Potential Gene Therapy GENV-HEM Earns Orphan Drug Status
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to GENV-HEM, GeneVentiv Therapeutics’ investigational gene…
Articles by Marta Figueiredo, PhD
Research Explores Umbilical Stem Cells as Treatment
Using a baby’s own umbilical cells as vehicles to deliver factor VIII (FVIII) — the missing or defective clotting protein…
Bleeding Disorders Conference Returns This Week, Online for 2021
To ensure the health and safety of participants given the ongoing COVID-19 pandemic, this years’ Bleeding Disorders Conference…
New Guidelines Issued to Self-administer Hemlibra
The National Hemophilia Foundation (NHF), through its Nursing Working Group (NWG), has released new guidelines to help hemophilia…
Gene Therapy SPK-8011 Prevents Bleeds Up to 4 Years
A single dose of Spark Therapeutics’ investigational gene therapy SPK-8011 safely and effectively prevented bleeding episodes and the…
Hemlibra’s Benefits for Hem A With Inhibitors Upheld in STASEY Trial
Two years of preventive treatment with Genentech’s Hemlibra (emicizumab) safely and effectively lowered the number of bleeds in…
Phase 1/2 Trial Set to Test ASC618, Gene Therapy for Hemophilia A
The U.S. Food and Drug Administration (FDA) has approved ASC Therapeutics’ request to open a clinical trial in the…
Extended Half-life Products Linked to Fewer Infusions, Low Bleed Rates
For boys and men with moderate to severe hemophilia, switching to extended half-life (EHL) products is linked to…
BioMarin Resubmits Roctavian Gene Therapy for EU Approval
BioMarin Pharmaceutical has resubmitted a regulatory application seeking approval in Europe for its experimental gene therapy Roctavian (valoctocogene roxaparvovec)…
Hemophilia B Gene Therapy AMT-061 Prevents Bleeds After 1 Year
After one year, the investigational gene therapy AMT-061 (etranacogene dezaparvovec) continued to safely and effectively increase factor IX (FIX)…