Words can’t express what it meant to work on such a historic case in medicine. It was truly a call to action across institutions to save a life. I think I speak for everyone involved when I say our greatest hope is that medical opportunity becomes accessible to all who need it.
A CRISPR therapy produced in record time benefits an infant with an often deadly hereditary disease, with help from @UCBerkeley https://t.co/JB3iWOwCLx @ChildrensPhila
“It Was Not a Cure”: Musunuru Cautions ASGCT on Baby KJ Promise
@kiranmusunuru, a 2026 ASGCT Catalyst Award winner, reflected on Baby KJ’s legacy. He outlined the FDA conversations required to streamline future #raredisease trials
#ASGCT26#geneediting
https://t.co/tzrKtSTO56
It was incredibly energizing to finally meet collaborators, mentors and longtime role models in person after years of reading their papers. I appreciate all those who took the time to visit our poster session to discuss off-targets and personalized medicine. #asgct26
First released one year ago, Evo 2 predicts variant effects & generates functional DNA across all domains of life, from bacteria to humans, from a single set of weights, with no fine-tuning required.
Now published in @NatureComms! See my thread from the preprint for the full breakdown. Stay tuned for more work in this direction! 📄 https://t.co/o5JoWzably
@lucapinello@NatureComms Holy cow!! -- "20.3%-22.9% self-editing frequency in ABE8e-SpRY presort samples" .. I wonder how common this actually is. Not many people analyze the gRNA sequence in their down stream analysis either
@honglue1 I really enjoyed this paper! It mirrot many trends I've observed in off-target screening throughout the years. It would be interesting to translate these findings into a more applied framework of off-target detection
Breaking News: A baby with a rare disorder made medical history by receiving the first custom gene-editing treatment. The technique used has the potential to help people with thousands of other uncommon genetic diseases. https://t.co/6RB5ttiKmX
In a medical milestone, a customized base editor was developed, characterized in human and mouse cells, tested in mice, studied for safety in non-human primates, cleared by @US_FDA for clinical trial use, manufactured as a complex with an LNP, and dosed into a baby with a severe, rapidly progressing genetic disease... all in an astounding 7 months. Best of all, the infant patient shows apparent benefit. Congratulations to @kiranmusunuru, Rebecca Ahrens-Nicklas, and other team members for this heroic and inspiring effort, which has implications for the hundreds of millions of patients that suffer from thousands of genetic diseases.
https://t.co/wsgvvRYPVM
Presented at #ASGCT2025:
A lipid nanoparticle–delivered base-editing therapy was custom designed for an infant with a ureacycle disorder. The affected infant was treated at approximately 7 and 8 months of age.
Read the Brief Report: https://t.co/B695kT9DRn
Editorial: Progress in the Development of N-of-1 Therapy https://t.co/PzvWMNfyVP
Science behind the Study: Personalized Gene Editing to Treat an Inborn Error of Metabolism https://t.co/mJSKHr5HIR
@ASGCTherapy
@honglue1@doudna_lab@Noor_AlSayyad@kevin_wasko Amazing! I wonder if this explains why we often see that high performing gRNAs also have high specifity. Possibly cutting efficiency is partially due to less time binding to off-target sites ?
@dr_alphalyrae So refreshing to hear!! I chose academia in the Bay Area to escape that mentality, but I was naive. Even academia is how "loud can we speak" instead of the integrity of our words. Biotechs are frequently funders instead of NIH and scientist using grandiloquent rhetoric.
@JohnDoench @CellGenomics Cloning a suite for Cas constructs in several days! …. But grad students around the world will spend less time doing tedious/time consuming tasks? How awful would that be