Innovative Therapies

Live Webinar on Thalassemia: Advances in 2021 with Dr M.D. Cappellini
Member News

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
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…
Clinical Trial Cleared for GPH101: The First Potentially Curative Sickle Cell Disease Therapy
Clinical News

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
News

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?
Clinical News

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
Clinical News

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
Clinical News

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
News

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…
IMR-687 Receives FDA Fast Track & Rare Pediatric Disease Designations for the Treatment of β-Τhalassaemia
Clinical News

IMR-687 Receives FDA Fast Track & Rare Pediatric Disease Designations for the Treatment of β-Τhalassaemia

IMARA Inc., a clinical-stage biopharmaceutical company dedicated to developing and commercializing novel therapeutics to treat patients suffering from rare inherited genetic disorders of haemoglobin, announced the U.S. Food and Drug…
TIF Publishes its Annual Report for 2019
News

TIF Publishes its Annual Report for 2019

This Annual Report showcases the Federation’s intensive efforts that took place in 2019 with the aim to ensure equal access to quality healthcare for every patient with thalassaemia and haemoglobin…
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