Expert Insights in Hemophilia Gene Therapy for Bleeding Disorders Awareness Month
In observance of Bleeding Disorders Awareness Month, held annually in March, catch up on some of the latest expert insights in gene therapies for hemophilia, which is among the most common of these disorders.
In recent months, CGTLive has interviewed multiple experts about the various gene therapies currently in development for the treatment of hemophilia A and hemophilia B, which are among the most commonly occurring bleeding disorders.
For Bleeding Disorders Awareness Month, observed annually in the month of March by the patient and clinician communities, the CGTLive team has gathered some of our latest interviews on hemophilia gene therapies below to offer expert insight on the therapeutic progress being made for these rare disorders.
Those featured include:
- Adam Cuker, MD, associate professor of medicine at the University of Pennsylvania
- Steven Pipe, MD, professor of pediatric hematology/oncology at CS Mott Children’s Hospital
- Brent Warner, president of gene therapy at Poseida Therapeutics
- Rigoberto Garcia, MPH, executive director of the Hemophilia Foundation Southern California
Click the respective slides below to watch each expert interview.
Click the image to view more, or click the arrows to see the next slide.
Adam Cuker, MD, on Higher Efficacy of Gene Therapy Over SOC in Hemophilia B
WATCH TIME: 4 minutes
A single infusion treatment of fidanacogeneelaparvovec demonstrated superiority over standard-of-care factor IX therapy in reducing annualized bleeding rates (ABR) in patients with hemophilia B, according to updated data from Pfizer’s BENEGENE-2 phase 3 clinical trial (NCT03861273). Compared with a 6-month lead-in period, patients treated with the therapy had a 78% reduction in ABR for treated bleeds (P = .0001)and a 92% reduction in the annualized infusion rate of exogenous FIX following infusion with fidanacogene elaparvovec (P <.0001).
CGTLive spoke with study investigator Adam Cuker, MD, associate professor of medicine at the University of Pennsylvania, to learn more about BENEGENE-2 and the new superiority data. He also touched on the positive durability data seen in patients treated with fidanacogeneelaparvovec.
