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, it would be first instance of gene-edited babies, shinning a bright light on what is possible.
It would also highlight investment opportunities.
Scientists have been excited about CRISPR since a team of Japanese researchers accidentally cloned clusters of cells at Osaka University in 1987. They discovered that bacteria used special enzymes to cut, copy and store bits of viral DNA for future reference. It 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.
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.
For all of the positives, CRISPR remains unregulated, expensive and has become something of a cottage industry. The Nuffield Council of Bioethics warned in 2016 of so-called garage scientists that 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 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 require a new set of healthcare tools.
For investors, the purest play is Illumina (ILMN) , the dominant maker of DNA sequencing machines and systems. The San Diego 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.
It's 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., 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, called AMEnDR, uses adeno-associated virus (AAV) vectors and homologous recombination to correct gene mutations.
AAVs are naturally occurring and 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 knockout, and replace an entire gene. And the AAVs can be delivered by intravenous, in one sitting. This opens up a broad range of potential uses.
The company is working on bone marrow, liver, lung, eye and the 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 November 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.
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