Ocugen has reported preliminary phase 2 data on its eye disease gene therapy, providing more evidence that the candidate may outperform existing treatments from Apellis Pharmaceuticals and Astellas ...

This image shows three brain cells with the faulty protein that causes Huntington’s disease. The bright yellow cell in the middle has built up a clump of this protein inside it. The blue spots in the ...

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

Three Philadelphia scientists won a $3 million Breakthrough Prize for developing the first FDA-approved gene therapy for a genetic disease, prize sponsors announced Saturday. Their work created a ...

For years, cell and gene therapies were mainly associated with rare and orphan diseases, those that impact small patient groups and lack effective treatment options. However, recent developments in ...

Three scientists have won the 2026 Breakthrough Prize for developing Luxturna — the world's first FDA-approved gene therapy ...