At the end of June, the groundbreaking gene editing technology known as Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) surpassed the tenth anniversary of its creation. MRP has been closely following CRISPR and the ever-evolving methods by which it is implemented since 2018.
The first human trial utilizing CRISPR in the US started all the way back in 2019. The trial, focused on two blood diseases effecting millions of people, transfusion-dependent beta-thalassemia (TDT) and sickle cell disease (SCD), remains 100% effective to this day. Originally dubbed CTX001, and now called exa-cel, this specific treatment developed by CRISPR Therapeutics and Vertex Pharmaceuticals as part of a $900 million agreement is a one-time treatment that genetically alters the patient’s own cells to produce the kind of hemoglobin found at birth, which isn’t distorted, allowing red blood cells to work properly.
MRP initially covered these trials in 2020 when a trio of patients, two with TDT and one with severe SCD, saw benefits from one-time treatment with the experimental CTX001. After a follow-up of five to 15 months, all three patients are free from their once-routine need for blood transfusions and painful events that came with their disorders. “The preliminary results… demonstrate, in essence, a functional cure for patients with beta thalassemia and sickle cell disease,” team member Haydar Frangoul of Sarah Cannon Research Institute said at the time.
That was a bold statement to make. But new results from the same trial, which now includes 75 patients treated, showed the therapy has continued to generate near perfect efficacy. Recently presented at the European Hematology Association Congress, New Atlas notes the data shows 42 patients suffering from TDT and 31 from SCD remained essentially cured of their ailments after 32.3 – 37.2 months after treatment. Two of the TDT patients still require blood transfusions, but the transfusion volume required has declined by 75% and 89%. All adverse side effects, observed in only two patients, resolved themselves.
As Fast Company notes, the drugmakers say they intend to submit exa-cel for regulatory approval in the US, UK, and Europe by the end of this year, meaning the drug could receive marketing authorization sometime in 2023.
Upward of 60 gene and cell therapies are projected to reach regulatory approval in the US by 2030, according to the MIT NEWDIGS collaborative, as highlighted by Orchard Therapeutics. |