Clinical News
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WHAT YOU NEED TO KNOW | EMA Recommends Suspension of Pfizer’s Sickle Cell Disease Drug Oxbryta
Oxbryta, which was designed to treat sickle cell disease, has been a crucial medication for patients with mild-to-moderate symptoms. Approved in the US under an accelerated process in 2019 and…
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SICKLE CELL DISEASE | FDA Approves Two Landmark Gene Therapies for the Treatment of the Disorder
The two therapies are Casgevy (exa-cel) by Vertex Pharmaceuticals and CRISPR Therapeutics – the world’s first drug to utilize the revolutionary CRISPR gene-editing system which earned its creators the 2020…
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TOP STORY | EU Approves Luspatercept for Anemia in Patients with Non–Transfusion-Dependent β-Thalassaemia
The approval was based on findings from the phase 2 BEYOND trial, which demonstrated that 77.1% of patients treated with luspatercept (n = 74/96) experienced a mean haemoglobin (Hb) increase…
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IRON CONTROL | Disc Medicine And Mabwell Sign Deal For Licence To Antibodies Portfolio
Disc plans to initiate a phase 1 trial in healthy volunteers in the second half of 2023. MWTX-003 has the potential to address a wide range of hematologic disorders including…
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CLINICAL UPDATE | Graphite’s Hopes For Sickle Cell Cure Blunted After First Patient Dosed Experiences Serious Event
The company cited a “serious adverse event in the first patient dosed” with its gene therapy nulabeglogene autogedtemcel (nula-cel) as part of a phase 1/2 trial for SCD. The event is likely to…
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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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