Two great technologies, CRISPR is a gene-editing tool that precisely alters DNA sequences in a reversible way, while PROTACs are molecules that tag specific proteins for degradation, removing harmful proteins instead of just inhibiting them.
The first CRISPR based human therapy was successfully deployed last december: a cure for sickle cell anemia and beta thalassemia...the updates about their phase 2-3 clinical trials say that for sickle cell disease at 16 months after Casgevy treatment (which is a treatment given just once), 92% of patients (36 of 39) were still responding to the cure.
Even better for beta thalassemia: of 52 patients with at least 16 months of follow-up, 49 (94%) achieved transfusion independence for at least 12 straight months. The mean duration of transfusion independence was 31 months, with one patient reaching 59 months. Additionally, all 52 patients in the study are now transfusion-free. Two of the three patients who did not initially achieve independence have now been transfusion-free for at least a year.
PROTACs are being evaluated in a number of clinical trials at the moment, with good results it seems, but we are still waiting for a commercial product.
If you like to know where we are with these two promising tools that are being used also for prostate cancer trials, here is an interesting link:
'Substandard' Control Arms in Clinical Trials– Practice found common in prostate cancer studies
current state of Immuno therapy , how we got here and where we are going - Peter Attia
It is likely mRNA technology is going to be what cures cancer in the next 5 to 10 years
Senolytic combo Dasatinib + Quercetin
Acai juice?
