Science is upstream of technology, which is upstream of markets. To understand the market of genomics, we must understand its underlying science, ethical frameworks, and the key scientists behind the powerful technology.
Gene editing via CRISPR and similar tools will undoubtedly change the world as we know it, and it will also change many industries including medicine, reproductive technologies, agriculture, just to name a few. The global genomics market size was valued at USD$33.25 Billion in 2022, and is expected to grow to USD$411.35 Billion by 2030. Within this market, the gene therapy market size was estimated at USD$8.67 Billion in 2023 and is expected to reach USD$10.12 Billion this year in 2024. Just last month, the FDA approved Casgevy to be used as a gene therapy drug for patients with sickle-cell disease. This event marked a major milestone, as Casgevy is the first FDA-approved medicine built with CRISPR gene editing technology.
I conducted an interview with the genomics scientist who had perhaps the most amount of international news coverage in the last decade: Dr. He Jiankui. In late 2018, Dr. He announced the birth of two twin girls – Lulu and Nana – whose genomes have been edited during the embryonic stage. The purpose of the experiment was to disable the CCR5 gene that enables HIV infection in humans. Gene editing human embryos violated the ethics committees’ guidelines, which caused immediate controversy within international media and academia. In 2019, Dr. He was sentenced to three years of jail time.
It has been almost six years since the initial announcement of the experiment in 2018. I had a special opportunity to speak to Dr. He to learn about the aftermath of the experiment, his personal journey as a scientific researcher, and his scientific aspirations moving forward in 2024 and beyond. I uncovered more complexities to the truth behind this newsworthy experiment, but more importantly, I believe that the results of this experiment is a zero to one moment for mankind.
In a world where we can now change our own biology at the base pair level, we will undoubtedly charter many courses where we will disagree with one another on its most ethical use. Science has always been a double-edged sword. The same technology that can erase diseases from human populations can also be used as a tool for harm, corruption, and greed. As we navigate the realm of possibilities, it is important to keep an open mind to truly understand the science and its implications for downstream uses in the global markets and our species’ future.
This transcript has been edited for length and clarity.
Cathy Tie (CT): Hi Dr. He Jiankui, it’s a pleasure to speak with you today. Can you please introduce yourself and your work?
Dr. He Jiankui (HJK): I am a genetics scientist working in China. I used to study in the United States, I received a PhD degree from Rice University in Houston Texas, and did postdoc training in Stanford University in California. I have research labs in Wuhan and Beijing, and I am currently working on gene editing to cure genetic disease and improve human health.
CT: Outside of science, what are you most passionate about in life? Do you have any hobbies that you like to do in your free time?
HJK: I love playing golf, I also enjoy walking on the beach at sunset.
CT: What inspired you to pursue a career in this field in the very beginning?
HJK: Many years ago, my mother was diagnosed with Alzheimer’s disease, and unfortunately, there are no medicines available to cure it. There are many more people who are suffering from diseases that do not have a cure, so I want to do something to change it.
CT: Can you tell us about the research that you led around Lulu and Nana that was publicized in 2018? It’s been almost six years since this research was shared with the world, how are they doing now?
HJK: Lulu and Nana’s parents are HIV infected patients and they want to have a baby, a healthy baby, a baby that is not worried about HIV any more. So we took the sperm and egg from their parents during the IVF procedure, using a tiny syringe needle to inject the gene editing formula to the fertilized egg, to change one gene, and closed the door that HIV virus used to enter human cell. We then transfer the fertilized egg from the peri dish back to their mother’s uterus, and after several months, Lulu and Nana were born. Lulu and Nana are five years old now and they are healthy and happy just like any other kids in the kindergarten. I am glad that I have helped two families using my science knowledge.
CT: How did you balance the need for progressive gene editing research with ethics and general public perception?
HJK: Science research must be transparent and open, and should be approved by an ethics committee composed of medical doctors, lawyers, patient representatives, and local resident representatives.
CT: Last month, the FDA approved a new CRISPR gene editing treatment, Casgevy, by Vertex Pharmaceuticals and CRISPR Therapeutics, for sickle cell disease. To give context to the audience, sickle-cell is caused by inheriting two bad copies of one of the genes that make hemoglobin. On top of severe symptoms, life expectancy with the disease is just 53 years and it affects 1 in 4,000 people in the US. However, sources are reporting the gene editing treatment price will be $2-3m USD per patient. First, can you tell us your thoughts on this FDA approval milestone and what it means for gene-editing based medicines? And second, do you see a future where the prices for gene therapies will be lowered, making them more accessible to patients?
HJK: The approval of Casgevy is a great success for science, but not for patients. It cost more than 2 million dollars, and few patients will be able to afford it. This drug also has significant side effects including infertility.
CT: Gene therapies aside, what are your thoughts on the current state of affairs of genomics-based reproductive technologies, such as embryo gene sequencing? How do you foresee reproductive technologies being transformed by genomics in the future?
HJK: Embryo gene sequencing such as PGT-P is not ready for clinic application. Many diseases such as diabetes are influenced by hundreds of genes, and we do not have solid science to determine the risk of diabetes by genomic information.
CT:I see. So you think it's still a little bit early for clinic use.
HJK: Yes.
CT: What are your aspirations for the next chapter of your scientific career?
HJK: I believe embryo gene editing can help us to defeat many diseases and improve human health. I have proposed a research project, using embryo gene editing to help prevent Alzheimer’s disease, so our next generation will no longer worry about Alzheimer’s. I am going to do it slowly and cautiously, make sure we comply with all local laws and the international ethics guidelines. We are going to do it in a mouse first and we have no plan to move on to human trials. At every step, we will disclose our progress in full to the whole world and post it in my personal social account on Twitter.
CT: Why focus on Alzheimer's?
HJK: As I said, my mother has Alzheimer's. So personally, I also have some high risk for Alzheimer's when I get old, and maybe my daughters are at risk of having it in the future too, and Alzheimer's has no cure. If this project is successful, perhaps Alzheimer’s disease can be completely eliminated from future generations.
CT: Wow. That would be very powerful if it’s successful – to be able to get rid of a disease in future generations. I have another question. If you could go back in time to 2018, would you have done anything differently?
HJK: I did it too quickly. One thing I did not finish is the health insurance. In the informed consent document we signed with the parents of Lulu and Nana, we agreed to buy additional health insurance for Lulu and Nana. However, after the birth of Lulu and Nana, due to too much negative media attention, no health insurance company wanted to get involved. Now, as an alternative, I am planning to set up a charity foundation in Singapore to raise money to cover any future medical expenses of Lulu and Nana.
CT: Let me know if you have a link to donations for the charity. I'd be happy to share it with interested individuals.
HJK: Thank you. That'd be great.
CT: What are some valuable lessons that you learned over the last few years that you can share with the viewers?
HJK: In the past few years, my wife and daughters were living in a hard time. In the future, I won’t let my family get into the same situation again.
CT: I'm sorry to hear that about your family. Thank you so much for answering all of my questions, Dr. He.
HJK: Thank you.
The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.
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Cathy Tie
Cathy Tie is the founder and CEO of Locke Bio, a digital health platform company that streamlines the launch of fully integrated, branded telehealth services. Cathy is recognized in Silicon Valley as the youngest founder to raise venture capital in biotech, is a Thiel Fellow, and has been recognized by Forbes in their annual 30 under 30 listing.
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