Zynteglo

Gene Therapy LentiGlobin Granted Priority Medicines Designation for Sickle Cell Disease in the EU
Clinical News

Gene Therapy LentiGlobin Granted Priority Medicines Designation for Sickle Cell Disease in the EU

Bluebird Bio’s investigational gene therapy LentiGlobin has been granted priority medicines (PRIME) designation by the European Medicines Agency (EMA) for the treatment of sickle cell disease (SCD). Given to candidate therapies with promising clinical data for diseases of high…
TIF.ACCESS: A Global TIF Initiative for the Promotion of the Availability & Accessibility of Innovative Therapies
News

TIF.ACCESS: A Global TIF Initiative for the Promotion of the Availability & Accessibility of Innovative Therapies

The swift scientific progress made in the field of thalassaemia research has gained momentum with the market authorisations of both the Zynteglo Gene Therapy by the European Medicines Agency (EMA)…
Gene Therapy & Thalassaemia
Surveys

Gene Therapy & Thalassaemia

 Millions of people worldwide suffer from inherited non-malignant blood disorders like thalassaemia and sickle cell disease. The culprit: genetic defects in their blood stem cells.   One of the novel…
Explaining Gene Therapy in thalassaemia (2020)
Publications

Explaining Gene Therapy in thalassaemia (2020)

The second educational brochure from the TIF’s ‘Gene Therapy’ booklet series, providing an in-depth view of how this revolutionary therapy works in patients with β-thalassaemia major.
The Patients’ Perspective on the Accessibility & Affordability of Novel Therapies for Thalassaemia
International Thalassaemia Day Webinars

The Patients’ Perspective on the Accessibility & Affordability of Novel Therapies for Thalassaemia

9 patients with thalassaemia, members of TIF’s Patient Advocacy Group for Thalassaemia and Sickle Cell Disease (T-PAG), are discussing with the Thalassaemia International Federation the accessibility and affordability of the…
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