Gene Therapy for Deadly Blood Disorder Restores Function in Patients' Cells

UCLA researchers have made strides in developing a stem cell gene therapy for alpha thalassemia major, a severe, inherited ...

Gene Therapy Could End Lifelong Transfusions For Rare Blood Disorder

A single one-time gene therapy could free patients with α-thalassemia, a rare and debilitating blood disorder, from the ...

New gene therapy now administered as Sickle Cell Anemia treatment in St. Louis Children’s Hospital

According to the CDC, nearly 100,000 Americans have Sickle Cell Anemia, or 1 in every 265 African American births.
WPLG Local 10

South Florida teen becomes first in area to receive FDA-approved gene therapy for sickle cell

BROWARD COUNTY, Fla. — A South Florida family is happy their son no longer lives with chronic pain. Plus, he’s the first in the area to go through FDA-approved gene therapy. Caden Major had been ...
Morningstar

Beyond Gene Therapy: Pyruvate Kinase Activators (Agios, Novo Nordisk) Offer a New Route to Address Hemoglobin, VOCs, and Transfusion Burden in Sickle Cell Disease

EXTON, PA, Aug. 06, 2025 (GLOBE NEWSWIRE) -- According to the latest findings from Spherix Global Insights’ syndicated Market Dynamix™ service, the US sickle cell disease (SCD) market is approaching a ...