With gene therapy now a clinical reality, innovators are designing delivery systems that can sustain lifelong correction, handle complex genes, and make treatments accessible to more patients.
Explore how single-dose gene therapy offers a potential long-term treatment for hemophilia, and discover the challenges contributing to its low adoption.
Learn how developmental and reproductive toxicology study selection supports regulatory decision-making and generates meaningful nonclinical safety data.
By combining organoid biology with precision automation, researchers developed a miniaturized organoid screening platform that could help speed personalized cancer treatment testing.