Innovative Therapies
Juggling Between The Cost And Value of New Therapies: Does Science Still Serve Patient Needs?
Press Releases
3 εβδομάδες ago
Juggling Between The Cost And Value of New Therapies: Does Science Still Serve Patient Needs?
In the aftermath of the withdrawal of Zynteglo, the first gene therapy for patients with β-thalassaemia, from Europe, TIF´s Executive Director, Dr Androulla Eleftheriou, reflects on the viability of the…
Coming Up This Thursday: Discussion On Access To Innovative Therapies – The Reblozyl Case
Νέα
3 εβδομάδες ago
Coming Up This Thursday: Discussion On Access To Innovative Therapies – The Reblozyl Case
After receiving numerous questions from patients regarding the innovative treatment for β-thalassaemia Reblozyl (luspatercept), approved for use in the US, Europe, and Canada, TIF proceeds to organise an interactive, online…
Live Webinar on Thalassemia: Advances in 2021 with Dr M.D. Cappellini
Νέα
6 Ιουλίου, 2021
Live Webinar on Thalassemia: Advances in 2021 with Dr M.D. Cappellini
The past decade has witnessed path-breaking advances with regard to novel therapeutic approaches of thalassaemia and, most significantly, phenomenal developments made in the direction of gene therapy of β- thalassaemia…
TIF’s Statement on Accessibility of Gene Therapy for Thalassaemia
Press Releases
26 Απριλίου, 2021
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…
Clinical Trial Cleared for GPH101: The First Potentially Curative Sickle Cell Disease Therapy
Κλινικά Νέα
22 Ιανουαρίου, 2021
Clinical Trial Cleared for GPH101: The First Potentially Curative Sickle Cell Disease Therapy
The U.S. Food & Drug Administration (FDA) has cleared an investigational new drug (IND) application for the experimental gene editing therapy GPH101 by Graphite Bio to initiate a Phase 1/2…
EdiGene Announces Approval in China of its IND Application for CRISPR/Cas 9 Gene Editing Therapy ET-01 in β-thalassaemia
Νέα
21 Ιανουαρίου, 2021
EdiGene Announces Approval in China of its IND Application for CRISPR/Cas 9 Gene Editing Therapy ET-01 in β-thalassaemia
The Center for Drug Evaluation (CDE) of China‘s National Medical Products Administration (NMPA) has recently approved EdiGene’s application for ET-01, an investigational CRISPR-Cas9 gene editing therapy for patients with transfusion dependent β-thalassaemia. This marks the first…
What’s New In Clinical Trials for Thalassaemia and Sickle Cell Disease?
Κλινικά Νέα
6 Νοεμβρίου, 2020
What’s New In Clinical Trials for Thalassaemia and Sickle Cell Disease?
There is a lot of hustle and bustle in the scientific fields of thalassemia and sickle cell disease during the past few months, with many companies making remarkable progress in…
EdiGene Application of CRISPR/Cas 9 Gene-editing Therapy ET-01 For β-Thalassaemia Has Been Accepted For Review
Κλινικά Νέα
27 Οκτωβρίου, 2020
EdiGene Application of CRISPR/Cas 9 Gene-editing Therapy ET-01 For β-Thalassaemia Has Been Accepted For Review
Τhe Chinese biotechnology company EdiGene announced yesterday that the Center for Drug Evaluation (CDE) of China’s National Medical Products Administration (NMPA) has accepted for review the Company’s Investigational New Drug…
Health Canada Approves REBLOZYL® For Adult Patients with β-Thalassaemia
Κλινικά Νέα
30 Σεπτεμβρίου, 2020
Health Canada Approves REBLOZYL® For Adult Patients with β-Thalassaemia
Bristol Myers Squibb Canada (BMS) and Acceleron Pharma Inc. announced today that Health Canada has approved REBLOZYL® (luspatercept) for the treatment of adult patients with red blood cell (RBC) transfusion-dependent anemia…
Gene-editing Therapy CTX-001 Granted Priority Medicines Designation for Severe SCD in EU
Νέα
25 Σεπτεμβρίου, 2020
Gene-editing Therapy CTX-001 Granted Priority Medicines Designation for Severe SCD in EU
CTX001, an investigational gene-editing cell therapy, has been granted priority medicines (PRIME) designation by the European Medicines Agency (EMA) for the treatment of severe sickle cell disease (SCD). The announcement was made by CRISPR Therapeutics and Vertex Pharmaceuticals, which are jointly…