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Aron Jaffe

@jaffeab.bsky.social
82 followers 17 following 66 posts

SVP @ curie.bio; Former SVP Head of Research @chromamedicine; EIR @ThirdRockV; Reg Med, uBiome group leader/Resp DA co-lead @NovartisScience. Opinions my own.

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Aron Jaffe @jaffeab.bsky.social · 27/11/2025
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
statnews.com
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.
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Aron Jaffe @jaffeab.bsky.social · 23/09/2025
www.statnews.com/2025/02/03/v...
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.
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Aron Jaffe @jaffeab.bsky.social · 11/09/2025
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...
laskerfoundation.org
Triple-drug therapy for cystic fibrosis - Lasker Foundation
Jesús (Tito) González, Paul A. Negulescu, Michael J. Welsh
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Aron Jaffe @jaffeab.bsky.social · 08/09/2025
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
cell.com
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...
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Aron Jaffe @jaffeab.bsky.social · 07/09/2025
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
statnews.com
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.
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Aron Jaffe @jaffeab.bsky.social · 04/07/2025
www.nature.com/articles/d41...
nature.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.
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Aron Jaffe @jaffeab.bsky.social · 27/05/2025
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
statnews.com
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.
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Aron Jaffe @jaffeab.bsky.social · 20/05/2025
www.nytimes.com/2025/05/20/o...
nytimes.com
Opinion | Joe Biden Has a Chance to Do Something Astounding
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Aron Jaffe @jaffeab.bsky.social · 29/04/2025
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...
endpts.com
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 ...
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Aron Jaffe @jaffeab.bsky.social · 15/04/2025
Congrats to Sek Kathiresan and the entire Verve Therapeutics team. Big day for the field. Bigger day for patients. www.globenewswire.com/news-release...
globenewswire.com
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%...
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Aron Jaffe @jaffeab.bsky.social · 06/04/2025
First game of the season with the boy. Never gets old.
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Reposted by Aron Jaffe
Endpoints News @endpts.com · 24/03/2025
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-...
endpts.com
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 ...
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Aron Jaffe @jaffeab.bsky.social · 10/03/2025
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...
investors.beamtx.com
Beam Therapeutics Announces Positive Initial Data for BEAM-302 in the Phase 1/2 Trial in Alpha-1 Antitrypsin Deficiency (AATD), Demonstrating First Ever Clinical Genetic Correction of a Disease-causin...
Single Dose of BEAM-302 Led to Durable, Dose-dependent Increases in Total and Functional Alpha-1 Antitrypsin (AAT), Production of Corrected M-AAT, and Decreases in Mutant Z-AAT in Circulation Across I...
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Aron Jaffe @jaffeab.bsky.social · 06/03/2025
This 💯
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Aron Jaffe @jaffeab.bsky.social · 27/02/2025
Completely preventable www.nytimes.com/2025/02/26/u...
nytimes.com
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.
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Aron Jaffe @jaffeab.bsky.social · 27/02/2025
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...
bluerocktx.com
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...
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Aron Jaffe @jaffeab.bsky.social · 26/02/2025
www.sandiegouniontribune.com/2025/02/25/o...
sandiegouniontribune.com
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…
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Reposted by Aron Jaffe
Lisa Jarvis @lisamjarvis.bsky.social · 16/02/2025
The flu, meanwhile, has been going from mild to wild in recent weeks.
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Reposted by Aron Jaffe
Eric Topol @erictopol.bsky.social · 10/02/2025
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...
nature.com
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 ...
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Aron Jaffe @jaffeab.bsky.social · 10/02/2025
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
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Aron Jaffe @jaffeab.bsky.social · 10/02/2025
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
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Aron Jaffe @jaffeab.bsky.social · 10/02/2025
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
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Aron Jaffe @jaffeab.bsky.social · 10/02/2025
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
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Aron Jaffe @jaffeab.bsky.social · 10/02/2025
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
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Aron Jaffe @jaffeab.bsky.social · 10/02/2025
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
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Aron Jaffe @jaffeab.bsky.social · 10/02/2025
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...
academic.oup.com
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
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Aron Jaffe @jaffeab.bsky.social · 10/02/2025
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...
x.com
x.com
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Aron Jaffe @jaffeab.bsky.social · 10/02/2025
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...
nature.com
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 ...
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Aron Jaffe @jaffeab.bsky.social · 10/02/2025
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
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Aron Jaffe @jaffeab.bsky.social · 10/02/2025
