Clinical News

  FT-4202, FORMA Therapeutics’ experimental disease-modifying therapy for sickle cell disease (SCD), shows a favorable safety and pharmacokinetic profile in healthy volunteers, according to data from a Phase 1 clinical trial. Patient recruitment is open for the second part of the trial (NCT03815695). Information about trial locations, all in the U.S., is available here. ...

  An investigational gene therapy for Sickle Cell Disease (SCD) and β-thalassaemia, known as ARU-1801, has been given Orphan Drug Designation an orphan drug by the U.S. Food and Drug Administration (FDA), a status that helps to advance and support its development. The therapy is expected to increase functioning red blood cells through ...

  Bluebird bio, Inc. announced earlier today the launch  of ZYNTEGLO™ in Germany,  the company’s one-time gene therapy for patients 12 years and older with transfusion-dependent β-thalassaemia (TDT) who do not have a β0/β0 genotype, for whom haematopoietic stem cell (HSC) transplantation is appropriate but a human leukocyte antigen (HLA)-matched related ...

The Committee for Medicinal Products for Human Use (CHMP), the European Medicines Agency‘ (EMA) committee responsible for human medicines, has recently adopted a positive opinion recommending the granting of a marketing authorisation for the medicinal product Deferasirox Accord, by Accord Healthcare S.L.U.   Deferasirox Accord is a generic of Exjade, ...

  The European Medicines Agency’s (EMA) has recently granted marketing authorisation for the medicinal product ”Deferasirox Mylan”, intended for the treatment of chronic iron overload due to blood transfusions in patients with β-thalassaemia major, non-transfusion-dependent thalassaemia syndromes and other anaemias. The manufacturer of this medicinal product is Mylan S.A.S. ”Deferasirox Mylan” will be ...

  In a scientific paper recently published in Hematology, researchers discussed the most common pathophysiologic and clinical manifestations of renal disease in patients with beta thalassaemia. “In recent years, the life expectancy of patients with thalassaemia has increased, and this has allowed previously unrecognized renal complications to reveal themselves,” said ...

Companies involved in human gene editing have entered the ethical debate about the controversial technology, issuing a joint declaration that DNA must not be altered in a way that passes genetic changes on to future generations. The Alliance for Regenerative Medicine (ARM), an international group representing the cell and gene ...

  Zynteglo, Bluebird Bio’s ex vivo gene therapy for β-thalassaemia, got a green light from the EMA for conditional marketing authorization making it the 1st gene therapy for patients with thalassaemia to win such an approval.  The one-time gene therapy has been approved for patients 12 years and older with ...

  The European Medicines Agency (EMA) has published a booklet describing the journey of a medicine for human use authorised through EMA, from initial research to discussions on patient access to medicines across the EU. The booklet’s title is ”From laboratory to patient: the journey of a centrally authorised medicine” ...