CHOP Researchers Discover Deep Structural Biology Connections that Help Improve CAR Therapy

Identifying “backbones” that link complexes can help maximize CAR therapy across different variants and tumor types.

At the Forefront of Breakthroughs: Cell and Gene Therapy Lab Sets Course for Expansion

Currently nestled in the Department of Pathology on the fifth floor of the Main Hospital at Children's Hospital of Philadelphia, the Cell and Gene Therapy Laboratory (CGTL) is poised to emerge in big way throughout 2024 and beyond as they expand their facilities to keep up with the pace of discovery in cell and gene therapy.

FDA Approves Two Gene Therapies for Sickle Cell Disease

The Food and Drug Administration (FDA) has approved CASGEVY™ (exagamglogene autotemcel) and LYFGENIA™ (lovotibeglogene autotemcel), the first two gene therapies for the treatment of sickle cell disease in patients 12 years and older with recurrent vaso-occlusive crises (VOCs).

Children’s Hospital of Philadelphia Performs First in U.S. Gene Therapy Procedure to Treat Genetic Hearing Loss

Children’s Hospital of Philadelphia is proud to announce the initial results of an experimental gene therapy treatment of a patient with hereditary hearing loss in the United States. Findings to date indicate that the treatment was successful.

Children’s Hospital of Philadelphia Receives Multi-Million Dollar Gift from Holveck Family to Support Groundbreaking Osteosarcoma Research

Children’s Hospital of Philadelphia (CHOP) has received a $6.4 million gift from the family of Connor Boyle, a Central Bucks East High School graduate who died at age 18 from osteosarcoma. This three-year gift, named The Connor Initiative: Precision Therapeutics for Osteosarcoma & Rare Cancers, will support cutting-edge research in osteosarcoma and other rare cancers.

Spotlight on Current BPD-Focused Research Studies

It’s an exciting time for research in the bronchopulmonary dysplasia (BPD) population here at Children’s Hospital of Philadelphia (CHOP). 

CHOP Researchers Successfully Employ New Method for Treating Pediatric Patients with Common Heart Valve Disease

This new method, typically used in adult patients, may spare patients from having to undergo open heart surgery.

Relief from Chronic Pancreatitis Pain: A Case Study

This case study describes a 10-year-old boy with acute, then chronic, pancreatitis and what CHOP did to treat his condition and improve his quality of life. 

Lab Results with a Twist: Child with Hypoglycemia, High Insulin, and Low C-Peptide

When Spinal Surgery Can Improve Mobility for Children with Spastic Diplegic Cerebral Palsy

A child’s ability to walk improved because of a partnership between CHOP’s Advanced Tone Management Clinic and Neurosurgery.