Aron Jaffe

Our work describing the development of a potent epigenetic editor targeting human #PCSK9 for durable reduction of LDL-C is out in @NatureMedicine today! Cc: @chromamedicine @nchromabio 1/9 nature.com/articles/s4159…

Very good news from @REGENXBIO and for Duchenne patients Presentation tomorrow @ASGCTherapy #ASGCT2026 Regenxbio says Duchenne gene therapy succeeded in clinical trial, paving way for FDA submission statnews.com/2026/05/14/reg… via @statnews

Congrats @bloodgenes !

Congratulations to the now Twitterless/Xless Ralph DeBerardinis on being elected to @theNASciences!!!

Congrats @IOB_Roska and all the awardees!

Congrats to all involved in getting this important medicine to patients - teams @Regeneron & DecibelTx, Joe Burns, Adam Palermo, Jonathan Whitton, @jkeilty, Laurence Reid, many others… & big props to @Regeneron for providing Otarmeni for free in the US investor.regeneron.com/news-releases/…

Couldn’t agree more! As my wife and I tell our two kids - actions have consequences!

Best of luck to all! #bostonmarathon

Congratulations to Jean Bennett, Katherine A. High and Albert Maguire, winners of the 2026 Breakthrough Prize in Life Sciences, for developing a therapy for inherited retinal degeneration that became the first FDA-approved gene therapy for a genetic disease. breakthroughprize.org/News/98 @Penn @ChildrensPhila @RockefellerUniv

That Phase 3 overall survival data is in second line #PancreaticCancer which makes the numbers even more astounding. I cannot recall any other example in pancreatic cancer when OS in a Phase 3 trial basically doubled over existing standard of care. statnews.com/2026/04/13/rev…

👏👏👏

A decade ago, these drugs tore apart the FDA. Today, they might be some patients’ best hope  Another wonderful piece from @Jasonmmast statnews.com/2026/04/08/duc… via @statnews

Great piece from @ercgrn I <3 genetics Huntington’s Disease and The Triad of Therapeutic Conviction lifescivc.com/2026/03/huntin…

Resharing this piece I wrote last year on human genetics and drug discovery. India’s genetic diversity is an underexplored asset, one that can fundamentally reshape how we discover new medicines. This is exactly the direction we’re building towards 🇮🇳 worksinprogress.co/issue/natures-…

India’s Boundless Biotech Potential That potential starts with human genetics! ⁦⁦@doctorveera⁩ wsj.com/opinion/indias…

Congrats @davidjglassMD!

Go @philandrew_IL9 !!!

Tirzepatide will do ~$45B in global sales this year (2026) Global iPhone sales will likely be about ~$230B this year, for perspective Yes, peptides are popular. Especially the rigorously tested, FDA approved kind 😎

I grew up hearing stories about my father @rcjaffe’s time in the O’Malley lab. Ultimately a persons legacy isn’t just the work they produce - it’s the people they influence and inspire. RIP Bert pnas.org/doi/10.1073/pn…

My friend @adamfeuerstein delivers some great reporting and smart commentary on the FDA's direction that $QURE conduct a new randomized trial for its Huntington's drug. A senior FDA official and the company disagree on the basic question of whether the FDA says patients on placebo need a burr drilled in their skulls. That kind of thing should be clear to everyone. It's easy to point to the differences between the FDA's Vinay Prasad and his predecessor, Peter Marks, as being opposites. Prasad is stringent, issuing lots of rejections. Marks had a yogi's regulatory flexibility. I agree with Adam: both of those are extreme views. What most people want is an FDA that is balanced somewhere in the middle. I also suspect that a lot of the controversies we are seeing from the current FDA are the result of refusing to learn lessons from past decisions or follow the basic steps of communicating with companies. Adam's column is very much worth your time. statnews.com/2026/03/05/vin…