Hemophilia and Bleeding Disorders Program News

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.

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.

In a study published in the journal Molecular Therapy Methods and Clinical Development, researchers at Children’s Hospital of Philadelphia (CHOP), in collaboration with researchers at the School of Veterinary Medicine at the University of Pennsylvania, found that adeno-associated viral (AAV) gene therapy has the potential to be a life-changing treatment for companion dogs –pets – with severe hemophilia, an inherited bleeding disorder caused by deficiency of clotting factor.

Results from multicenter study are first to show improved production of coagulation factor VIII over prolonged period, leading to reduction or complete elimination of bleeding events.

CHOP researchers were interviewed as a part of a comprehensive article about cell and gene therapy in Philadelphia Magazine.

CHOP researchers have identified BAFF as a key target that may be responsible for treatment failure in about 30% of patients with hemophilia A.

CHOP researchers identified an in vivo role for activated protein C (APC), which could potentially be exploited for future hemophilia A treatments.

A laboratory study may set the stage for a new preventive treatment for hemophilia patients who now develop antibodies to the standard treatment and then require a bypass therapy.

CHOP breakthrough helps scientists edge closer to defeating a longtime enemy of human health: hemophilia, the inability to form blood clots.

A team of gene therapy researchers has reported on a highly active gene variant that achieved sustained factor levels in 10 adults, with no adverse effects.