Clinical News
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NEW HOPE FOR THALASSAEMIA/SCD | UK Trial Of Lab Grown Red Blood Cells Begins
The Restore trial, led jointly by NHS Blood and Transplant and the University of Bristol, is studying the lifespan of the lab grown cells compared with infusions of standard red blood…
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ADVANCED THERAPIES | Vertex, CRISPR To Submit Exa-Cel to FDA For β-Thalassaemia And Sickle Cell Disease
Vertex will submit its biologics licensing application (BLA) for exa-cel for rolling review, beginning in November 2022 and expects to complete the submission package by the end of Q1 2023.…
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CDC | Many Children With Sickle Cell Anaemia In The US Not Receiving Lifesaving Screening And Treatment
According to a recent CDC study, fewer than half children with the disease in the US get the necessary screening, and only about half or fewer get treatment with hydroxyurea…
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SAFER BLOOD | EU Patients Have Begun Receiving Transfusions With Hemanext ONE® RBC Processing & Storage System
Doctors have begun transfusing patients suffering from hematological malignancies using RBCs processed and stored with the Hemanext ONE® system, as part of a post-market clinical study in Bergen, Norway. This is…
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BREAKING NEWS | Bluebird’s Gene Therapy For β-Thalassaemia Wins US Approval
The green light is “monumental” for both patients and the company itself, Tom Klima, Bluebird’s Chief Commercial Officer, said in an interview. Bluebird has endured its share of “bumps and…
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Hemanext® Inc. Receives CE Mark Certification For Innovative Red Blood Cell (RBC) Processing & Storage System
Hemanext Inc. announced today it has received a CE Certificate of Conformity for the CE Mark for its Hemanext ONE Red Blood Cell (RBC) Processing and Storage System, allowing the…
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TIF’s Public Statement on Current Developments in Gene Therapy SCD Clinical Trials and Thalassaemia Gene Therapy Zynteglo
The Thalassaemia International Federation (TIF) has been informed through official communication by both the European Medicines Agency (EMA) and bluebird bio that the latter has placed Phase 1/2 and Phase…
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The Lancet: Russian Coronavirus Sputnik V Vaccine Achieves Over 90% Efficacy
Russia’s Sputnik V vaccine was 91.6% effective in preventing people from developing Covid-19, according to peer-reviewed results from its late-stage clinical trial published yesterday in The Lancet international medical journal.…
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EMA Accepts MAA For Oxbryta To Treat Haemolytic Anaemia In Sickle Cell Disease
The European Medicines Agency (EMA) has accepted Global Blood Therapeutics (GBT) marketing authorisation application (MAA) for Oxbryta® (voxelotor) for the treatment of haemolytic anaemia in sickle cell disease (SCD). According to the company,…
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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…
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