John Maraganore 🇺🇸🇬🇷🇺🇦

Founding CEO of @Alnylam. Pioneered RNAi therapeutics as a whole new class of medicines for patients. Now bioentrepreneur-at-large. Opinions are my own.

[Adam Feuerstein ✡️ @adamfeuerstein]

The PCSK9 pill is here. FDA approved $MRK Lipfrenda.

https://t.co/c2laNybXZa

[Eric Topol @EricTopol]

"The world’s largest drugmaker is betting big on preventive medicines"
The future of medicine is not just to come up with more treatments, which has been the fixation for pharma companies. It can and should be to prevent diseases, with what I call "prevmeds." We're starting to see a shift.
https://t.co/doUTytIFHi

[BioCentury @BioCentury]

.@IamBIO, No Patient Left Behind call for withdrawal of OMB rule politicizing science funding; PhRMA wants revisions -- via BioCentury's @SteveUsdin1 https://t.co/HHydvt0vPk

[John Maraganore 🇺🇸🇬🇷🇺🇦 @JMaraganore]

👏👏👏

[Svati Shah @SvatiShah]

Congratulations to two of the most incredible leaders I know! @NPagidipati is the new @DukeHeartCenter Section Chief of #preventivecardiology. She has a bold vision for clinical care and research of preventive approaches to reduce CVD burden

[John Maraganore 🇺🇸🇬🇷🇺🇦 @JMaraganore]

Super exciting data! So thrilled to be part of this transformative effort at @Hemab_Tx!

[Hemab Therapeutics @Hemab_Tx]

Current treatment options for Von Willebrand disease (VWD) are limited, but what if we could address the underlying biological drivers of the disease with a subcutaneous, non‑replacement approach?

That is the vision behind HMB-002, our novel monovalent human antibody designed for the prophylactic treatment of VWD. This week at the 2026 @isth Congress, we are proud to share updated clinical data.

Check out our key takeaways from the ISTH 2026 oral presentation and why we’re progressing to multiple‑dose assessment in the ongoing Phase 1/2 study.

And read more about HMB-002 and HMB-003 at https://t.co/EF7RVMrt6O

🗓️ UPCOMING: Sutacimig updates on Tuesday!

[John Maraganore 🇺🇸🇬🇷🇺🇦 @JMaraganore]

I admittedly have a “dog in the race” here, but I couldn’t agree MORE with @BioCentury and @StephenPHansen!

The facts, are the facts, after all!

[BioCentury @BioCentury]

siRNAs are better than ASOs: a Perspective by @StephenPHansen https://t.co/SCTyQPxkb4

[John Maraganore 🇺🇸🇬🇷🇺🇦 @JMaraganore]

1. RNAi is not ASO
2. TTR Kd with Vutri > Eplon…
Nucre>>Eplon
3. HR for Taf + Vutri vs. Taf is 0.66 in HELIOS-B
4. HR for Taf + Eplon vs. Taf is likely >1.0 (!!) in CardioTTRansform (inferred based on 0.71 monotherapy HR and 81% of patients on stabilizer)… ASO creating harm?

[Ronald Witteles @Ron_Witteles]

The main issue: Stabilizers are really good therapies! We have not known the clear answer to the 'combination therapy' question despite a favorable signal with tafamidis +/- vutrisiran in HELIOS-B (mortality curves shown below).

[John Maraganore 🇺🇸🇬🇷🇺🇦 @JMaraganore]

Not great news for patients, unfortunately, since more Rx options always best…

But also a reminder that all silencers are NOT created equal and that deeper and more durable silencing achieved with RNAi vs. ASOs.

[Dirk Haussecker @RNAiAnalyst]

$ions Thursday morning SHOCKER. CARDIO-TTRansform in ATTR-CM did not meet primary.

https://t.co/sINzlQSzTS

Presumably too many patients on stabilizers (81% by end of study) to see a monotherapy TTR ASO knockdown effect.

Great news for $ALNY, some caution for $ntla study as investors will question whether enough patients there on mono.

