Gene Therapy
Top in hem/onc: Sickle cell gene therapy, primary care in cancer centers
Last week, bluebird bio suspended clinical trials with its LentiGlobin product for sickle cell disease due to reported cases of hematologic malignancies among patients who received the treatment. It was the top story in hematology/oncology.
Gene therapy shows durable efficacy for transfusion-dependent beta-thalassemia
Long-term follow-up data showed betibeglogene autotemcel to be a safe and efficacious treatment for patients with transfusion-dependent beta-thalassemia, according to study results presented at TCT Meetings Digital Experience.
Collaboration examines nanoparticle drug delivery system in Charcot-Marie Tooth disease
Manufacturer suspends clinical trials for gene therapy to treat sickle cell disease
Gene therapy developer bluebird bio suspended clinical trials using its LentiGlobin product for sickle cell disease due to reported cases of hematologic malignancies among patients who received the treatment.
Investigational gene therapy well tolerated in patients with AMD
A majority of patients with age-related macular degeneration treated with investigational gene therapy GT005 experienced increases in complement factor I levels in an ongoing phase 1/2 clinical trial.
After challenging year, cell and gene therapy industry poised for further growth in 2021
Challenges presented by the COVID-19 pandemic did not stop progress in research or clinical development of gene and cell therapies, according to a state-of-the-industry briefing by Alliance of Regenerative Medicine.
FDA grants fast track designation to gene therapy candidate for achromatopsia
VIDEO: Gene therapy presentations are ‘groundbreaking’
In this video, Caroline R. Baumal, MD, professor at Tufts University School of Medicine, discusses topics in age-related macular degeneration from the virtual American Academy of Ophthalmology annual meeting and her hopes for 2021.