Cell & Gene Therapy News
CHOP Researchers Develop Allogeneic CART for Relapsed or Refractory T-ALL Using Base Editing
CHOP researchers have tested and developed an “off-the-shelf” chimeric antigen receptor T-cell (CART) using base editing.
Children’s Hospital of Philadelphia Awards Gold Medal to Dr. Katherine High, Gene Therapy Pioneer
Prestigious award recognizes Dr. High’s contribution to improving children’s health through her gene therapy research, which began during her time at CHOP.
First Child to Receive Revolutionary CAR T Therapy Celebrates 10 Years Cancer Free
Emily Whitehead, now 17, received life-saving treatment at CHOP, transforming the field of cancer immunotherapy.
Emily Whitehead, First Pediatric Patient to Receive CAR T-Cell Therapy, Celebrates Cure 10 Years Later
Whitehead received life-saving treatment at CHOP, under the care of Dr. Stephan Grupp, transforming the field of cancer immunotherapy.
CHOP Brings Together Experts to Discuss Precision Medicine in Pediatric Cancer Care
More than 250 experts gathered in Philadelphia and virtually to discuss the latest scientific advancements and breakthroughs in pediatric precision medicine oncology.
CHOP-led Study Shows Novel Gene Therapy for Hemophilia A Leads to Sustained Expression of Clotting Factor and Reduced Bleeding Events
Results from multicenter study are first to show improved production of coagulation factor VIII over prolonged period, leading to reduction or complete elimination of bleeding events.
CHOP Researchers Develop a New Class of CAR-T Cells that Target Previously Untargetable Cancer Drivers
CHOP researchers have developed a novel cancer therapy that targets proteins inside cancer cells that are essential for tumor growth and survival but have been historically impossible to reach.
Positive Preliminary Data on CRISPR Treatment for Blood Diseases
A collaborative team of researchers, including Dr. Stephan Grupp, recently presented preliminary data showing that a CRISPR-based gene-editing therapy for inherited blood disorders is safe and effective.
Children’s Hospital of Philadelphia Researchers Develop “Dimmer Switch” to Help Control Gene Therapy
CHOP researchers have developed a “dimmer switch” system based on alternative RNA splicing that can control levels of proteins expressed from gene therapy vectors.
CHOP Researchers Develop Humanized CAR T-Cell Therapy that Shows Potential for Patients with Relapsed B-ALL
The treatment modifies a domain in the CAR that targets CD19, making it look more human and therefore less likely to be rejected by patients.