note, American Heart Association tells us that for Scientific Sessions '22, this abstract the highest ranked one of all submitted from U.S. #AHA22 Press release: globenewswire.com/news-release/2… Paper: ahajournals.org/doi/10.1161/CI…

In <a href="/CircAHA/">Circulation</a>, we publish today pre-clinical data for VERVE-101

Of note, <a href="/American_Heart/">American Heart Association</a> tells us that for Scientific Sessions '22, this abstract the highest ranked one of all submitted from U.S. 

#AHA22 

Press release: globenewswire.com/news-release/2…

Paper: ahajournals.org/doi/10.1161/CI…
Sek Kathiresan MD (@skathire) 's Twitter Profile Photo

👇news Verve Therapeutics and Eli Lilly and Company establish global collaboration to advance an in vivo gene editing program targeting Lipoprotein(a) for the treatment of atherosclerotic cardiovascular disease ir.vervetx.com/news-releases/…

👇news

<a href="/VerveTx/">Verve Therapeutics</a> and <a href="/EliLillyandCo/">Eli Lilly and Company</a>

establish global collaboration to advance

an in vivo gene editing program targeting

Lipoprotein(a)

for the treatment of atherosclerotic cardiovascular disease

ir.vervetx.com/news-releases/…
Sek Kathiresan MD (@skathire) 's Twitter Profile Photo

Thrilled to announce that U.S. 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

Thrilled to announce that <a href="/US_FDA/">U.S. FDA</a> 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
Sek Kathiresan MD (@skathire) 's Twitter Profile Photo

We are thrilled to gain Eli Lilly and Company as a new partner for @VerveTx PCSK9 & ANGPTL3 programs👇 Lilly has purchased from Beam Therapeutics all of their previously held product rights in PCSK9 & ANGPTL3 programs for a $600M total potential deal value. For the PCSK9 & ANGPTL3 product

We are thrilled to gain <a href="/EliLillyandCo/">Eli Lilly and Company</a> as a new partner for @VerveTx PCSK9 &amp; ANGPTL3 programs👇

Lilly has purchased from <a href="/BeamTx/">Beam Therapeutics</a> all of their previously held product rights in PCSK9 &amp; ANGPTL3 programs for a $600M total potential deal value.

For the PCSK9 &amp; ANGPTL3 product
Sek Kathiresan MD (@skathire) 's Twitter Profile Photo

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

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
Sek Kathiresan MD (@skathire) 's Twitter Profile Photo

In first test for base editing, Verve’s one-time treatment lowers cholesterol levels in patients statnews.com/2023/11/12/in-… via STAT

Sek Kathiresan MD (@skathire) 's Twitter Profile Photo

Glad to announce the dosing of the first patient with VERVE-102 in the Heart-2 clinical trial VERVE-102: *in vivo base editor to permanently turn off the PCSK9 gene in liver and durably lower blood LDL-C *delivered using Verve Therapeutics proprietary GalNAc-LNP delivery system as a

Glad to announce the dosing of the first patient with VERVE-102 in the Heart-2 clinical trial

VERVE-102:
*in vivo base editor to permanently turn off the PCSK9 gene in liver and durably lower blood LDL-C
*delivered using <a href="/VerveTx/">Verve Therapeutics</a> proprietary GalNAc-LNP delivery system as a
Sek Kathiresan MD (@skathire) 's Twitter Profile Photo

@VerveTx 3Q earnings: 1. VERVE-102 targeting PCSK9 being tested in Heart-2 trial *treated 7 patients now across 2 dose levels (0.3 mg/kg and 0.45 mg/kg) *well tolerated, no serious adverse events, no clinically significant lab abnormalities *escalation to next dose after

@VerveTx 3Q earnings:

1. VERVE-102 targeting PCSK9 being tested in Heart-2 trial

*treated 7 patients now across 2 dose levels (0.3 mg/kg and 0.45 mg/kg)
*well tolerated, no serious adverse events, no clinically significant lab abnormalities
*escalation to next dose after
Amit V. Khera (@amitvkhera) 's Twitter Profile Photo

We are hiring! Delighted to share an opportunity to join the clinical development team at Verve Therapeutics Eli Lilly and Company , where we are on a mission to transform the care of ASCVD through single-course gene editing medicines! Apply here: lnkd.in/eVDDP85i

We are hiring!

Delighted to share an opportunity to join the clinical development team at Verve Therapeutics <a href="/EliLillyandCo/">Eli Lilly and Company</a> , where we are on a mission to transform the care of ASCVD through single-course gene editing medicines!

Apply here:
lnkd.in/eVDDP85i