The First Crispr Treatment Is Making Its Way to Patients
It’s been a year since the gene-editing treatment Casgevy was approved for sickle cell disease and a related blood disorder.
Glutamine metabolic switch is key to red blood cell development and disease, researchers reveal
Blood stem cells develop through different stages to become fully mature red blood cells. This fundamental biologic process ...
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Indonesian Thalassemia Survivor: "I've broken the inheritance chain of thalassemia"
Stanley Joewono is an Indonesia social media influencer and exercise enthusiast. Diagnosed with thalassemia at just 8 months ...
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Vertex's Casgevy breaks ground with 1st commercial patient infusions as company readies another major launch for 2025
With new approvals on the horizon and the closely watched Casgevy launch picking up steam in sickle cell disease and beta ...
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Beliefs About Chelation Among Thalassemia Patients
We were able to analyze a large, racially diverse, international sample of thalassemia patients, almost all on transfusion therapy, mostly using deferoxamine or deferasirox for chelation.
Gene Therapy Developer Beam Therapeutics Reports One Patient Death In Early-Stage Sickle Cell Disease Trial
Beam Therapeutics enrolls 35 patients in BEACON trial for BEAM-101 in sickle cell disease, with promising early safety and ...
Crispr Therapeutics Q3 Earnings: No Casgevy Revenue, But Plenty Of Optimism
Crispr has already secured a historic first approval for a CRISPR/Cas9 drug and has several more potential blockbuster ...
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Editas prioritizes in vivo gene therapies, looks to partner reni-cel
“We also continue to dose patients in the EdiTHAL trial of reni-cel for the treatment of beta thalassemia and will share additional clinical data this year,” they added. On a call with ...