Innovative Therapies
Clinical Trial Cleared for GPH101: The First Potentially Curative Sickle Cell Disease Therapy
Clinical News
Ιανουάριος 22, 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
Ιανουάριος 21, 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?
Clinical News
Νοέμβριος 6, 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
Clinical News
Οκτώβριος 27, 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
Clinical News
Σεπτέμβριος 30, 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
Σεπτέμβριος 25, 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…
IMR-687 Receives FDA Fast Track & Rare Pediatric Disease Designations for the Treatment of β-Τhalassaemia
Clinical News
Αύγουστος 5, 2020
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
Νέα
Ιούλιος 30, 2020
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…
Novartis sickle cell drug Adakveo put on path to EU approval
Clinical News
Ιούλιος 27, 2020
Novartis sickle cell drug Adakveo put on path to EU approval
Novartis announced last Friday that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion recommending conditional marketing authorization of Adakveo® (crizanlizumab)…
TIF.ACCESS: A Global TIF Initiative for the Promotion of the Availability & Accessibility of Innovative Therapies
Νέα
Ιούλιος 16, 2020
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)…