Sickle Cell Center News

Children’s Hospital of Philadelphia (CHOP) hosted the Cure Sickle Cell Walk & Family Fun Day presented by West Pharmaceutical Services on Saturday, June7. It was a morning of walking, cheering and celebrating sickle cell warriors and their families at Citizens Bank Park. The event, which included a ceremonial walk to honor those with sickle cell disease, along with fun activities for the whole family, raised more than $242,000 for sickle cell disease research and patient care.

The life and legacy of the late Dr. Kwaku Ohene-Frempong was celebrated by family, friends and colleagues at his portrait unveiling ceremony on CHOP’s campus on June 20.

Earlier this month, Children’s Hospital of Philadelphia (CHOP) hosted the Cure Sickle Cell Walk & Family Fun Day presented by West Pharmaceutical Services at Citizens Bank Park raising more than $256,000 (and counting!) to support sickle cell warriors.

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). 

CHOP and Penn researchers have developed a proof-of-concept model for delivering gene editing tools to treat blood disorders directly within the body.

Case: Langston is a 12-year-old with sickle cell disease whom you have followed in your practice since infancy.

CHOP researchers have discovered a new part of the regulatory cascade responsible for silencing fetal hemoglobin in adult cells.

A group of researchers that includes CHOP presented new data on CRISPR-edited gene therapy for blood disorders at an international meeting.

CHOP researchers have identified two transcription factors that are involved in silencing fetal hemoglobin in adult red blood cells.

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.