Reni-cel gene therapy shows transfusion independence in small phase 1-2 trial for β-thalassemia
New Gene Edit Stops Blood Transfusions
A single-group phase 1-2 trial in 9 adults with transfusion-dependent β-thalassemia found all participants achieved neutrophil engraftment a…
A new gene edit reactivates natural red blood cell production, potentially ending the need for lifelong transfusions in adults with severe b…
Systematic review finds first FDA-approved CRISPR therapy and broader clinical trial exploration
First CRISPR gene therapy approved for blood disorders, with broader research underway
A systematic review of clinical trials registered at clinicaltrials.gov found the first CRISPR-Cas9 therapy obtained FDA approval in late 20…
The first CRISPR gene therapy is approved for sickle-cell anemia and β-thalassemia, with ongoing trials exploring its use for cancer and inf…