The event was surreal. A Chinese researcher announced on YouTube that he had successfully modified the genes of Lulu and Nana, healthy newborn twins.
According to a Scientific American report, He Jiankui, a professor at Southern University of Science and Technology in Shenzhen, claims that he used CRISPR-Case 9 gene editing to prevent the spread of HIV. The twins’ father is HIV-positive.
If true, this would be first instance of gene-edited babies, shining a bright light on what is possible. It would also highlight investment opportunities.
Scientists have been excited about CRISPR since 1987, when a team of Japanese researchers accidentally cloned clusters of cells at Osaka University. They discovered that bacteria used special enzymes to cut, copy and store bits of viral DNA for future reference.
This was a revelation that seemed to hold so many possibilities. Yet, lab successes proved fleeting.
Two decades later, biologists Jennifer Doudna and Emmanuelle Charpentier demonstrated how an organism’s genome could be precisely edited. It changed everything.
|Illumina has become the gatekeeper of the precision medicine era, a market that’s set to see $12 billion in sales by 2024.|
Patented in 2014, CRISPR is a gene-editing technique that uses molecular scissors to precisely snip genetic code. It’s a scientific marvel.
With it, researchers have modified genes to help blind people see, cure sickle cell disease in some patients and speed up the development of numerous new drug treatments.
Yet for all the positives it offers, CRISPR remains unregulated, inexpensive and has become somewhat of a cottage industry. The Nuffield Council of Bioethics warned in 2016 of so-called garage scientists who might unwittingly create a modified organism that could kill millions of humans.
Others have worried about a rush toward designer babies. The allure of removing perceived imperfections, or otherwise changing genes to improve desired traits, may prove irresistible.
In the case of Lulu and Nana, it seems to be a bit of both.
He Jiankui says that both twins have had their genomes sequenced since birth. Neither appears to be at risk to developing HIV.
The development of CRISPR is pushing health sciences toward a more personalized form of healthcare. Precision medicine tailors treatments, practices and products to the individual patient based on their genomic sequence.
It’s less one-size-fits-all and more custom fit. It also requires a new set of healthcare tools. And one company is already supplying them …
For investors, the purest play is Illumina (ILMN), the dominant maker of DNA sequencing machines and systems. The San Diego-based company has become the gatekeeper of the precision medicine era. Its systems claim 70% market share among labs, clinics and hospitals. And it is taking steps to fully integrate its stack with cutting-edge, computational software.
Precision medicine is a market that is expected to reach $11.9 billion in sales by 2024, according to a research note from BIS Research, an independent investment research firm.
Homology Medicines (FIXX) is an investment idea on the other end of the spectrum. The Bedford, Mass., firm is building a new type of gene-editing tool based on the research of its founder, Saswati Chatterjee, a professor of Virology at Beckman Research Institute.
The proprietary technology is called AMEnDR. It uses adeno-associated virus vectors and homologous recombination to correct gene mutations.
AAVs are naturally occurring, derived from human stem cells. After entering the bloodstream, they carry a corrected DNA element directly to the cell nucleus. That element targets and attaches itself to the mutated region in the genome. At that point, homologous recombination, a natural cellular process, corrects the mutated gene.
AMEnDR has been effective with both gene therapy and editing. Corrected DNA can be engineered to fix a single mutation, or knock out and replace an entire gene.
And the AAVs can be delivered intravenously, in one setting. This opens up a broad range of potential uses.
Homology company is working on bone marrow, liver, lung, eye and central nervous system applications.
The company issued shares to the public in March 2018, raising $100 million. Homology Medicines is still unprofitable, as it invests in research and development. Losses were $14.8 million in the fiscal third quarter, reported Nov. 13, on sales of $954,000.
Despite the red ink, the stock has been on the rise. Investors are waking to the potential of gene editing, given the recent news flow — a trend that is certain to continue.
Jon D. Markman
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