The business of editing genes to battle disease is bringing in record funding
Sep 8, 2021

The business of editing genes to battle disease is bringing in record funding

Janet Lambert, CEO of the Alliance for Regenerative Medicine, discusses CRISPR, the surge of investment and the progress being made in developing treatments.

When you’re sick, you can get treated with medicine or surgery. There’s a growing field, though, that looks at our own cells as treatment-delivery systems. Many see it as the future of medicine, and that’s prompting a lot of investment in the field. This year, the industry is on track to raise more than $20 billion, a record. That’s according to the Alliance for Regenerative Medicine, an advocacy group whose members include universities, foundations and major biopharma companies like Pfizer, Bayer and Johnson & Johnson.

Janet Lambert is the group’s CEO. We asked her for an example of a treatment in this field. The following is an edited transcript of our conversation.

Janet Lambert: Physicians can take a patient’s own cells, genetically engineer them to be super-cancer-fighting cells, and put them back into a patient’s own body. We’re taking advantage of what the body already does naturally in the course of fighting off disease, taking advantage of that understanding of the body’s functioning.

Jed Kim: Now, I imagine that a lot of our listeners might have heard of CRISPR, the technology we use to actually do the gene editing. What are the latest advancements?

Lambert: CRISPR many people have heard of because the two women who played a key role in discovering CRISPR recently won the Nobel Prize for that discovery. And the technology underlies a number of companies and development programs in our space. CRISPR is often described as molecular scissors. It’s the ability to affect parts of your genome. So some diseases are caused by essentially a mistake in your genome, and if we could fix that mistake using these molecular scissors, we could get out of a world where we’re constantly treating a disease and we could get into the world of solving the underlying problem that caused the disease in the first place. So this ability to use these molecular scissors is very exciting to those seeking to develop new medicines. So what’s happening in the field of CRISPR right now is a number of developments aimed at making that cutting of the genome more precise. Just giving it capabilities beyond what the original CRISPR can do, and it seems that there is a wide-open field of possibility for how we’re going to apply CRISPR and other gene-editing technologies to the medicines of the future. 

Janet Lambert sits smiling for her headshot wearing a grey blazer and black top.
Janet Lambert (courtesy Alliance for Regenerative Medicine)

Kim: You reported that the industry raised $14 billion in the first half of the year, which is a record. How much of this is based on the extra attention that we’re all paying to biotech right now? 

Lambert: Yes, well, we certainly all are paying extra attention to biotech. I think that’s a factor. Investors are very enthusiastic, in particular about immuno-oncology or gene-modified cell therapy, because those therapies can address the majority of cancers that people face, and cancer is still such a devastating disease. And so the ability to really make a significant contribution to cancer, I think is part of what’s driving the record investment.

Kim: The [Food and Drug Administration] this week suspended testing of a gene therapy over possible connections between the treatment and cancer in mice, and I understand nothing had been proven yet but how real are these risks?

Lambert: You may be aware that last week, the FDA spent two days having a workshop on a particular gene therapy and exploring potential safety concerns. It’s early days in the field. I think the field is strongly committed to understanding, what are the things that could cause problems for patients when it comes to gene therapy? I think so far we haven’t seen indications that there is a link between gene therapy and cancer, but it’s certainly an area where a lot of attention is being focused because obviously the people in this field are in it to help cure disease and are very sensitive to any potential safety concerns.

Kim: I understand that some biotech companies are looking at using gene editing to reverse aging. Are there concerns with how this technology is going to be used? Is there a place for it when it’s not being used to treat diseases?

Lambert: There are a huge number of rare diseases and prevalent diseases that really right now have no effective treatments, so that’s really the focus of our community. Some of these technologies have capabilities that we need to understand and that we need to think about. You may remember, there was controversy about a Chinese scientist who used gene-editing techniques in utero on a pair of embryos. And I think our community came together and said, “Look, the science is not ready for that. The science is early. We don’t fully understand its applicability in that kind of a domain. We’re just not ready to go there.” So for right now, I think we’re carefully proceeding focused on nonheritable gene editing that’s focused on important unmet medical needs. And I think that’s where the field really is today.

A lab technician wearing blue scrubs and gloves holds a blood bag before genetically modifying the patient's cells using Chimeric antigen receptor (CAR) T-Cell therapy. He stands beside a white large centrifuge.
A lab technician at the Paoli-Calmettes Institute in Marseille, France, holds a blood bag before genetically modifying a patient’s cells. (Gerard Julien/AFP).

Related links: More insight from Jed Kim

Want a primer on regenerative medicine? The University of Nebraska Medical Center helped me get up to speed for this interview. If nothing else, you too can learn what compensatory hyperplasia means. You’ll be the toast of your next Zoom dinner party. Are those still happening?

You can also read more about that FDA decision to halt testing on an experimental gene-therapy treatment that’s meant to treat a rare disease called phenylketonuria. The halt came after some mice that were given the treatment developed liver cancer. The mice were given a much higher relative dose than humans would get, and there’s no evidence that such treatment causes cancer in people. Still, one market analyst said the FDA’s decision shows it’s being conservative in how it handles these treatments.

As I discussed with Janet Lambert, some companies are looking into the possibilities that gene therapy has for anti-aging. MIT Technology Review has an article on Altos Labs, a company that’s looking into “reprogramming” cells to younger states. Jeff Bezos is apparently one of the company’s investors, along with other very rich people. Oodles of money means they can attract scientists who don’t want to deal with grant proposals.

My question is, do we want to live for really long amounts of time, considering where the planet’s going? I mean, maybe these billionaires should work on launching themselves into space. Oh, right…

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