New work, now online in @CellCellPress! Using a phased, binder-first strategy, we identified a ligandable pocket on CARD9 and discovered a small-molecule inhibitor of CARD9 inflammatory signaling. (1/7)
https://t.co/Iol4elpGSR
The new Plausible Mechanism Pathway will bring the power of personalized therapies — like we saw with Baby KJ — to more patients with rare diseases. Our piece in this week’s New England Journal of Medicine:
https://t.co/1BMeLSfyAl
As #2024 comes to a close, our Research Annual Report is a celebration of the life-changing solutions we've created to give every child the best opportunity for a bright future. View the report online now. https://t.co/smYXohymzi
#YearInReview#ChildrensHealth#CHOPResearch
Sharing this recent publication on identifying therapeutic vulnerabilities of aneuploid cells. I enjoyed developing the small molecule repurposing screen for this project!
https://t.co/dnouvxdxBJ
.@childrensphila received a 2024 Fire Award from @phlbizjournal and PHL Inno for advancing #CellAndGeneTherapy innovation forward. Dr. Sue Furth, Peter Grollman, Dr. Johannes van der Loo, & Katherine Helbig represented CHOP at the award ceremony. https://t.co/bEMyu4AzRi
The Cell & Gene Therapy Collaborative at @ChildrensPhila is celebrating 5 years of ingenuity + innovation! Learn how the unique center is making their resources available to as many faculty as possible: https://t.co/5WPMUy0jfe
BREAKING: Today, we announced the U.S. FDA has expanded the product label for our gene therapy and more people may be able to receive treatment.
Intended for U.S. audiences
RNA is…complicated. If it’s not confusing enough that there are many types of RNA, some RNA molecules also fold into different shapes that help their stability and functions. Not to worry! We break it all down for you in our new fact sheet: https://t.co/f0bpvxV1ov
Decades ago, Dr. Kathy High, with @ChildrensPhila & @PennMedicine colleagues, began an unprecedented journey of basic & clinical research, which paved the way for the @US_FDA approval of a gene therapy to treat #Hemophilia patients. Learn more: https://t.co/h0VZjWZGh6
Our Clinical Vector Core team says hello from #ASGCT2024! Now until May 11, these @ChildrensPhila cell & gene therapy experts are at Booth #613 to answer your questions about affordable, flexible, & high-quality vector production. Learn more: https://t.co/ZA2iaKRaRs
Coming up soon at #ASGCT2024: Hear from Drs. Beverly Davidson (@ChildrensPhila) + Philip Gregory (@Regeneron) during today's George Stamatoyannopoulos Memorial Lecture and from @davidrliu during the Outstanding Achievement Award Presentation! https://t.co/dwsUS1CWm3
An international consortium led by Dr. Stephan Grupp published final results of a key clinical trial on gene therapy CASGEVY for sickle cell disease treatment. The findings, published in @NEJM, led to FDA approval of CASGEVY™ in December 2023.
More: https://t.co/Yd1HogdVfO.
Sharing this recent paper in Nature Methods which describes the creation of a publicly available cellular imaging dataset known as CPJUMP1.
It was a privilege to be part of this collaborative team while working at the Broad Institute! @DrAnneCarpenter
https://t.co/PVJkkBowrT
For readers interested in bispecific antibodies for cancer therapy, this Review in the April issue covers bispecific T-cell and NK-cell engagers, tumour-targeted receptor agonists, checkpoint inhibitors and more https://t.co/pc2gZlszFE https://t.co/qzsydjWLd4 #AACR24