Samarth Kulkarni

CEO and Chairman at CRISPR Therapeutics @CRISPRTX, which is pioneering a new era of gene-based medicines. Tweets are my own.

[Yair Einhorn @yaireinhorn]

1/🚨WOW! CRISPR Therapeutics has presented an updated clinical data from its Phase 1b clinical trial evaluating CTX310 - $CRSP leading In-Vivo CRISPR Cas9 Gene Editing therapy targeting angiopoietin-like protein 3 (ANGPTL3) in patients with severe hypertriglyceridemia (sHTG). The clinical data which was presented at #AHA2026 - the The annual American Heart Association Scientific Sessions, demonstrated that a single administration of CTX310 produced mean reductions from baseline at Day 30 of 65% in circulating ANGPTL3 and 63% in triglycerides (TG) in participants with a median baseline TG level above 1,000 mg/dL. These results show that a single-course treatment with CTX310 can deeply reduce ANGPTL3 and triglycerides in patients with severe hypertriglyceridemia, reinforcing its potential as a one-time “one and done” Gene Editing treatment for sHTG patients.

[Samarth Kulkarni @CrisprSam]

Busy couple of days for our clinical team. Great data for Zugocel in SSc at #ACR2026 and now great data with CTX310 in sHTG at #AHA2026 $CRSP

[CRISPR Therapeutics @CRISPRTX]

Today at the @American_Heart Scientific Sessions, we presented data from our Phase 1b clinical trial evaluating CTX310, our investigational in vivo CRISPR/Cas9 gene editing therapy targeting ANGPTL3, in patients with severe hypertriglyceridemia: Details: https://t.co/OAf2KZZpgR

[Samarth Kulkarni @CrisprSam]

Excited to welcome Siva on board!! $CRSP

[CRISPR Therapeutics @CRISPRTX]

We’re excited to welcome Siva Sakhamuri, Ph.D. as Chief Technical Officer of @CRISPRTX. His 30+ years of technical operations & manufacturing leadership will be important as we advance our gene editing & RNA therapies into late-stage clinical development. https://t.co/SFaFSwl8mv

[NEJM @NEJM]

Correspondence: Durability of CRISPR-Cas9 Gene Editing Targeting ANGPTL3 with CTX310 https://t.co/WCAL8aqz04

#Cardiology #ESCCongress

[CRISPR Therapeutics @CRISPRTX]

We’re excited to announce the acceptance of a poster presentation on our zugo-cel autoimmune clinical program at the upcoming American College of Rheumatology (ACR) Convergence 2026, taking place November 6-11 in Orlando, Florida. Learn more here: https://t.co/VHLjJzQfmy @ACRheum

[BiotechTV @BiotechTV]

𝐇𝐂𝐖 𝟐𝟎𝟐𝟔: @HCWCO Senior Biotechnology Analyst Mitchell Kapoor touches on current biotech sentiment, the cardiovascular space, in vivo CAR-T, and a pancreatic study to watch.

Full video: https://t.co/Lvg8CQFv7j

[Yan Leyfman, MD @YLeyfman]

What if lowering cholesterol didn’t require taking a pill every day?

In a Phase 1a trial, a single dose of CRISPR-Cas9 gene editing therapy CTX310 produced sustained reductions in ANGPTL3 and atherogenic lipids that persisted for up to 1 year.

No daily medication.
No repeated injections.
One-time gene editing.

The concept is striking: instead of continuously treating a pathway involved in lipid metabolism, CRISPR aims to permanently modify the underlying biology.

And importantly, the early safety signal was encouraging—there were no treatment-related dose-limiting toxicities reported in this initial study.

But this is still early clinical development. Phase 1 studies are primarily designed to evaluate safety and establish whether the biological effect is achievable. Larger, longer studies will be needed to determine durability, cardiovascular benefit, and long-term safety.

Still, this represents a fascinating shift in medicine:

From treating disease repeatedly → to potentially editing the biology that drives it.

