Freeline’s Gene Therapy for Hemophilia B Is Promising, Phase 1/2 Data Show
Data from the first two patients treated with FLT180a, Freeline’s single-infusion gene therapy for patients with hemophilia B,…
Articles by Patricia Inácio, PhD
FDA Grants Sigilon Therapeutic’s SIG-001 Orphan Drug Status for Hemophilia A
The U.S. Food and Drug Administration (FDA) has granted orphan drug status to Sigilon Therapeutics’ candidate cell therapy,…
Variations in Certain Immune Genes May Help Predict Risk for Developing FVIII Inhibitors in Hemophilia A
Genetic variations in certain immune-related genes, namely the HLA and IL-10 genes, are linked with an increased risk for developing…
Parent’s Sense of Burden Higher When Child Overweight and Has Inhibitors, Study Finds
The presence of inhibitors and excessive body weight increase the sense of disease burden felt by parents of children with…
Two-stage Gene Therapy for Hemophilia A Shows Promising Results in Mice, Researchers Say
Casebia’s two-stage gene therapy approach was successful in delivering the human gene coding for clotting factor VIII (FVIII) in…
Vaccination of Hemophilia Patients Varies in Germany and Should Be Reassessed to Minimize Fear, Survey Suggests
Recommendations for vaccination practices for patients with hemophilia should be reassessed in an effort to minimize fear and emphasize the…
Higher FVIII Blood Levels Seen and Better Maintained with Jivi Than Eloctate, Phase 1 Study in Severe Hemophilia A Finds
Bayer’s Jivi, an FVIII replacement therapy for hemophilia A, has a longer half-life and a slower clearance from…
BioMarin’s Gene Therapy for Severe Hemophilia Yields Sustained Benefit, Data Show
BioMarin Pharmaceutical’s investigative gene therapy, called valoctocogene roxaparvovec, is capable of sustained control of bleeding rate requiring factor VIII…
Web-Based Calculator Helps Physicians to Finetune Dosage of Hemlibra for Hemophilia A Patients
BrightInsight has launched a web-based dosing calculator to help physicians select the correct loading and maintenance dose of the…
Candidate Cell Therapy Controls Bleeding in Mouse Model of Hemophilia A
Sigilon Therapeutics’ candidate cell therapy for hemophilia A, called SIG-001, delivers sustained production of factor VIII for over…