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.
EurekAlert!2d
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 ...
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 ...
Gilead, Vertex initiated as new big biotech buys at Citi
Citi initiates coverage of Gilead Sciences and Vertex Pharmaceuticals at buy in the large U.S. biotech sector. Read more here.
Medscape28d
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.
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 ...
FierceBiotech24d
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 ...