Hematology

The therapy attempts to restore fetal hemoglobin production.

The first patient has been treated in a phase 2b dose-confirmation study of AMT-061, an investigational gene therapy for the treatment of patients with severe and moderately severe hemophilia B. Once the dosing of AMT-061 is confirmed, the safety and efficacy of the therapy will be evaluated in the global phase 3 HOPE-B clinical trial.

The US Food and Drug Administration (FDA) has accepted the Bioverativ's Investigational New Drug (IND) application for BIVV003, a gene-edited cell therapy candidate for the treatment of people with sickle cell disease.

The FDA gave a gene therapy for bleeding in hemophilia B breakthrough status.

Researchers discovered a clinically translatable gene therapy protocol, which is a gene delivery system that introduced the production of therapeutic clotting proteins in platelets, so that the inhibition of inactivating antibodies is avoided.

A gene therapy treatment for men with hemophilia B proved effective, and the researchers involved in the study estimate it saved $2.5 million for the trial's patients.

Scientists have successfully engineered platelet cells in dogs to express higher levels of factor VIII, paving the way for further tests in humans.