Designer Humans?

Headlines called it “the CRISPR-baby scandal”…

In yesterday’s Bleeding Edge – CRISPR’d Babies issue, we revisited one of the biggest stories in the world of genetic editing since CRISPR technology was invented more than a decade ago back in 2012.

In 2018, He Jiankui and his team in China used CRISPR-Cas9 technology to edit human embryos in an effort to make the resulting children immune to HIV. This is something known as germline editing, and it’s been off-limits to the international scientific community.

I wrote about these developments in The Bleeding Edge back in 2018 and 2019 when it happened. Despite the ethical issues surrounding the trial, the scientific community was still curious about the technology and whether or not it had worked.

Three children were born whose embryos had been edited.

It was so early in the development of CRISPR technology. No one could have known the outcome… As I said yesterday, He and his team were rolling the dice with the lives of three children on the line. There simply hadn’t been enough research and testing, which is why the experiment was so reckless.

And yet, looking back over the six years since the trial, there has been incredible progress with CRISPR genetic editing technology and similar approaches to correcting unwanted genetic mutations.

And with each year that has passed, the confidence in using this “tool” has grown.

Venture capital has been eager to fund genetic editing startups. Several renowned CRISPR-related companies have gone public. Therapeutic pipelines are full of potential to provide cures for diseases of genetic origin.

The industry has discovered that different enzymes can be used for different therapeutic applications. There is a clear path towards optimizing the technology for each disease to make the therapies both safe and effective.

And it’s not just the new enzymes being proven to be effective for different therapeutic applications. Additional techniques like prime editing, and base editing have also been developed building upon the foundation of CRISPR genetic editing technology.

Regulatory Approvals

Aside from the incredible research and development, there have also been concrete milestones for CRISPR technology. Specifically, there have even been the first regulatory approvals of CRISPR-based therapies. To summarize:

• On November 16, 2023, the United Kingdom Medicines and Healthcare Products Regulatory Agency (MHRA) approved a CRISPR-Cas9-based therapy for the treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT) from Vertex and its partner, CRISPR Therapeutics (CRSP). This was the first CRISPR genetic editing therapy approved in history.
• On December 8, 2023, the U.S. Food and Drug Administration (FDA) approved the same CRISPR therapy – Casgevy – for the treatment of sickle cell disease for patients 12 years and older. This was the first CRISPR genetic editing therapy approved in the U.S. And I fully expect approval will be forthcoming for transfusion-dependent beta thalassemia.
• On December 15, 2023, the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion of the same CRISPR therapy for the treatment of sickle cell disease and transfusion-dependent beta thalassemia.
• On February 13, 2024, the European Commission approved Casgevy for the treatment of patients 12 and older with severe sickle cell disease and transfusion-dependent beta thalassemia.

While Vertex (VRTX) received most of the attention for the approvals, the company behind the development of Casgevy is CRISPR Therapeutics and its CRISPR-Cas9 genetic editing technology which it has since licensed from Editas Medicine (EDIT).

Aside from the incredible improvement these approved therapies will have on those who suffer from sickle cell disease and transfusion-dependent beta thalassemia, this is a sign of something much larger…

CRISPR genetic editing works… just as I predicted it would back in 2015.

The implications for the human race are extraordinary. We now have a set of tools we can use to “correct” all genetic mutations that cause human disease.

The field is wide open right now with countless biotech companies working towards the next round of regulatory approvals for genetic editing therapies.

The Future of Genetic Editing

But what about the future? What about the question of whether or not there will be the editing of embryos to eliminate unnecessary diseases and suffering?

He Jiankui was asked that question. His reply was…

I would say in 50 years, like in 2017, embryo gene editing will be as common as IVF babies to prevent all the genetic diseases we know today. So babies born at that time will be free of genetic disease.

If we assume that the technology is proven to be safe and effective, with no off-target edits or unwanted side effects, his answer is a logical conclusion.

But the far more controversial subject is whether or not this kind of technology should be used for designing “better” humans.

Just like it is possible to edit DNA to eliminate a certain disease, it’s also possible to make edits capable of improving strength, speed, intelligence, and even something as vain as eye color.

He Jiankui was asked about whether or not that kind of work had already started. His reply might come as a shock:

There are people from somewhere, an island in Honduras or maybe some small East European country, inviting me to do that. And I refused. I refused.

When asked, point blank, whether or not that kind of research is happening, he refused to offer any insights. His response was simply, “I’m not answering that.”

It Goes Beyond Disease

Given the human desire to always be improving, to outperform past generations, and to keep stretching the limits of human potential, I strongly believe that this kind of research is happening.

Is it really that much of a stretch to believe that a nation-state is researching how to make its soldiers stronger to improve military performance? Or using the technology to make its state-backed scientists and engineers smarter than the previous generation?

I don’t think it’s a stretch at all.

And I’m sure that we all know a few parents who would seemingly go to any length to give their kids a leg up or competitive advantage in life. Is it crazy to think that they wouldn’t consider this kind of technology for their future children?

I couldn’t help but be reminded of a magazine cover from Time about 5 years ago:

Source: TIME

The report tackled some of these issues, despite CRISPR technology having not been invented until 2012.

This is a subject that we’ll soon be hearing a lot more about. With each genetic editing therapy approval, confidence will build for leveraging the technology to improve the overall human condition.

And the discussion will inevitably extend beyond just disease.

And unlike He’s prediction about 2074, it won’t take anywhere near 50 years before that happens.