Gene-editing has become one of the top investing buzzwords.
All as gene editing shows progress in identifying and potentially curing diseases.
Plus, according to Ark Investment Management CEO Cathie Wood said, “I would have to say the biggest upside surprises are going to come from the genomic space. That’s because the convergence of DNA editing, artificial intelligence, and gene therapies, importantly CRISPR gene editing, is going to cure disease,” as quoted by The Motley Fool.
Scientists using gene editing were able to extend the lives of mice with progeria, a rare rapid-aging disease that in children typically leads to death in their teens.
Gene editing is showing promise against HIV.
Neurodegenerative diseases like Alzheimer’s and Parkinson’s could potentially become a thing of the past. There’s hope it can treat major degenerative diseases, and even fix genetic conditions like muscular dystrophy.
Even more impressive, according to Interesting Engineering, “researchers have already identified DNA errors as the cause of nearly 7,000 diseases. Thankfully, the growing world of genome editing could be the ‘spell-checker’ needed to detect and eventually fix these.”
In short, everyone should be excited about the potential here.
As added by Interesting Engineering:
“Gene editing tools like CRISPR could give scientists the keys to the DNA kingdom, allowing us to find “molecular mistakes” and remove them. According to Nicola Patron, a molecular biologist at the Earlham Institute, “We are getting to a point where we can investigate different combinations of genes, control when, where, and how much they are expressed, and investigate the roles of individual bases of DNA. Understanding what DNA sequences do is what enables us to solve problems in every field of biology from curing human diseases, to growing enough healthy food, to discovering and making new medicines, to understand why some species are going extinct.”
In short, it’s a game-changer. And it’s already having a big impact on related stocks.
Gene Editing Stock No. 1 – Editas Medicine (EDIT)
Editas Medicine is one of the top names in gene-editing with several trials. It’s working on treatments for ocular diseases such as Leber Congenital Amaurosis 10, Usher Syndrome, neurological diseases, B-Thalassemia, and cancer.
It’s also working on EDIT-301 for sickle cell disease. In fact, Editas Medicine just filed a request with the US FDA to begin a Phase 1/2 study of EDIT-301 for sickle cell disease, which could be a winner given the success of preclinical data.
With regards to sickle cell disease, it’s latest US FDA submission “is a key milestone for Editas as we continue to advance several ex vivo cell therapy medicines. This submission brings us one step closer to entering the clinic with our potentially best-in-class, transformative, and durable medicine for people living with sickle cell disease,” said Cynthia Collins, Chief Executive Officer, Editas Medicine. “This moment is very exciting for the Editas team. We know patients are counting on us, and we look forward to next steps for the clinical development of EDIT-301, including dosing sickle cell disease patients.”
Its investor presentation can be found here:
Gene Editing Stock No. 2 – Global X Genomics and Biotechnology ETF (GNOM)
One of the best ways to diversify your gene editing portfolio is with an ETF such as the Global X Genomics and Biotechnology ETF.
According to Global X, it “seeks to invest in companies that potentially stand to benefit from further advances in the field of genomic science, such as companies involved in gene editing, genomic sequencing, genetic medicine/therapy, computational genomics, and biotechnology.”
Better, at less than $26 a share, it offers diversification among top stocks like CRISPR Therapeutics, Editas Medicine, Intellia Therapeutics, PTC Therapeutics, Agilent Technologies, Sorrento Therapeutics, and dozens more. If you were to buy 100 shares of each stock in the ETF, it would cost you thousands of dollars. To buy 100 shares of GNOM costs $2,600.
That’s the beauty of an ETF – diversification at low cost.
For more information on the ETF, visit https://globalxetfs.co.jp/en/funds/gnom/
Gene Editing Stock No. 3 — Intellia Therapeutics (NTLA)
Intellia Therapeutics, Inc., a genome editing company, focuses on the development of therapeutics. It utilizes a biological tool known as the Clustered, Regularly Interspaced Short Palindromic Repeats/CRISPR associated 9 (CRISPR/Cas9) system.
This year, the company expects to submit Investigational New Drug applications to the US FDA. One is an IND for NTLA-5001 for the treatment of acute myeloid leukemia, mid-year. The other is an IND for NTLA-2002, as a possible treatment for hereditary angioedema in the second half of the new year. Approval could send the stock to higher highs.
It’s also working on treatments for transthyretin amyloidosis, hemophilia A and B, sickle cell disease, and solid tumors.
“Since our founding, we set out to develop modular platform components that could serve as the engine powering an expansive portfolio of curative therapeutics. We have paved a rapid and reproducible development path for both in vivo and engineered cell therapies to address serious genetic diseases and cancers,” said Intellia President and Chief Executive Officer John Leonard, M.D.
“Over the next 12 months, we will evaluate the clinical profile of NTLA-2001, both as a one-time treatment option for ATTR patients and as a validation of our non-viral approach to in vivo delivery. In addition, we anticipate first-in-human regulatory submissions for NTLA-5001 and NTLA-2002, at least one new development candidate and new platform innovations to create the next wave of genomic medicines. These priorities for 2021 reflect our long-term vision for Intellia: to unlock genome editing’s full therapeutic potential.”
Its investor presentation can be found here: https://ir.intelliatx.com/static-files/a57908a6-aaca-404d-9072-32471cd3d41e