Despite rapid therapeutic advances in sickle cell disease, from red blood cell exchange to gene therapies, delivery remains constrained by infrastructure, geography, and care fragmentation.
At the Advanced Therapies conference, industry leaders showcased how in vivo CAR T cell and gene delivery innovations could transform accessibility and clinical outcomes.
The therapy lengthened telomeres and improved immune function in people with telomere biology disorder, showing promise for treating bone marrow failure safely.
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.