Aron Jaffe

What if RFK Jr. reacted to his cousin’s terminal cancer as JFK reacted to his baby’s death? When President Kennedy’s newborn son died in 1963, it galvanized research. A new Kennedy family tragedy should spur RFK Jr. to support cancer research, not cut it.

What if RFK Jr. reacted to his cousin’s terminal cancer as JFK reacted to his baby’s death? www.statnews.com/2025/11/26/t... via @statnews.com

Here is how we know that vaccines do not cause autism Vaccines do not cause autism. You’ve almost certainly read that before — but often without a full explanation of how we know it to be true. Here's how we know.

www.statnews.com/2025/02/03/v...

Triple-drug therapy for cystic fibrosis - Lasker Foundation Jesús (Tito) González, Paul A. Negulescu, Michael J. Welsh

Congratulations to Mike Welsh @iowamed.bsky.social, Paul Negulescu @vertexpharm.bsky.social, & Jesus Gonzalez
on their well-deserved award. If you're not familiar with the path to CFTR therapies, it's worth a read.

laskerfoundation.org/winners/comb...

Reassessment of antibody-based detection of the murine T cell GLP-1 receptor Glucagon-like peptide 1 receptor (GLP-1R) agonists exhibit anti-inflammatory actions, yet the importance of direct immune cell GLP-1R signaling remains uncertain. Although T cells respond to GLP-1, lo...

Reassessment of antibody-based detection of the murine T cell GLP-1 receptor
cell.com/cell-metabol...

'Our findings reveal caveats governing use of multiple widely used GLP-1R antibodies, reemphasizing the importance of rigorous antibody validation'

Thanks @danieljdrucker.bsky.social & team

I inserted myself into a conversation at a bar about Covid and vaccines. Here’s what happened What happened when a public health scientist overheard four men sharing misinformation about Covid — and decided to talk to them about it.

I inserted myself into a conversation at a bar about Covid and vaccines. Here’s what happened www.statnews.com/2025/09/06/c... via @statnews.com

How we’re rebuilding the Weizmann Institute — and our hopes for a better future Scientific research is at the heart of Israel’s success and has worldwide benefits. Our work will continue despite adversity.

www.nature.com/articles/d41...

What Baby KJ means for the CRISPR gene editing industry The custom-built gene editing treatment for 6-month-old KJ Muldoon could not have come at a more welcome or jarring time for CRISPR-powered biomedicine.

When a therapeutic approach works, it's on us as a global community to figure out how to get it to ALL patients in need. Great piece by @jasonmast.bsky.social

What Baby KJ means for the CRISPR gene editing industry www.statnews.com/2025/05/26/w... via @statnews.com

Opinion | Joe Biden Has a Chance to Do Something Astounding

www.nytimes.com/2025/05/20/o...

FDA delays PDUFA date for small biotech, raising questions about impact of agency cuts The fate of a small, privately-held biotech’s new drug has been delayed, in what appears to be a sign that broad cuts to the FDA by the Trump administration may ...

FDA delays are bad in general, but for a small biotech they can be existential. Let’s hope this is an edge case.

endpts.com/fda-delays-s...

Verve Therapeutics Announces Positive Initial Data from the Heart-2 Phase 1b Clinical Trial of VERVE-102, an In Vivo Base Editing Medicine Targeting PCSK9 Single infusion of VERVE-102 led to dose-dependent decreases in blood PCSK9 and LDL-C, with mean reduction in LDL-C of 53% and a maximum reduction of 69%...

Congrats to Sek Kathiresan and the entire Verve Therapeutics team.

Big day for the field. Bigger day for patients.

www.globenewswire.com/news-release...

First game of the season with the boy. Never gets old.

Tempero Bio, led by ex-Novartis execs, gets $70M to reduce substance use disorders A small Oakland, CA-based biotech hoping to take on the sprawling landscape of substance use disorders and addiction has reeled in $70 million. The drug development startup, named Tempero Bio ...

Tempero Bio raises $70M for TMP-301, a daily pill in development for alcohol and cocaine use disorders. Phase 2 data expected in 2026.
endpts.com/tempero-bio-...

This is a big day for patients with AATD and a big win for genomic medicine. Congrats to the entire Beam Team @BeamTx!

investors.beamtx.com/news-release...

This 💯

Unvaccinated Child Dies of Measles in Texas Outbreak At least 124 cases have been reported since late January, mostly among children and teenagers who were unvaccinated or whose vaccination status was unknown.

Completely preventable

www.nytimes.com/2025/02/26/u...

