Innovative Therapies

Juggling Between The Cost And Value of New Therapies: Does Science Still Serve Patient Needs?
Press Releases

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
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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
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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

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
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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
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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?
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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
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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

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
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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…
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