Showing 2828 results for "hemophilia"

IND-enabling studies for hemophilia A gene-editing therapy to advance

Metagenomi, which is developing MGX-001, a gene-editing therapy for hemophilia A, is preparing to initiate investigational new drug (IND)-enabling activities that seek to support the therapy’s transition from preclinical to clinical testing. The move is backed by preclinical studies in nonhuman primates that show the treatment led to…

Understanding the nuances of hemophilia bleeding

After a decade of living alongside my husband, Jared, and his severe hemophilia B, I’ve learned that not all bleeding is as straightforward as it may seem. Bleeds vary widely, not just in size but in severity and impact. What might look minor can be excruciating, while larger bleeds,…

Centessa discontinues development of SerpinPC for hemophilia

Centessa Pharmaceuticals is discontinuing the clinical development of SerpinPC, its experimental under-the-skin therapy for people with hemophilia A and hemophilia B with or without inhibitors. The therapy was being evaluated in several Phase 2 clinical trials. The decision follows the recent approval of Pfizer’s Hympavzi…