Still, think that the power of sustained, genetic TTR lowering gives it much better chance at succeeding in combo patients than eplontersen. $bbio

[John Maraganore 🇺🇸🇬🇷🇺🇦 @JMaraganore]

Really enjoyed the 8th Annual RNARx Conference at the UMass RNA Therapeutics Institute.

Here’s a cool infographic of my talk.

Much more on this in an upcoming book… stay tuned!

[John Maraganore 🇺🇸🇬🇷🇺🇦 @JMaraganore]

Congrats John!

[BU Amyloidosis Center @BU_Amyloidosis]

Join us in wishing Dr. John Berk a happy retirement after 30+ years of dedicated service to the Amyloidosis Center! Our multidisciplinary clinical team continues with his legacy of excellent patient care. Thank you, Dr. Berk!

[Messi World @M10GOAT]

تعليق المعلق الأرجنتيني بابلو غيرالت على هدف التعادل من الأسطورة ليو ميسي 😍🐐 https://t.co/Wysh7WYYnp

[BioCentury @BioCentury]

Takeda's orexin program: The hits, the misses, and the payoff. BioCentury's @LaurenMMartz speaks with $TAK researchers on how they executed the oveporexton program for narcolepsy https://t.co/QrMi2a9yc4

[Matthew Herper @matthewherper]

So last week I spoke to @AnthropicAI CEO @DarioAmodei about how AI could change biotech. I also got to speak to other Anthropic execs, large pharmaceutical CEOs, and GLP-1 pioneer @lotte_bjerre.

Here's what I learned: The most convincing thing to me is that Amodei recognized the limitations of biotech in way he didn't in his 2024 essay, “Machines of Loving Grace.” He forecasts that someday AI will allow researchers to make a decade's worth of progress every year.

He said he's "very specifically not saying we’re going to get all this crazy stuff by 2028 or something. There’s simply no way to do it."

I asked Amodei why we should believe in this tech/biotech revolution when past ones have failed. Pharma execs listed every reason to be cautious -- and why they'd be happy with 40%, 10%, or 5% gains -- and the nightmare scenarios that could make it all worse.

But Lotte said this: "I would say AI is not going to replace scientists. I’m not worried about that at all. But scientists who are not using AI are going to get replaced."

[John Maraganore 🇺🇸🇬🇷🇺🇦 @JMaraganore]

Happy 250th America! The greatest country in the world, even with its challenges!

Here, anyone can come from anywhere in the world to become an American, just like my Mom and Dad over 75 years ago!

[John Maraganore 🇺🇸🇬🇷🇺🇦 @JMaraganore]

Looking at life thru rose-colored glasses right now…

Happy 4th to all! 250!!! 🇺🇸🇺🇸🇺🇸

[John Maraganore 🇺🇸🇬🇷🇺🇦 @JMaraganore]

Wonderful to see this turnaround… and for their Sickle Cell and b-thal products helping more and more patients… and leading to profitability!

I believe a first of many more to come with gene Rx.

[STAT @statnews]

Bluebird Bio was a dark biotech morality tale when David Meek bought it. He says the new company, Genetix, is already making money. https://t.co/5wN6TC6xT5

[John Maraganore 🇺🇸🇬🇷🇺🇦 @JMaraganore]

The start of an exciting new chapter for Thymmune and its entrepreneurial founder Stan Wang!!!

Thymmune was pioneering thymic cell engineering for a range of rare and more prevalent Dzs.

Stan is a #TermeerFellow.

Long live the Thymus, for all!

[Bio Stocks™ @BioStocks]

$UTHR acquires Thymmune Therapeutics for $140M in cash

[Republicans against Trump @RpsAgainstTrump]

Ronald Reagan: “You can go to live in France, but you cannot become a Frenchman…But Anyone, from any corner of the Earth, can come to live in America and become an American…This, I believe, is one of the most important sources of America's greatness.”🇺🇸

[John Maraganore 🇺🇸🇬🇷🇺🇦 @JMaraganore]

Amazing! A major scientific milestone!

Some will criticize and fear monger, but much to learn here!

[The New York Times @nytimes]

Breaking News: For the first time, researchers have created a cell with most of the hallmarks of life. https://t.co/jkwKMXfkVh