Zynteglo

Bluebirdbio Announces The Lifting Of FDA Clinical Hold for Sickle Cell Disease And β-Thalassaemia Studies
Clinical News

Bluebirdbio Announces The Lifting Of FDA Clinical Hold for Sickle Cell Disease And β-Thalassaemia Studies

bluebird bio, Inc. today announced that the U.S. Food and Drug Administration (FDA) has lifted the clinical holds on the Phase 1/2 HGB-206 and Phase 3 HGB-210 studies of LentiGlobin…
TIF’s Statement on Accessibility of Gene Therapy for Thalassaemia
News

TIF’s Statement on Accessibility of Gene Therapy for Thalassaemia

The Thalassaemia International Federation (TIF) expresses its disappointment about the outcome of the reimbursement negotiation process concerning gene therapy (Zynteglo™) for transfusion-dependent thalassaemia patients, announced last week. The Federation’s distress…
A Head-to-Head Comparison of BMT, Gene Therapy & Gene Editing Through A Brand New TIF Infographic
News

A Head-to-Head Comparison of BMT, Gene Therapy & Gene Editing Through A Brand New TIF Infographic

The expansion of management options and therapies for patients with thalassaemia is nowadays a reality, thanks to the application of new, revolutionary scientific procedures. However, patients‘ understanding on the distinct…
TIF’s Submission To The UK NICE’s Consultation Document For Zynteglo®
News

TIF’s Submission To The UK NICE’s Consultation Document For Zynteglo®

On February 12, in an appraisal consultation document, the UK National Institute for Health and Care Excellence (NICE) recommended against the use of Bluebird Bio’s Zynteglo (betibeglogene autotemcel) gene therapy for the…
TIF’s Public Statement on Current Developments in Gene Therapy SCD Clinical Trials and Thalassaemia Gene Therapy Zynteglo
Clinical News

TIF’s Public Statement on Current Developments in Gene Therapy SCD Clinical Trials and Thalassaemia Gene Therapy Zynteglo

The Thalassaemia International Federation (TIF) has been informed through official communication by both the European Medicines Agency (EMA) and bluebird bio that the latter has placed Phase 1/2 and Phase…
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)…
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
News

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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