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 Kiran Musunuru , Sek Kathiresan MD, Andrew Bellinger, Nicole M. Gaudelli, & other scientists at Verve Therapeutics and Beam Therapeutics on these

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 Parker Moss Richard Scott Nicola Blackwood Tim Yu

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

Our first session ‘After Mila’ with Mila's Miracle Fyodor Urnov Jason Mast #STATBreakthrough

Thank you Natasha Loder 🦋🐝 and The 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-health/…

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/s4158… I break the key findings down panel by panel in this thread: x.com/cureffi/status…

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, AnnemiekeAartsma-Rus, @roon_mom, and our colleagues from the N1 Collaborative hindawi.com/journals/humu/…

This Thursday April 25 from 4:00-6:30 pm at the Broad Institute Julia Vitarello (Mila's mom Mila's Miracle), Casey McPherson (Rose's dad ToCureARose), Termeer Foundation, Cookies4Cures, Broad Institute, and others will host "Rock n' Rare", a special event of inspirational stories

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

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

Thanks to co-authors @roon_mom Marlen Lauffer Rob Collin ype elgersma The paper describes how Tim Yu & Mila's Miracle 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 N1 Collaborative)

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

David Cheerie and I will present at the first N1 Collaborative annual meeting tomorrow! We will discuss the newly established N1C VARIANT (Variant Assessments towards Eligibility for Antisense Oligonucleotide Treatment) Guidelines. medrxiv.org/content/10.110…

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

Privileged to be a physician-scientist in a time when efforts like these spanning genetics, engineering, & medicine come to fruition. Hats off to Kiran Musunuru, Rebecca Ahrens-Niklas ,Fyodor Urnov & more for this milestone in interventional genetics. nejm.org/doi/full/10.10…

New paper out. Thanks to IRDiRC N=1 taskforce members for a very positive, proactive and pleasant collaboration N1 Collaborative n-Lorem Foundation Tim Yu and others! bsky.app/profile/oligog…

Gene editing pioneer David R. Liu 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