Emerging Tech

The Ethics of Gene Editing

Humans have long sought to transcend their biological limitations – to become stronger, faster, smarter and healthier.

But what was once just imagination is now an exciting – and, for some, scary – reality we are challenged to embrace.

That’s because gene editing technologies make the dream of transcending our genetic limitations a very real possibility.

For better or worse.

A few weeks ago, I discussed the incredible profit potential behind the growing use of gene editing technologies in healthcare.

The development of these breakthrough therapies is driven by a universal desire to treat – and potentially cure – rare diseases.

So far, we’ve seen some promising progress on that front. But the ethics of gene editing remain controversial.

Case in point: A scientist in China recently claimed to have genetically engineered twin girls with HIV resistance (with possibly a third genetically engineered person on the way).

These “designer babies” – as they are sometimes called – were genetically modified to knock out (read: pause) a key gene that’s connected to developing HIV.

You may feel that such a breakthrough is a good thing. After all, we want to find a cure for HIV/AIDS. And a preventive method would be a great too.

Here’s the problem: Scientists are uncertain whether making unproven and inheritable genetic modifications to humans is ethical or not.

Think of it this way… If gene editing goes terribly wrong, that mutation could get passed on to future generations.

It could even lead to unforeseen consequences down the road.

So careful consideration needs to be taken on what sort of gene editing is allowable. Especially since the field is still very new.

International Response

National Institutes of Health Director Francis Collins – who previously led the Human Genome Project – said, “An international moratorium should be put into effect immediately.”

The proposed moratorium is neither a blanket ban on gene editing research nor a ban on developing treatments. Instead, it’s a proposal to prohibit specifically what’s called “germline editing.”

Germline editing refers to editing inheritable human DNA. This usually takes place through methods like CRISPR-Cas9 that change the genetic code in reproductive cells.

So the proposed moratorium would prevent gene editing on inheritable DNA until international guidelines are set.

However, germline editing for research purposes – not in human subjects – would still be allowed.

Dr. Collins isn’t alone in this belief.

A few weeks ago, the international science journal Nature published a commentary – backed by 18 scientists – proposing a global ban on germline editing until clear international guidelines are set.

One of those scientists was Emmanuelle Charpentier – one of the creators of the CRISPR-Cas9 methodology.

Risk and Reward

I’ve argued that early investment in the gene editing treatment space is bound to produce sizable returns over the coming years. I’d say this is indisputable.

But with all new innovations – especially those that bring with them uncharted ethical problems – we have to be aware of legislative responses to the evolving science.

(Just as we’ve watched the legislative progress in the North American cannabis industry.)

Even so, investors have something positive to look forward to.

In just the last year, the Food and Drug Administration has given the green light to research on a couple of CRISPR-based treatments.

Editas Medicine (Nasdaq: EDIT) submitted an investigational new drug application to begin a clinical study on a treatment for Leber congenital amaurosis type 10 (LCA10) – a rare genetic disorder that causes blindness – and it was approved in November.

More recently, CRISPR Therapeutics (Nasdaq: CRSP) and Vertex Pharmaceuticals (Nasdaq: VRTX) jointly announced the world’s first patient had been successfully treated with CRISPR therapy.

The bottom line: In spite of the ethical questions being raised, the benefits gene editing can unleash will make the journey worthwhile.

Good investing,

Anthony