BlueRock Therapeutics receives FDA Fast Track Designation for OpCT-001 for the treatment of primary photoreceptor diseases  - BlueRock Therapeutics LP The U.S. Food and Drug Administration (FDA) has granted Fast Track designation for OpCT-001, an investigational induced pluripotent stem cell (iPSC)-derived cell therapy for the treatment of primary p...

Congratulations to Stefan Irion, Seth Ettenberg, and the entire Blue Rock team!

BlueRock Therapeutics receives FDA Fast Track Designation for OpCT-001 for the treatment of primary photoreceptor diseases

www.bluerocktx.com/bluerock-the...

Opinion: Science funding cuts are based on flawed logic. Here’s the truth. Earlier this month, the National Institutes of Health issued supplemental guidance indicating it would immediately impose a 15% cap on indirect costs for research grants, an unprecedented and abrup…

www.sandiegouniontribune.com/2025/02/25/o...

The flu, meanwhile, has been going from mild to wild in recent weeks.

A potent epigenetic editor targeting human PCSK9 for durable reduction of low-density lipoprotein cholesterol levels - Nature Medicine Epigenetic editing of PCSK9 in the liver, designed to induce DNA methylation and silence gene expression, achieved long-lasting reductions in LDL cholesterol levels in both transgenic mice expressing ...

Most genome editing work to date has been DNA based. A new paper out today assessed a single dose of epigenetic editing (thru PCSK9) for marked (~70%) and durable reduction of LDL cholesterol [in non-human primates]
www.nature.com/articles/s41...

This work was led by the BlueSky-less Fred Tremblay. Fred’s scientific rigor and unflappable leadership style truly drove this program. Congratulations mon ami to you and the entire team!
9/9

Our preclinical data strongly supports the promise of epigenetic editing as a potential ‘one-and-done’ approach for the treatment of hypercholesterolemia, and more generally as a therapeutic approach to silencing loci without cutting, nicking, or changing the DNA sequence
8/9

Our human PCSK9-targeting epigenetic editor is fully cross-reactive against cyno PCSK9, & we demonstrated that a single administration led to rapid and sustained, 90% reduction of PCSK9 protein, resulting in a clinically meaningful 70% reduction in LDL-C in non-human primates
7/9

Not only was this methylation-driven silencing durable in a homeostatic liver, but both silencing and methylation were fully maintained during liver regeneration, indicating durability through cell division in vivo!
6/9

We confirmed that mRNA/gRNA encoding our human PCSK9-targeting epigenetic editor packaged into an LNP was able to reduce circulating PCSK9 protein 98% for a year after a SINGLE ADMINISTRATION in a human PCSK9 transgenic mouse!
5/9

We designed an mRNA-based #epigeneticeditor: DNA-binding domain fused to a DNA methyl-transferase domain & a transcriptional repressor domain which, together w/ human PCSK9-targeted gRNA, deeply & specifically methylated & silenced PCSK9 in primary human hepatocytes in vitro
4/9

Low-density lipoproteins cause atherosclerotic cardiovascular disease. 1. Evidence from genetic, epidemiologic, and clinical studies. A consensus statement from the European Atherosclerosis Society Co... AbstractAims. To appraise the clinical and genetic evidence that low-density lipoproteins (LDLs) cause atherosclerotic cardiovascular disease (ASCVD).Metho

One of the first programs we launched sought to develop a potent, transiently expressed epigenetic editor to durably silence #PCSK9, a genetically and pharmacologically validated target that regulates plasma LDL-C, a causal factor for heart disease
3/9

academic.oup.com/eurheartj/ar...

When @chromamedicine launched in November, 2021, we set out to build on the pioneering work from our founders, & develop therapeutics that leverage epigenetics to precisely and durably control gene expression
2/9

x.com/jaffeab/stat...

A potent epigenetic editor targeting human PCSK9 for durable reduction of low-density lipoprotein cholesterol levels - Nature Medicine Epigenetic editing of PCSK9 in the liver, designed to induce DNA methylation and silence gene expression, achieved long-lasting reductions in LDL cholesterol levels in both transgenic mice expressing ...

Our work describing the development of a potent epigenetic editor targeting human #PCSK9 for durable reduction of LDL-C is out in @naturemedicine.bsky.social today!
Cc: @chromamedicine @nchromabio
1/9

www.nature.com/articles/s41...

This work was led by the BlueSky-less Fred Tremblay. Fred’s scientific rigor and unflappable leadership style truly drove this program. Congratulations mon ami to you and the entire team!
9/9

Our preclinical data strongly supports the promise of epigenetic editing as a potential ‘one-and-done’ approach for the treatment of #hypercholesterolemia, and more generally as a therapeutic approach to silencing loci without cutting, nicking, or changing the DNA sequence.
8/9