Gene therapy is a revolutionary field in modern medicine, offering transformative potential to treat and potentially cure a wide range of genetic and acquired diseases. By addressing the root cause of ...

Backed by a handful of prominent investors including the Gates Foundation, the startup is pursuing an approach it believes ...

After Pfizer let limited patient interest get the best of its hemophilia B gene therapy ambitions and discontinued Beqvez, CSL Behring naturally faced the question of whether it can hold its ground ...

It’s now possible to treat inherited blood diseases, such as sickle cell disease, with gene editing. Blood stem cells are extracted from the patient, modified, and infused back into their bone ...

Scientists at St. Jude Children's Research Hospital and Northwestern University identified a previously unknown treatment opportunity for sickle cell disease and β-thalassemia. The discovery, ...

Lowering cholesterol is one of the most effective ways to reduce your risk of heart disease, and it may soon be possible to get a one-and-done gene therapy to keep cholesterol and triglyceride levels ...

The great hope for gene therapy is that one day it will be out of the clinic and into mainstream therapeutics. Bearing enormous potential to treat or cure disease, such technology can replace a ...

Fractyl Health, Inc. announced promising preclinical results for its gene therapy RJVA-001 at the 2025 ASGCT Annual Meeting, showing that a single dose leads to substantial metabolic improvements ...

As we continue our series exploring genetic medicine, both in this story and in Destiny's Child No Longer: Rewriting Genetic Fate, gene therapy stands at a crossroads. On June 16, Sarepta Therapeutics ...