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sickle cell disease
This page shows the latest sickle cell disease news and features for those working in and with pharma, biotech and healthcare.
bluebird bio sells second FDA priority review voucher for $95m
bluebird bio has agreed to sell its second Rare Pediatric Disease Priority Review Voucher (PRV) for $95m, the gene therapy developer announced. ... commercial launches of our two FDA-approved gene therapies and the opportunity ahead in sickle cell
Latest news
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Takeda therapy shows promise in rare blood clotting disorder
The disorder has both acute and chronic manifestations, including cardiovascular disease and stroke, and is associated with a significant disease burden. ... As well as the cTTP clinical development programme, TAK-755 is also being investigated in
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Jasper Therapeutics’ briquilimab given orphan drug designation in EU
Successful stem cell engraftment and immune reconstitution have also been observed in the trial. ... The company also announced positive results earlier this month from a phase 1/2 trial of briquilimab as a conditioning treatment in patients with sickle
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FDA lifts partial clinical hold on bluebird bio’s sickle cell gene therapy studies
The US Food and Drug Administration (FDA) has lifted its partial clinical hold on bluebird bio’s studies evaluating its lovotibeglogene autotemcel (lovo-cel) gene therapy for sickle cell disease (SCD) ... The disease can cause serious health problems,
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Genomics England receives government funding for cutting-edge genomics research
Currently, the NHS heel prick blood test carried out as part of newborn screening is used to detect nine rare but serious health conditions in babies, including cystic fibrosis and sickle ... cell disease.
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bluebird bio sells Rare Pediatric Disease Priority Review Voucher
creating milestones, including the planned submission and subsequent FDA review of our biologics licensing application for lovo-cel for sickle cell disease.”. ... Prior to the approval of Skysona, effective options were limited to allogeneic
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Latest Intelligence
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Bluebird: on a mission to ‘recode’ the DNA of healthcare
It is also investigating the therapy in sickle cell disease, with a phase 3 trial set to begin this year. ... Increasingly factored into this equation is competition from companies following Bluebird into beta thalassemia and sickle cell disease.
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Vertex: a vision of treating all CF patients worldwide draws closer
Luckily, that plan B was waiting in the wings: Kalydeco (ivacaftor), the first ever disease-modifying treatment for cystic fibrosis gained approval in 2012. ... with sickle cell disease or beta thalassemia with FDA Fast Track Designation.
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The Orkambi blame game
for other diseases, such as sickle cell disease and alpha-1 antitrypsin, which we are working on. ... for our disease.”.
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2019: CRISPR and therapeutic gene editing comes of age
The FDA placed the CTX001 programmed in beta thalassemia and a planned trial in sickle cell disease on a clinical hold shortly afterwards, but relaxed that last October. ... consequences – for example if it led to the expression of a disease gene.
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Bluebird Bio: on the cusp of a gene therapy revolution
While previous read-outs had prepared the ground for this remarkable result, a similar success in sickle cell disease also unveiled at EHA came as a very welcome surprise for the ... sickle cell disease.
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Latest appointments
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Cellerant appoints William Reed as VP clinical development
Will lead development of stem cell-based treatments for blood conditions and cancer. ... He has previous experience in treatments for blood conditions, spending time as director, clinical research and medical affairs at Cerus, where he worked on a
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Latest from PMHub
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The promised land of gene therapy: Commercialization of novel gene-editing technology in beta-thalassemia
patients and families affected by devastating diseases such as sickle cell disease and thalassemia. ... Several manufacturers are developing novel treatments for the disease, including Vertex, which has partnered with CRISPR Therapeutics to develop a gene
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Overcoming Patient Recruitment Challenges in Rare Disease Trials
This webinar will:. explore some of the patient recruitment challenges in rare disease studies, particularly relating to sickle cell trials. ... Learning Objective. Patient recruitment challenges in rare disease studies. Challenges in sickle cells studies
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FREE Live Webinar - Overcoming Patient Recruitment Challenges in Rare Disease Trials
patient recruitment challenges in rare disease studies, particularly relating to sickle cell trials. ... hear from a patient and family member about what it means to live with a rare disease.
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October 2020: diversity and inclusion in clinical trials round-up
In such settings, specialised sickle cell disease services and treatment is often inaccessible or unaffordable. ... As a result, adolescents and young adults with sickle cell disease in the U.S.
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How are patient insights applied to clinical trials?
For example, Black African and Latinx individuals are more adversely affected by sickle cell disease, so it’s important to reach out to these diverse groups and gain their insights, to
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