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). 

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

Today, Children’s Hospital of Philadelphia’s Center for Violence Prevention (CVP) marks a major milestone -- 10 years of unified programming and research focused on reducing exposure to and impact of violence on youth and their families.

This major step allows for the creation of clinical trials to test key endpoints that will determine the efficacy of new therapies.

All three genes had variants affecting splicing and resulted in symptoms like developmental delays, intellectual disability, hypotonia, seizures and autism.

An experimental compound might be further developed as an effective treatment approach for affected patients.

Researchers found CAR T-cell therapy may serve as effective alternative for patients with these disorders.

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.

 In an abstract presented at AHA, researchers found Black children are more than four times as likely to experience cardiac arrest and have significantly worse survival outcomes.

The two studies, presented at ASHG 2023, identified base editing and prime editing approaches for treating the rare newborn genetic disease