1/3 Wonderful to finally be able to share our paper on the development and deployment of milasen, now online at nejm.org/doi/full/10.1056/NE…. It’s accompanied by thoughtful commentary from Janet Woodcock and Peter Marks of the FDA (nejm.org/doi/full/10.1056/NE…).

Gene editing pioneer @davidrliu discusses baby K.J. Muldoon, the first person treated with a customized gene editing therapy, what made K.J.'s treatment possible, and how on-demand treatments for rare genetic diseases could one day become routine. broad.io/DLQA

New paper out. Thanks to @irdirc N=1 taskforce members for a very positive, proactive and pleasant collaboration @N1Collaborative @n_lorem @timyu and others! bsky.app/profile/oligogirl.b…

Privileged to be a physician-scientist in a time when efforts like these spanning genetics, engineering, & medicine come to fruition. Hats off to @kiranmusunuru, Rebecca Ahrens-Niklas ,@UrnovFyodor & more for this milestone in interventional genetics. nejm.org/doi/full/10.1056/NE…

Save the date - you're invited to attend the 21st Annual Meeting of the Oligonucleotide Therapeutics Society. This event will be held on October 19-22, 2025, at the Budapest Congress Center in Budapest, Hungary. oligotherapeutics.org/ #ots25 #oligomeeting #OTS #careers

David Cheerie and I will present at the first @N1Collaborative annual meeting tomorrow! We will discuss the newly established N1C VARIANT (Variant Assessments towards Eligibility for Antisense Oligonucleotide Treatment) Guidelines. medrxiv.org/content/10.1101/…

Announcing Zebronkysen: Amelia & Makenzie receive personalized ASO drug. This milestone would not be possible without the incredible support of the ForeBatten community and the passionate development team. More information on this exciting news is on forebatten.org/

Thanks to co-authors @roon_mom @LaufferMarlen @collinrwj @ypeelgersma The paper describes how @timyu & @stopbatten inspired us to start DCRT and lessons we have learned in our first 4 years (spoiler: most important lesson: it is important to collaborate & share @N1Collaborative)

Join us for a webinar on July 25th at 8am PDT titled "Nucleic Acid Therapeutics: Successes, Milestones, and Upcoming Innovation." Register now: oligotherapeutics.org/nuclei… #nucleicacidtherapeutics #biotechnology #pharmaceuticalresearch #otswebinar #ots #nucleicacid

A remarkable milestone yet buried the lede: He wants to be a geneticist. nytimes.com/2024/05/06/healt…

This Thursday April 25 from 4:00-6:30 pm at the Broad Institute Julia Vitarello (Mila's mom @stopbatten), Casey McPherson (Rose's dad @ToCureARose), @The_Termeer_Fdn, @Cookies4Cures, @broadinstitute, and others will host "Rock n' Rare", a special event of inspirational stories and music celebrating family and friends of rare disease patients, scientists, doctors, and the interested public. Stories and appearances by Julia, Casey, @timyu, @SammyBasso, Dana Perella, Cole Quinn, Weston McPherson, and myself. Music by Casey McPherson and AlphaRev. Registration: betterunite.com/rocknrare

Very happy that our recommendations for the identification of suitable variants for individualized ASO treatments are now published! Big thanks go to our PhD candidate Bianca Zardetto, @oligogirl, @roon_mom, and our colleagues from the @N1Collaborative hindawi.com/journals/humu/20…

Published today, our reasons for optimism that success rates in drug discovery can be improved by picking the right targets using human genetics: nature.com/articles/s41586-0… I break the key findings down panel by panel in this thread:

Thank you @natashaloder and The @Telegraph for sharing Mila's story and the critical role the UK is playing in proving a model for individualized medicines at scale. telegraph.co.uk/global-healt…

Our first session ‘After Mila’ with @stopbatten @UrnovFyodor @Jasonmmast #STATBreakthrough

We have many excellent resources on the website from the OTS President's perspective & educational videos, to in-depth articles. View our resources on the menu under "Science News." oligotherapeutics.org/ #ots #oligonucleotide #biotechnology #science #sciencenews

Today is a very exciting day. The UK government announced in the Autumn Statement by Chancellor Jeremy Hunt, their support for the individualized medicines pilot MMF initiated over a year ago with support from Daniel O'Connor @parkermoss @Rich_Genomics @nicolablackwood @timyu

The first in vivo base editing clinical trial outcomes are now reported, following last year’s positive ex vivo base editing clinical outcomes! Congratulations to @kiranmusunuru , @skathire, @ambellinger, @NicoleGaudelli, & other scientists at @VerveTx and @BeamTx on these positive data.

I’m in awe of Sonia Vallabh and Julia Vitarello, two warriors whose personal journeys and relentless commitment to connecting cutting-edge science with patients are helping to make treatments for rare genetic disease a reality.