β-thalassaemia

Ali Sattarpour: ”Thalassaemia did not stop me from living a normal life”
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Ali Sattarpour: ”Thalassaemia did not stop me from living a normal life”

In occasion of the International Thalassaemia Day 2021, Ali Sattarpour, 45, France, Fédération SOS GLOBI Vice President and TIF’s Thalassaemia Patient Advocacy Group (T-PAG) Member, recounts a life of struggles,…
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…
Blood Donors’ COVID-19 Antibodies: Do They Have A Positive Significance for Blood Recipients?
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Blood Donors’ COVID-19 Antibodies: Do They Have A Positive Significance for Blood Recipients?

According to recent data released by the American Red Cross, in the first week of March 2021 more than 20% of blood donations from unvaccinated people in the US had…
A Head-to-Head Comparison of BMT, Gene Therapy & Gene Editing Through A Brand New TIF Infographic
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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…
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…
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…
Imara Announces First Patient Dosed in Forte Phase 2b Clinical Trial of IMR-687 in β-Thalassaemia
Clinical News

Imara Announces First Patient Dosed in Forte Phase 2b Clinical Trial of IMR-687 in β-Thalassaemia

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 dosing of the first patient…
Fulcrum Therapeutics to Initiate Phase 1 Trial with FTX-6058 for Sickle Cell Disease
Clinical News

Fulcrum Therapeutics to Initiate Phase 1 Trial with FTX-6058 for Sickle Cell Disease

Fulcrum Therapeutics, Inc.  a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, announced that FTX-6058, its investigational treatment for sickle cell disease (SCD) and…
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…
IMR-687 Receives FDA Orphan Drug Designation for the Treatment of β-Τhalassaemia
Clinical News

IMR-687 Receives FDA Orphan Drug Designation for the Treatment of β-Τhalassaemia

IMARA Inc., a clinical-stage biopharmaceutical company operating in the haemoglobin disorders field, announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for IMR-687 for the…
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