the FDA just approved the first ever crisper based therapy a medicine called cevi the treatment from vertex Pharmaceuticals and crisper Therapeutics is considered a milestone in genetic medicine it is used to treat both CLE cell disease and beta Thia these diseases affect hundreds of thousands of people mostly of African and Asian descent here's how it works both disorders stem from mutations that cause problems with hemoglobin the protein and red blood cells that carries oxygen in patients with sick cell disease the mutated hemoglobin twists red blood cells into a crescent or sickle shape and can create
painful blockages that inhibit the flow of blood and oxygen to the body patients with beta D don't produce enough hemoglobin in the first place and have lower blood oxygen levels as a result cevi can be used to treat both conditions by using crisper to edit a gene in a patient's own blood stem cells so they resume production of fetal hemoglobin fetal hemoglobin is naturally produced during fetal development but this stops shortly after birth it's a health healthy form of hemoglobin that carries oxygen and when infused into a patient can greatly improve their condition this footage
provided by vertex shows how the drug is made after receiving a bag of the patient's blood cells a machine is used to isolate the stem cells from there the cells are prepped for Gene editing the components of crisper a cutting enzyme called C 9 and a guide RNA that steers the gene editing molecule to the right place in the genome are added to the cells and the mixture is put through a device called an electroporation machine which uses electricity to shuttle the materials into the cells once the edited cells have had time to grow and
be harvested they are put into vials and Frozen until it is time to infuse the therapy into a patient unfortunately the treatment is difficult for patients to make room for the edited cells they must first receive chemotherapy to wipe out the existing bone Mar this can lead to hair loss painful M sores and infertility patients have to spend weeks in the hospital after the infusion the whole process can take months and will cost millions of dollarars [Music]
