Gene Edited Twins Spawn a New Epoch in US BioPhysics

Image Credit: Augusto Zombonato

With Vertex Pharmaceuticals and CRISPR Therapeutics announcing that they had completed the rolling submission of their Biologics License Applications (BLAs) for the investigational CRISPR therapy exagamglogene autotemcel (exa-cel; previously known as CTX001) for the treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT), an insight into the exploratory pioneering done on a seemingly negligent level seemed appropriate. In order to fully understand the trajectory of events one should know that a set of human twins were genetically modified, successfully, in 2018. The renown Chinese scientist that claimed to have created the world’s first gene edited twin babies was released from a three year jail sentence a year ago. He Jiankui’s three year sentence and 3 Mn yuan (US$430,000) fine came on the heels of his initial announcement of his experiments after the birth of the twins in October of 2018. Known as a ‘rogue scientist’, Jiankui was subsequently fired from from SUSTech (Southern University of Science and Technology) in January of 2019; jailed and fined in December of that same year.

Jiankui learned CRISPR/Cas9 (Clustered Regularly Interspaced Short Palindromic Repeats/CRISPR associated nucleases) gene editing techniques at Stanford University. A technique that involves the use of gRNA as a guide for targeting RNA and DNA enzymes. It’s was originally discovered as an acquired immune response mechanism used by archaea and bacteria. Utilizing the process with higher organisms has proven useful in the case of the twins and a cloning technique known as nuclear transfer was used in the case of Dolly, one of the first cloned sheep who was born in 1996. The first cloned sheep was actually born in 1984 in Cambridge, UK. Which means our ability to artificially clone from embryonic cells has been a reality for nearly 40 years. Monsanto who spun off to Bayer, Solutia Inc. (Eastman Chemical Company), and Pfizer (Pharmacia LLC.), obtained the first CRISPR license to modify crops in 2016 from the The Broad Institute, Harvard University, and the Massachusetts Institute of Technology (MIT).

Dolly and her surrogate mother. Courtesy of The Roslin Institute/University of Edinburgh

Initially, the experiment on the twins was to prompt a mutation in the CCR5 gene, CCR5 Δ32. This was to engineer an HIV resistance to a majority of HIV strains, as seen in the Berlin Patient. The experiment had recruited couples who wanted to have children; in order to participate, the man had to be HIV-positive and the woman uninfected. Lulu and Nana were born healthy and now scientific evidence has indicated that the inactivation of the CCR5 gene may have inadvertently increased the cognition of these IVF miracles. The Chinese government put a full stop to Jiakui’s research with a full condemnation by our NIH. All this, ironically, followed by the WHO has launching a global registry to track research on human genome editing, after a call to halt all work on genome editing in 2019.

It seems that our adherence to regulation is prompted by the concept that slow growth and scarcity are more profitable and therefore more important than great strides. At least for general consumption. If the ability to create a set of humans with a failsafe against a deadly virus in conjunction with higher cognition is possible then what other punches are we holding?