No real COVID-19 infection impact seen for hemophilia patients
Having a COVID-19 infection had no significant impact on bleeding episodes, nor on the levels of factor replacement therapy…
Articles by Andrea Lobo, PhD
MGX-001 gene-editing therapy shows promise in preclinical study
MGX-001, a gene-editing therapy being developed by Metagenomi for hemophilia A, has shown promising safety and durability in…
Hemophilia A, B gene therapies now at Loma Linda University Health
Loma Linda University Health (LLUH) is now offering the gene therapies Hemgenix (etranacogene dezaparvovec) to people with hemophilia B…
B-cell therapy BE-101 granted FDA’s orphan drug status for hemophilia B
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to BE-101, Be Biopharma’s B-cell treatment candidate…
Panel recommends EU approve hemophilia gene therapy Durveqtix
A European Medicines Agency (EMA) committee has issued a positive opinion recommending conditional marketing authorization of gene therapy Durveqtix…
FDA OKs clinical testing of BE-101 as potential hemophilia B treatment
The U.S. Food and Drug Administration (FDA) has cleared clinical testing in adults of BE-101, a B-cell treatment candidate for…
Avenacy launches generic DDAVP for bleeding in mild hemophilia A
A generic version of DDAVP (desmopressin acetate), a therapy approved to control bleeding episodes in people with mild hemophilia…
Novoeight safely and effectively treats, prevents bleeds long term
When used long term, Novoeight (turoctocog alfa) safely and effectively prevented and treated bleeding episodes in people with…
FDA OKs first-in-human study of F9 gene therapy for hemophilia B
Intellia Therapeutics and Regeneron are planning to begin, by mid-year, the first-in-human trial testing a CRISPR/Cas9-based Factor 9 (F9) gene-editing…
FDA grants orphan drug status to TI-168 for hemophilia A inhibitors
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Baudax Bio’s investigational regulatory T-cell therapy TI-168…