United states of America has its first case of CRISPR mediated medication treatment approved by the nation’s leading healthcare authority, the Food and Drug Administration (FDA).
What is CRISPR?
It is a technology invented to slice and potentially alter genes in the human genome. Putting simply, it is a method of editing genes to alter their functions. Science has made amazing advancements in the last decade and in my opinion, CRISPR-Cas9 is the best one yet. There are immense possibilities of this technology and we have barely scratched the surface.
How does CRISPR work?
A wealth of information is present that explicitly explains and illustrates the mechanism of CRISPR gene editing. While the aim of this article is not to introduce CRISPR as a technology and how it functions, I’d like to drop some resources that can assist in elevated understanding of this beautiful technology. Here are some quick favourite visual aids for this.
CRISPR in medicine:
This technology has been used to alter genes in cells and animals in a laboratory setting for many years now. From agriculture to disease management, the uses are unending. Especially in the field of medicine, there is so much potential. Diseases with a genetic disorder such as down’s syndrome, thalassemia, many cancers and hemophilia (to name a few) are now under the radar for treatment. Altering diseases at the genetic level has the potential to sustain a more permanent solution to the disease in comparison to the drugs and medication.
This looks so promising, has this been done in Human patients?
CRISPR-cas9 system of gene editing is a very accurate technology but caution is always aired in the use for human population. Previously, a clinical trial has been done at the University of Pennsylvania with the CRISPR edited T cells for cancer treatment. CAR T cells was genetically engineered to better identify cancer cells within the body. These cells were prepared in the laboratory and then used for the treatment for patients with certain blood and lymph cancers. 5 out of 7 patients treated showed good control of the disease which seems to be a very solid start for this treatment. This also paves way for more personalized treatment which is one of the most crucial advantages according to me as every cancer is intrinsically different and this offers a solution for this.
What’s new now?
Intella’s investigational new drug (IND) is designed to be injected into the patient directly for hereditary angioedema treatment. All the previous clinical trials show the CRISPR edited products introduced into the patients. IND is capable of targeting only the liver genome to correct the gene for Kallikrein B1, a gene responsible for causing angioedema. This is the first case of CRISPR assisted in-vivo gene editing application known to mankind and that makes it super exciting!
Fun Fact:
Intella therapeutics’s stock surged post this announcement.