Medscape

CRISPR-Based Gene Therapy Earns Beta Thalassemia Approval

The US Food and Drug Administration (FDA) has approved Vertex Pharmaceuticals' CRISPR gene-editing–based therapy exagamglogene autotemcel, or exa-cel, (Casgevy) for individuals aged 12 years or older ...
The American Journal of Managed Care

FDA Approves First Gene Therapy for Beta-thalassemia

The FDA Wednesday approved betibeglogene autotemcel (Zynteglo), the first cell-based gene therapy for the treatment of adult and pediatric patients with beta-thalassemia who require regular red blood ...
Inverse

The First CRISPR Gene Therapy Was Finally Approved In The US — Here’s How It Works

In the ever-evolving realm of modern medicine, a once-distant dream to conquer disease through genetics has now inched closer to reality. Last Friday, the U.S. Food and Drug Administration approved ...
Healio

Gene therapy potentially curative for patients with transfusion-dependent beta-thalassemia

Please provide your email address to receive an email when new articles are posted on . Thompson described beta-thalassemia as a severe chronic inherited blood disorder due to one of many mutations in ...
Monthly Prescribing Reference

One-Time Gene Therapy Gets Priority Review for Beta-Thalassemia

Credit: Getty Images. Betibeglogene autotemcel (beti-cel) is a one-time gene therapy that works by adding functional copies of a modified form of the β-globin gene into a patient’s own hematopoietic ...
Business Wire

FDA Advisory Committee Unanimously Supports beti-cel Gene Therapy for People with beta-thalassemia Who Require Regular Red Blood Cell Transfusions

SOMERVILLE, Mass.--(BUSINESS WIRE)--bluebird bio, Inc. (Nasdaq: BLUE) today announced the outcome of the U.S. Food and Drug Administration’s (FDA) Cellular, Tissue, and Gene Therapies Advisory ...
UC San Francisco

Oakland Hospital First in West to Pioneer Beta Thalassemia Drug

UCSF Benioff Children’s Hospital Oakland is the first hospital in the West to administer a newly approved gene therapy to treat beta thalassemia, a rare, genetic blood disorder that causes severe ...
News Medical

JRC releases a new CRM to aid the fight against beta-thalassemia

Our blood is a window to our health. Doctors examine biomarkers in our blood to determine our health status. These molecules can indicate if any abnormal processes are taking place, which could point ...
Business Wire

Graphite Bio Presents Preclinical Gene Replacement Data for GPH102 for Beta-thalassemia at the ASGCT 25th Annual Meeting

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Graphite Bio, Inc. (Nasdaq: GRPH), a clinical-stage, next-generation gene editing company harnessing the power of high-efficiency precision gene repair to ...
STAT

Gene therapy offered this 7-year-old freedom. The price: a grueling year

PHILADELPHIA — The meds Shelby Campbell needed for her rare blood disorder stopped working just after her sixth birthday. She lost her appetite and was often doubled over in pain. She continued ...
STAT

Patients with rare blood disorder benefit from Bluebird gene therapy while denied access in Europe

People with severe beta-thalassemia who live in Europe have been denied access to Bluebird Bio’s approved gene therapy after the company withdrew it from the market last April. European health systems ...