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In 2020, American biochemist Jennifer Doudna and French microbiologist Emmanuelle Charpentier were awarded the Nobel Prize in Chemistry for their discovery of the CRISPR gene-editing technology. The Royal Swedish Academy of Sciences summed up the significance of their discovery by saying, “This year’s prize is about rewriting the code of life.”
Gene-editing companies using CRISPR technology have the potential to treat and even cure diseases caused by genetic variants. The gene-editing market is projected to reach $15 billion by 2028.
These are some of the gene-editing companies investors should have on their watch list:
||$11 billion||Developing CRISPR-Cas9 gene editing for protein disorders.|
||$9 billion||Developing CRISPR-Cas9 gene editing for blood diseases.|
||$7 billion||Developing base editing for blood, liver, and ocular diseases.|
||$4 billion||Developing CRISPR gene editing for ocular, blood, and oncology purposes.|
Intellia was co-founded by Jennifer Doudna in 2014 with a mission to “develop potentially curative gene editing treatments that can positively transform the lives of people living with severe and life-threatening diseases.”
Intellia and its partner, Regeneron Pharmaceuticals (NASDAQ:REGN), recently made headlines with clinical data showing NTLA-2001 CRISPR-Cas9 gene editing in vivo (in the body) was safe and effective in treating ATTR amyloidosis, a debilitating and usually fatal "protein misfolding disorder.” The treatment may hold promise for other protein misfolding disorders, including Alzheimer's, Parkinson's, and Huntington's diseases.
In Q2, Intellia raised $690 million in a stock offering and ended the quarter with $551 million on its balance sheet. The offering will help fund the company’s pipeline, including hereditary angioedema, acute myeloid leukemia, and other research-stage programs.
Intellia’s net loss in Q2 was $68.8 million, compared to $32.4 million during Q2 of 2020.
CRISPR was co-founded by Emmanuelle Charpentier in 2013 with a mission of "developing transformative gene-based medicines for serious human diseases." CRISPR and its partner Vertex Pharmaceuticals (NASDAQ:VRTX) are hoping to be first to market with the groundbreaking CTX001 therapy using CRISPR-Cas9 gene editing ex vivo (out of body) to treat sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).
These blood disorders are particularly good targets for CRISPR techniques since blood stem cells can be readily harvested, edited, and reintroduced into patients. Based on the latest clinical results, Vertex paid CRISPR $900 million in Q2, with the potential for an additional $200 million upon regulatory approval. In return, Vertex increased its share of CTX001 future profits from 50% to 60%.
With a Q2 2021 cash burn of $141 million, the $2.6 billion in cash on CRISPR’s balance sheet should fund years of R&D.
Beam was co-founded by David Liu and Feng Zhang in 2017 with a vision of “providing life-long cures to patients suffering from serious diseases.” Beam is pioneering the use of base editing -- a potential new class of precision gene editing described as a “molecular pencil” able to erase and rewrite a mutation rather than slicing it out.
Beam is concentrating its efforts on blood diseases, liver diseases, ocular diseases, and oncology. Beam anticipates filing an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA) for BEAM-101 to treat sickle cell disease (SCD) by using gene editing to make changes in Hereditary Persistence of Fetal Hemoglobin (HPFH). In the second half of 2021, Beam plans to start IND-enabling studies for BEAM-102 to directly correct the genetic mutation causing SCD.
In Q2 2021, Beam’s net loss was $76 million, with cash on the balance sheet of $615 million.
Editas was co-founded by Feng Zhang, George Church, J. Keith Joung, and David Liu in 2013 with a mission to “translate the power and potential of genome editing into a broad class of gene edited medicines that transform lives of people living with serious diseases.”
Editas is focused on using CRISPR-Cas9 and CRISPR-Cas12a technology to treat ocular diseases, blood diseases, and oncology. Editas is in clinical trials testing EDIT-101 in adults and children to treat Leber Congenital Amaurosis 10 (LCA10), which causes severe visual impairment. Editas plans to begin clinical testing EDIT-301 by year-end using CRISPR/Cas12a to mimic Hereditary Persistence of Fetal Hemoglobin to treat sickle cell disease.
In August, Editas presented data on a new gene-editing technology termed SLEEK (SeLection by Essential-gene Exon Knock-in). Editas management believes SLEEK may enable the development of next-generation cell therapeutics for cancer.
In Q2 2021, Editas had a net loss of $55 million, with cash on the balance sheet of $698 million.
The four companies were started by clinical researchers to capitalize on the advancements in CRISPR gene-editing technology. CRISPR (pronounced “crisper”) is an acronym for Clustered Regularly Interspaced Short Palindromic Repeats. The name describes the DNA sequences spelling out the genetic code that guides the development of life.
CRISPR is based on the immune system that bacteria have developed to thwart viruses. The technique has been described as a “molecular scissor” honed in the battle of bacteria vs. virus over millions of years. When attacked by a virus, bacteria are able to target, slice, and destroy the DNA of the virus.
In 2012, American biochemist Jennifer Doudna and French microbiologist Emmanuelle Charpentier studied this phenomena and discovered how "CRISPR-associated" (Cas) proteins could be used to slice strands of DNA. At the same time, RNA molecules made in a lab could guide Cas proteins to exactly where to cut the DNA. The technique works in any cell and opens the possibility for gene editing in humans, plants, and animals.
The medical implications of CRISPR-Cas9 human gene editing are profound. The door is now open for using gene editing as a “one-and-done” treatment for a wide range of deadly and debilitating genetic conditions. In his recent New York Times best-seller The Code Breaker, author Walter Isaacson described CRISPR as changing "the future of the human race." (If you're unfamiliar with the book, you might start by watching Fool.com healthcare bureau chief Corinne Cardina interview Isaacson on Fool Live.)
These gene-editing companies have the potential to be very successful. Both Intellia and CRISPR have found large pharmaceutical partners to help fund development and usher the new treatments to market. Both Beam and Editas are pursuing innovations that meaningfully address some of the challenges and shortcomings in CRISPR-Cas9.
Many investors find gene-editing companies complicated. Other investors find them too volatile, with prices stagnating for long periods then moving strongly up or down depending on publicity about new research findings or the outcome of clinical trials.
One approach for starting to invest in gene-editing stocks is to consider a basket strategy of putting money into several companies and watching them progress over several years. Billions in market value will be created by companies with the ability to cure diseases.
For patient, buy-and-hold investors, gene-editing companies provide an opportunity to expand your portfolio during the coming genomic revolution.
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