Italy has begun administering the first CRISPR-based treatment. The therapy for sickle cell disease and beta thalassemia (Casgevy) has already been delivered to four patients across three clinical centers within a month. The announcement was made during the conference “Italian Primacy in the Treatment of Hemoglobinopathies” held yesterday at the Senate.
For more information, we recommend Francesca Ceradini’s article in Osservatorio Terapie Avanzate.
Not only sickle cell anemia, now thalassemia as well. On January 16, the Food and Drug Administration completed the approval process for the second type of hemoglobinopathy too, while the European Medicines Agency is expected to give the green light in the coming months. It took just over a decade to go from the invention of the Cas9 genetic scissors to the first approved treatment, and the excitement over the milestone achieved in record time is more than justified. Yet an article in MIT Technology Review has already turned the spotlight on the next challenges. The title is, “Vertex developed a CRISPR cure. I’ts already on the hunt for something better”. Gentler conditioning for ex vivo gene editing, new vectors for in vivo delivery, and maybe even a pill mimicking the Casgevy mechanism without modifying DNA. This is how CRISPR researchers try to out-innovate themselves.
Emanuele and Erika Guarini are brother and sister. They were treated for thalassemia respectively in November 2020 and August 2021 by the team of Franco Locatelli at Bambin Gesù hospital in Rome. Before the CRISPR-based treatment, they needed a blood transfusion every 15-20 days (source La Repubblica).
Off-target paper retraction: Nature Methods has retracted a controversial study questioning CRISPR precision, after its authors admitted they were probably wrong. This blog’s wish is that future studies on CRISPR flaws and virtues are as reliable as the genome-editing technique.
Deregulation statement: US Secretary of Agriculture Sonny Perdue has announced that USDA will not regulate edited plants as long as they could have been created through conventional breeding. Let’s hope Europe will follow the example.
Gene therapy going “organic”: that’s the hope expressed by Merlin Crossley, when commenting his Nature Genetics paper on mutations beneficial to patients with β-thalassemia and sickle cell anemia. The word organic here means that fetal hemoglobin production can be boosted without inserting foreign DNA.
CRISPeR FRENZY 