Kaustuva Das
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kaustuvadas.bsky.social
Kaustuva Das
@kaustuvadas.bsky.social
CEO, Thermal + PR for Jennifer Doudna, Nobel laureate and CRISPR pioneer
thermalpr.com
6/ 📕 NEJM paper www.nejm.org/doi/full/10....

📺 The IGI’s announcement innovativegenomics.org/news/first-p... and video about CRISPR on-demand, a first in the history of medicine www.youtube.com/watch?v=H7pj...

📺 CHOP's announcement page with videos www.chop.edu/centers-prog...
Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease | NEJM
Base editors can correct disease-causing genetic variants. After a neonate had received a diagnosis of severe carbamoyl-phosphate synthetase 1 deficiency, a disease with an estimated 50% mortality ...
www.nejm.org
May 16, 2025 at 3:57 AM
5/ This landmark achievement was made possible by @kiranmusunuru.bsky.social at @pennmedicine.bsky.social, @ahrensnicklas.bsky.social at Children's Hospital of Philadelphia, @urnov.bsky.social and @innovativegenomics.bsky.social team, Danaher, and more.
May 16, 2025 at 3:57 AM
4/ @jenniferdoudna.bsky.social, #CRISPR co-inventor and Nobel laureate, said of the milestone, “This was a remarkable team effort. The ability to develop an on-demand CRISPR therapy in such a short time opens up a new era for treating previously untreatable genetic diseases.”
May 16, 2025 at 3:57 AM
3/
The patient, KJ, was born with CPS1 deficiency, a rare and severe metabolic disease affecting 1 in 1.3 million newborns. In just 6 months, scientists and doctors designed a CRISPR treatment that specifically targeted a genetic mutation in KJ’s liver cells.
May 16, 2025 at 3:57 AM
2/
- This was medically vital, developed as a Manhattan Project for genetic medicine.

- It’s an N-of-1 therapy, a proof of concept showing how scientists can unite around a patient in need.

- It affirms the goal: Create scalable on-demand personalized CRISPR therapies accessible to all patients.
May 16, 2025 at 3:57 AM