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
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Aron Jaffe @jaffeab.bsky.social · 10/02/2025
Our human PCSK9-targeting epigenetic editor is fully cross-reactive against cyno PCSK9, and 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 7/9
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Aron Jaffe @jaffeab.bsky.social · 10/02/2025
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
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Aron Jaffe @jaffeab.bsky.social · 10/02/2025
We confirmed that mRNA/gRNA encoding our human PCSK9-targeting epigenetic editor packaged into an LNP was able to reduce circulating PCSK9 protein by 98% for a year after a SINGLE ADMINISTRATION in a human PCSK9 transgenic mouse! 5/9
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Aron Jaffe @jaffeab.bsky.social · 10/02/2025
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
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Aron Jaffe @jaffeab.bsky.social · 10/02/2025
One of the first programs we launched sought to develop a potent, transiently expressed epigenetic editor to durably silence #PCSK9, a genetically and pharmacalogically validated target that regulates plasma LDL-C, a causal factor for heart disease. 3/9 academic.oup.com/eurheartj/ar...
academic.oup.com
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
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Aron Jaffe @jaffeab.bsky.social · 10/02/2025
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...
x.com
x.com
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Aron Jaffe @jaffeab.bsky.social · 10/02/2025
Congrats @rjdlab.bsky.social . And good luck with your talk today!
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Aron Jaffe @jaffeab.bsky.social · 01/02/2025
As usual, @lisamjarvis.bsky.social absolutely nailing it.
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Aron Jaffe @jaffeab.bsky.social · 30/01/2025
iPSC-derived cardiomyocytes + stromal cells = engineered heart muscle Science is truly amazing www.nature.com/articles/s41...
nature.com
Engineered heart muscle allografts for heart repair in primates and humans - Nature
Epicardial engineered heart muscle allografts from induced pluripotent stem cell-derived cardiomyocytes can safely and effectively remuscularize chronically failing hearts in rhesus macaques, leading ...
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Aron Jaffe @jaffeab.bsky.social · 29/01/2025
💯👇🏼 ‘These founders have this idea, and for $10 million we can literally go from what those founders had to a development candidate…. It’s extraordinarily exciting’ www.statnews.com/2025/01/29/c...
statnews.com
Biotech accelerator Curie.Bio raises $340 million for a new seed fund
Curie.Bio, the biotech accelerator and investment firm, has raised another $340 million to invest in startups.
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Aron Jaffe @jaffeab.bsky.social · 14/01/2025
Welcome to curie.bio Ben! Excited to be working together! endpts.com/after-gsk-de...
endpts.com
Exclusive: Day after GSK buyout, IDRx chair takes new investing role at Curie.Bio
SAN FRANCISCO — Ben Auspitz, a veteran biotech investor and repeat entrepreneur, is reuniting with longtime collaborator Alexis Borisy and his
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Aron Jaffe @jaffeab.bsky.social · 13/01/2025
Congrats to Seth Ettenberg, Stefan Irion, and the entire Blue Rock team. #Parkinsons patients are waiting. www.bayer.com/media/en-us/...
bayer.com
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Aron Jaffe @jaffeab.bsky.social · 13/01/2025
BIG SPLASH to start #JPMWeek! #JPM25 www.businesswire.com/news/home/20...
businesswire.com
Johnson & Johnson Strengthens Neuroscience Leadership with Acquisition of Intra-Cellular Therapies, Inc.
Johnson & Johnson (NYSE: JNJ) and Intra-Cellular Therapies, Inc. (Nasdaq: ITCI) announced today that they have entered into a definitive agreement
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Aron Jaffe @jaffeab.bsky.social · 13/01/2025
Nice start to the week. Congrats to the IDRx team. #JPM25 www.businesswire.com/news/home/20...
businesswire.com
GSK Enters Agreement to Acquire IDRx, Inc.
GSK plc (LSE/NYSE: GSK) and IDRx, Inc. (IDRx) today announced that they have entered into an agreement under which GSK will acquire IDRx, a Boston-bas
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Aron Jaffe @jaffeab.bsky.social · 12/01/2025
Safe travels to friends and colleagues heading to #JPM25
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Aron Jaffe @jaffeab.bsky.social · 07/01/2025
Big day for patients with #T1D ir.sana.com/news-release...
ir.sana.com
Sana Biotechnology Announces Positive Clinical Results from Type 1 Diabetes Study of Islet Cell Transplantation Without Immunosuppression | Sana Biotechnology, Inc
First-in-Human Study Provides Evidence that Sana’s Hypoimmune (HIP) Technology Enables Transplanted Islet Cells to Avoid Immune Rejection and Produce Insulin Without Immunosuppression Results Demonstr...
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Aron Jaffe @jaffeab.bsky.social · 03/01/2025
That feeling when you hear your wife singing 'Roy Kent! Roy Kent! He's here! He's there! He's every f*ckin' where!' and realize she's binge watching Ted Lasso cc: @brendanhunting.bsky.social
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Aron Jaffe @jaffeab.bsky.social · 29/12/2024
www.nature.com/articles/d41...
nature.com
Stem cells head to the clinic: treatments for cancer, diabetes and Parkinson’s disease could soon be here
More than 100 clinical trials put stem cells for regenerative medicine to the test. It’s a turning point for a field beset with ethical and political controversy.
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Aron Jaffe @jaffeab.bsky.social · 17/12/2024
Chicago’s biotech scene is growing up. What’s fueling it? - endpts.com/chicagos-bio...
endpts.com
Chicago’s biotech scene is growing up. What’s fueling it?
Over the years, a handful of cities have tried catching up to the flourishing biotech hubs in Boston or the San Francisco Bay Area. Chicago, the third-largest city in the US, is putting together its b...
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Aron Jaffe @jaffeab.bsky.social · 15/12/2024
The number also changes (dramatically) depending on whether you include #microproteins Cc: @thomasmartinez.bsky.social
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