Cell Therapy

• The FDA granted accelerated approval to a CRISPR-based gene therapy for severe sickle cell disease on April 20, marking the first approved genetic treatment editing patient blood cells to produce functional hemoglobin. • Clinical trials demonstrated 95% of treated patients experienced elimination or near-elimination of vaso-occlusive crises within 12 months, with durable responses observed for over two years post-treatment. • The therapy addresses a disease affecting approximately 100,000 Americans, primarily of African descent, offering potential cure for patients previously reliant on pain management and blood transfusions.

• Elijah Dottery, diagnosed with sickle cell anemia as a baby, became the first U-M patient to receive the newly FDA-approved stem cell therapy. • The treatment uses patient's own stem cells edited via CRISPR to produce functional hemoglobin, administered at Michigan Medicine. • This breakthrough matters for over 100,000 U.S. sickle cell patients, offering potential cure where prior therapies only managed symptoms.

Treatment reset wayward immune system of patient with life-threatening conditions, say scientists, in a world firstA woman who lived with three life-threatening autoimmune diseases for more than a decade has returned to a near-normal life after a cell therapy reset her wayward immune system.The 47-year-old had had nine different treatments, none of which had a lasting impact, before receiving the therapy last year at University Hospital Erlangen in Germany. At the time, she required daily blood transfusions and permanent blood thinning medication to control her illness. Continue reading...