CRISPR & China’s “Designer Babies” – The Future, or an Ethical Dilemma?

CRISPR & China’s “Designer Babies” – The Future, or an Ethical Dilemma?


Shannon Jones: Let’s talk a little bit more
on the topic of transparency and risk. For many of the healthcare investors that
listen to this show, I’m sure your newsfeed has been slammed with
news about designer babies. Simon, designer CRISPR babies. What do you say to all of this?
Simon Erickson: Holy, cow! This is opening
Pandora’s box, Shannon. This is a science that has
been around technically since the mid-90s. That’s when the first abstracts and papers
were coming out for what became CRISPR. It’s basically gene editing. You’re taking the DNA strand, you’re snipping
out small pieces of it, and you’re removing or editing genes. In terms of the science itself, the reason
I say this is Pandora’s Box being opened is because the name He Jiankui, scientist in
China, has now not only genetically engineered a human embryo, but the mother actually gave
birth to twins with this genetically-engineered embryo. We have a genetically-engineered baby that
has been born. This is not just animal testing anymore. This is now very much more real than just
the lab work that was being done before. This is now a science that can be incredibly
innovative, incredibly progressive, and prevent a lot of genetic diseases, but that’s counterbalanced
because this is also a very controversial question. It’s not black or white. It’s not a good or a bad thing, but a lot
of people are taking opposition to what’s being done and questioning whether or not
it was even legal for this scientist to do this in the first place. Jones: Exactly. I believe he was actually Stanford trained,
and as you mentioned, went back to China to go back and build up biotech,
build up some companies there. With this CRISPR, one of the advantages of
using CRISPR is that it really is a do-it-yourself kit. We even have high school students that are
doing experiments with CRISPR at their schools, which is kind of insane to me. The ease of access, the ease
of use of CRISPR is so intriguing. You can see it applied in many different applications.
You can see it in agricultural biotech. You can also see it being used, and it’s starting
to be tested, here in the U.S. on human trials, not for germline editing, but for certain
diseases that are occurring in adults. One of the interesting things with this designer
baby story — it seems like new headlines come out every day — it sounds like he was
able to actually go in and do what he intended to do, or at least halfway. Basically, what he was attempting to do was
to make these babies resistant to HIV, the virus that causes AIDS. Basically, the way he wanted to go
about it was disabling a gene called CCR5. He was able to disable the gene
in one of the little girls who was just born. But, it was interesting, because in the
other little girl — of course, we get two copies of every gene — only one copy of that CCR5
gene was actually disabled. The other was not. So, the question is,
is she actually resistant to HIV? Also, CCR5 is a mutation
that already exists. There are adults that are
HIV-resistant in the world now. What’s so interesting about that is that they’re
actually more vulnerable to diseases like West Nile virus, even seasonal flu. So now, you’ve got an
immune system that is out of whack. We don’t know what will happen long-term. When these children grow up and become adults
and parents and have kids, this will pass on to their kids. And we don’t know
what that will look like. For this scientist, who kind of did this in
secrecy, nobody really knew about it until they came out at a conference and said,
“Hey, guys, I just did something incredible,” I think there’s going to be a lot more questions than
there are answers in regard to what happens with this. I do know that China actually
came out and banned germline editing. Of course, it’s illegal here in the U.S. China
finally came out and banned it… but, there’s no actual enforcement arm in China. As we talked about with regulatory infrastructure,
they don’t have a way to really monitor this. So, this is something that
could continue to happen. This could be a potential downside,
especially if you hear about off-target effects that happen in other trials in China, as well.
Erickson: It’s a huge question mark. Like you said, it is
prohibited in China legally. But people are speculating that the Chinese
government might have given Dr. He special permissions to do these experiments. You haven’t seen anything solid as far as
prosecution of what’s going to happen from this. It’s an investigation that’s
undergoing right now. You’re also seeing some of the leading IP
researchers, such as Feng Zhang down the Broad Institute in Boston, calling for a moratorium
on a lot of the IP that he’s created for CRISPR, saying, “Hey, there are risks we don’t know
about that might happen from this.” Just like you mentioned about CCR5 potentially
having babies more susceptible to West Nile virus. There are still off-target mutations. Even if it does work correctly, what’s going
to be the unintended consequences of this? And, of course, you’ve got
the slippery slope argument. What’s OK and what’s not OK?
I think it’s going to be very difficult. In fact, I’ve never even heard of a globally-agreed-upon
framework for what is permitted and what is not permitted ethically in
scientific discoveries like these. It’s going to be hard to say,
“This is OK, but we’re not going to do this. This is a medical necessity
vs. this is a designer baby.” There’s a lot of gray area there in the middle
that is, I think, going to halt the adoption. But I will counteract all of that, Shannon,
if I can say one other thing on this subject, which is, if we go back to the year 1978 and
we look at in vitro fertilization, this was something that was hugely controversial in
London, when the first baby was born from an embryo that was
fertilized in vitro, outside of the womb. Then the baby still came out totally normal. But this was being called the greatest threat
to society since the atomic bomb at the time, and the mother that actually delivered the
baby in London had to use an assumed identity because of the outrage
from the public about this. Of course, course IVF has caught on
since then. The creator won a Nobel Prize in 2010. There’s now eight million babies that have
been born IVF in the world for something that was hugely controversial 40 years ago. So, I think
that, as I’ve said, we’ve opened Pandora’s box. There’s a lot of controversy
around gene editing today. I’m not certain that that same level of controversy
is going to be there even five or 10 years into the future.
Jones: Yeah, fair enough. I think that’s a very good point. Before we dive into a couple of
stocks that we think are worth watching, Simon, would you say holistically for, let’s say, the average
investor out there who’s maybe considering investing in Chinese biotech, what would be
your words of wisdom to that person? Erickson: The biggest thing is, science is
still going to win at the end of the day. We can be excited about China’s population
that needs good medicines out there. We can be excited about the people that are
coming on board, and the companies that are being created, and the policy. All of that stuff is very good in terms of
supporting what is still founded upon good science. At the end of the day, the most important
thing is that you have really effective drugs that are based upon good technology and good
science that are curing or treating these very serious diseases. The takeaway for investors is, there are going
to be a lot of headlines that you’re going to see that contain that sizzle and the buzz
and the excitement around what’s going on in China. But at the end of the day, we still need to
look at the data from those readouts that are presented from these trials and see how
that’s compared against the standards of care that have been used for
decades in westernized countries. It’s a great opportunity, but still,
data is going to prove how this shakes out at the end of the day.
Jones: That’s right. It all comes down to data. I would add, until the Chinese infrastructure
and the regulatory reforms are really in place and we can trust it, for me, it’s just making
sure that if they’re running trials in China, how easy is it for them to set up a trial
in the western market, run those trials, and then be able to reproduce the same
clinical results that they saw in China? I want to see more U.S.-based trials. From there, assuming it’s a good opportunity,
it’s an innovative approach, and the company’s management team is clear
and transparent, you might be on the right track.


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