Cell & Gene Therapy News


FDA Approves First Potentially Curative Gene Therapy for Beta Thalassemia
A leading study site for nearly a decade, CHOP will be part of a network of treatment centers in the country offering the therapy to patients who require regular blood transfusions.
CHOP Researchers Join Global Team to Take on Solid Tumors in Children
Four CHOP researchers are part of a world-class research team that will receive $25 million to study solid tumors in children, funded by Cancer Grand Challenges.
First Approved CAR T-Cell Therapy Shows Continued Benefits Five Years Later
New data on show CAR T-cell therapy leads to durable remission and long-term survival in children and young adults with relapsed or refractory B-ALL.
New Data Show Benefits of CRISPR-edited Gene Therapy for Blood Disorders in a Larger Patient Group with Longer-Term Follow-Up
A group of researchers that includes CHOP presented new data on CRISPR-edited gene therapy for blood disorders at an international meeting.

Penn Medicine and Children’s Hospital of Philadelphia Announce Partnership with Costa Rica for CAR T Cell Therapy
International collaboration aims to ensure more equitable access to transformative personalized cancer therapy.
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.