From Pain to Purpose: Local Woman Transformed by Groundbreaking Cleveland Clinic Sickle Cell Trial

From Pain to Purpose: Local woman transformed by groundbreaking Cleveland Clinic sickle cell trial

For most of her life, Annyanna Cash of Oakwood Village knew hospitals better than her own home.

Gene editing therapy shows success against severe sickle cell disease

New results from a clinical trial show promising outcomes for a gene-edited treatment for severe sickle cell disease, a genetic blood disorder with few curative options. After research conducted as part of the multicenter RUBY Trial, researchers have published their latest findings in the New England Journal of Medicine. Remarkably, 27 out of 28 patients did not have any painful sickle cell crises after treatment, achieving what physicians call …

Base-Editing Gene Therapy Shows Promise in Sickle Cell Disease

(MedPage Today) -- Treatment with the investigational base-editing gene therapy ristoglogene autogetemcel (risto-cel) showed promise in patients with sickle cell disease, according to an interim analysis of the phase I/II BEACON study. Neutrophil...

New Gene-Editing Approach Eliminates Painful Crises in Sickle Cell Disease New Results From A Clinical...

New results from a clinical trial show promising outcomes for a gene-edited treatment for severe sickle cell disease, a genetic blood disorder with few curative options.

Beam Therapeutics Announces Publication of BEACON Phase 1/2 Data for risto-cel in Patients with Sickle Cell Disease (SCD) in The New England Journal of Medicine

Gene Editing Therapy Succeeds in 96% of Sickle Cell Disease Patients

Editas Medicine has reported positive outcomes for a gene editing treatment to treat severe sickle cell disease, a hereditary blood disorder with limited treatment options. Published today in the New England Journal of Medicine, results from the RUBY trial show a functional cure was achieved in 27 out of 28 patients, who had no more sickle cell crises in the two years after the therapy was administered.  The treatment in question, called renizga…