The era of one-time genetic medicines may be getting closer. 🧬

https://t.co/rLa2zxSw6i

[Samarth Kulkarni @CrisprSam]

Captures it well! https://t.co/pFbIboE4b5

[Samarth Kulkarni @CrisprSam]

Durability data for CTX310 at #ESCCongress and concurrent @NEJM publication. ANGPTL3 reduction is durable along with LDL and Triglycerides paving the way for potential one-time treatments for life! New era in cardiovascular medicine

[CRISPR Therapeutics @CRISPRTX]

We announced positive late-breaking data for CTX310 at @escardio Congress, demonstrating deep and durable reductions in circulating ANGPTL3, triglycerides, and LDL sustained through one year of follow-up. The data are also published in @NEJM. https://t.co/QphPndvjRm #ESCCongress

[ARK Invest @ARKInvest]

64 infusions. $116 million in revenue. Less than 0.1% of eligible patients treated. Wall Street models CASGEVY at $1 to 2 billion. ARK's model says that's billions off. @Ovid_ARK, PhD breaks down CRISPR Therapeutics for our Stock Stories series.

[CRISPR Therapeutics @CRISPRTX]

Our CEO Samarth Kulkarni @CrisprSam joined the Biotech CEO Panel at the @BiotechTV U.S. Science Summit alongside Nello Mainolfi, Jason Kelly, and Kendalle Burlin O'Connell to discuss where the science and industry are headed. Thanks to @bradloncar and @BiotechTV for hosting us.

[David R. Liu @davidrliu]

Experts in gene editing, n=1 medicine development, in vivo delivery, rare diseases, and cell & animal models of genetic disorders have founded the Center for Therapeutic Genetics (CTG), a non-profit effort involving @broadinstitute @BostonChildrens @jacksonlab, as well as others from industry, academia, and patient advocacy organizations, to bring the promise of programmable medicines including base and prime editors to patients suffering from rare genetic diseases. This article from @ginakolata @nytimes frames the compelling need: https://t.co/OyrYCZtWIk

Official announcement: https://t.co/MwJCUarQnX

[John Maraganore 🇺🇸🇬🇷🇺🇦 @JMaraganore]

An honor to meet @oleghereJ , one of the first sickle cell patients to achieve a cure with gene editing.

Our industry efforts to bring innovative medicines to patients can only succeed with the courage of people like Jimmy!

[Ted @BlackInk67]

$VRTX $CRSP: New CASGEVY paediatric data looks strong. In ages 5–11, 11 SCD patients dosed; 8/8 evaluable were VOC-free ≥12 months. In TDT, 15 dosed; 8/8 evaluable achieved transfusion independence ≥12 months. US review underway; UK & Saudi submissions completed. Increases TAM

[avidresearch @avidresearch]

$BDTX and $AVBP comparable based on mPFS.

$BDTX clear advantage in CNS metastases ?

“Robust CNS activity, with 86% CNS ORR; no patients developed de novo brain metastases”

[Alex Harding @AlexHarding7]

I am extremely skeptical of the long-term prospects for in vivo gene therapy with viral vectors that do random or semi-random integration.

[Samarth Kulkarni @CrisprSam]

Congrats to Kathy High and Stu Orkin (and their colleagues) on winning the Breakthrough Prize, both of whom I’ve had the pleasure of working closely with! Luxturna and Casgevy are groundbreaking therapies that will pave the way for many more.

[Samarth Kulkarni @CrisprSam]

Today we shared an update on zugo-cel (formerly CTX112) - best-in-class allogeneic CD19 CART! The data reflect encouraging progress across autoimmune disease and hematologic malignancies and support the continued advancement of zugo-cel across multiple disease settings. @CRISPRTX

[CRISPR Therapeutics @CRISPRTX]

Today, we shared a clinical update on zugo-cel (formerly known as CTX112™), reflecting continued progress across autoimmune disease and hematologic malignancies. Learn more here: https://t.co/eYb2rDwtKv

[Samarth Kulkarni @CrisprSam]

@VertexPharma and @CRISPRTX new data in children ages 5-11 with SCD or TDT were presented today at #ASH25, opening up the possibility of treating these conditions earlier in life.

[Samarth Kulkarni @CrisprSam]

#AHA25 and @NEJM data for CTX310 by @ProfSNicholls . A single in vivo administration safely and durably lowered ANGPTL3, reducing triglycerides and LDL, progress toward applying gene editing to more common diseases. @CRISPRTX