Fitusiran Continues to Prevent Bleeds in Hemophilia A or B, Trial Shows
As a long-term preventive treatment, fitusiran leads to sustained reductions in bleed frequency in people with moderate-to-severe hemophilia…
Articles by Joana Carvalho, PhD
Efficacy of Gene Therapy SB-525 Now at 14 Months in Alta Hemophilia A Trial
Up to 14 months after a one-time infusion of SB-525, an investigational gene therapy, durable increases in the…
Catalyst Completes Phase 1 Trial of MarzAA, Is on Track to Start Phase 3
Despite the COVID-19 pandemic, Catalyst Biosciences has successfully completed a Phase 1 trial of marzeptacog alfa activated (MarzAA),…
Infection by New Coronavirus Can Induce AHA, Case Report Says
Infection by the severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2), the coronavirus causing COVID-19, can induce the…
FDA Agrees to Phase 1 Trial of ET3, Gene Therapy for Hemophilia A
The U.S. Food and Drug Administration (FDA) has approved Expression Therapeutics‘ request to open a Phase 1 trial of…
Long-term Rebinyn Can Safely Prevent Bleeds in Children With Severe Hemophilia B, Trial Shows
Long-term treatment with Rebinyn (nonacog beta pegol, or N9-GP) is safe and effective at preventing and treating…
Hemophilia A Gene Therapy, AMT-180, Continues to Show Activity, Safety in Animals
A single dose of the experimental gene therapy AMT-180 continues to be well-tolerated and to enhance blood clotting activity independent…
BioMarin’s Gene Therapy for Hemophilia A Remains on Track for US Approval
Despite the COVID-19Â pandemic, BioMarin Pharmaceutical‘s application to the U.S. Food and Drug Administration (FDA) seeking approval of…
Risk of Bleeds in Hemophilia A Influenced by Treatment History, Other Factors, Study Suggests
The risk of spontaneous bleeding episodes in patients with severe hemophilia A receiving preventive treatment changes over time…
Sangamo Concludes Collaboration Agreement with Biogen
After handing over the development of the gene therapy candidate SB-525 to Pfizer, Sangamo Therapeutics has…