Division of Hematology News

CHOP announced today that it has been awarded a five-year up to $38.9 million award from the Advanced Research Projects Agency for Health (ARPA-H) THRIVE program to support the advancement of personalized gene therapy.

CHOP is proud to announce the selection of Stella T. Chou, MD, as its new Chief of the Division of Hematology. Dr. Chou served as interim chief since August 2025 and will officially assume the role July 1, leading one of the nation’s largest and highly ranked pediatric hematology programs.

Earlier this month, Children’s Hospital of Philadelphia (CHOP) hosted the Cure Sickle Cell Walk & Family Fun Day, presented by West Pharmaceutical Services, a morning where over 1,000 participants walked, cheered and celebrated patients with sickle cell disease at Citizens Bank Park.

Children’s Hospital of Philadelphia (CHOP) announced today that the National Academy of Medicine (NAM) named Lindsey A. George, M.D., Director of Clinical In Vivo Gene Therapy, and an attending physician in the Division of Hematology at CHOP, as a member of its 2026 class of Emerging Leaders in Health and Medicine (ELHM) scholars.

CHOP researchers reported today in the New England Journal of Medicine about a rare but noteworthy outcome in a child treated with an adeno-associated virus (AAV) gene therapy for severe mucopolysaccharidosis type I (MPS I). MPS I, a lysosomal storage disorder also known as Hurler Syndrome, is characterized by an abnormal build-up of various toxic materials, called glycosaminoglycans (GAGs) in the body's cells.

Researchers at Children’s Hospital of Philadelphia (CHOP) are shedding light on how two genetic factors – an extra copy of chromosome 21 (trisomy 21) and mutations in the GATA1 gene – interact to derail early blood cell development.

Researchers at Children’s Hospital of Philadelphia (CHOP) sought to better understand how the body regulates activated FVIII function, especially in interactions with other clotting factors, to inform associated pathology and therapeutic drug design.

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.

Researchers at Children’s Hospital of Philadelphia (CHOP) found compelling preclinical evidence that a molecule called tumor necrosis factor alpha (TNFα), typically known for promoting inflammation, also plays a helpful role in recovery from anemia that is caused by ongoing immune disturbances.

Researchers at Children’s Hospital of Philadelphia (CHOP) announced the results of the longest follow-up study to date for individuals receiving the gene therapy fidanacogene elaparvovec to treat hemophilia B, a rare, X-linked disorder resulting from a deficiency in coagulation factor IX (FIX) activity.