Clinical News
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CLINICAL NEWS | PYRUKYND® Receives Positive CHMP Opinion For The Treatment of PKD in Adults
PYRUKYND® is a first-in-class, oral PK activator that was recently approved by the U.S. Food and Drug Administration (FDA). If approved by the European Commission (EC), PYRUKYND® will be the first approved disease-modifying therapy…
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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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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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Roche and Atea Pharmaceuticals Link Up To Co-Develop Remdesivir Rival Drug For COVID-19
The two companies will jointly develop, manufacture and distribute AT-527, Atea’s investigational oral antiviral for the potential treatment of COVID-19. According to Roche, AT-527 blocks the viral RNA polymerase enzyme…
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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…
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EU Approves Adakveo to Treat Vaso Occlusive Crises in Sickle Cell Disease Patients
The European Commission has approved Novartis’ Adakveo (crizanlizumab) as a preventive treatment for recurrent Vaso-Occlusive Crises (VOCs) in patients, 16 and older, with sickle cell disease (SCD). This approval follows a recommendation for conditional approval issued in July…
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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…
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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…
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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…
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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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