The Rise of CRISPR Technology

CRISPR technology is a rapidly growing component of the overarching genetics industry, and has proven to be an essential part of human genome editing.

It was only in 2012 that CRISPR was initially discovered, according to a CB Insights report, but it is now completely transforming how some of the world’s biggest problems are solved. Cancer, food shortages and organ transplants are just some of the areas CRISPR technology is working to evolve.

Broad Institute states that in early 2013, Zhang Lab revealed the first method to spearhead CRISPR to edit the genome in mouse and human cells.

A few years later — in 2016, to be exact — cells modified with CRISPR-Cas9 were injected into a human subject. CRISPR-Cas9 gene editing systems are comprised of short, non-coding RNA molecules as the DNA-binding domain and Cas9 enzymes that have DNA-cleaving functions.

In October 2018, it was revealed that a new CRISPR tool can open up more of the genome for editing. According to a report, researchers were able to detect a Cas9 enzyme that can target nearly half the locations on the genome, “significantly widening its potential use.”

While CRISPR technology is still in the very early stages of development, it’s clear that it will have a significant impact on real-world applications. As Doudna Labs puts it, “CRISPR-CAS9 gene-editing strategies have revolutionized our ability to engineer the human genome for robust functional interrogation of complex biological processes.”

On that note, here the Investing News Network provides an overview of the rise of CRISPR technology, its market outlook and what trials are currently in progress.

What is CRISPR technology?

What exactly is CRISPR technology? As CB Insights puts it, CRISPR is an integral feature of the bacterial genetic code and the immune system. It is a defense system bacteria use to protect against virus attacks.

Put more technically, CRISPR is a series of short repeating DNA sequences with “spacers” in between. Bacteria use these genetic sequences to understand the viruses that attack them; they do this by fusing a virus’ DNA into a bacterial genome. The spacers in a CRISPR sequence are made of this viral DNA.

When a virus attacks bacteria, human DNA is merged into a CRISPR sequence in the genome’s bacteria. What this means is that when a virus attacks, it will be remembered by the bacteria and will send RNA and Cas to find and destroy the virus.

This is where CRISPR-Cas9 comes in. Bacterial defense systems form CRISPR-Cas9 genome editing technology, which is a new DNA sequence that carries a “fixed” version of a gene. CRISPR-Cas9 is a Cas variety that is used to cut both human and animal DNA.

In order for Cas9 to function, it also requires a PAM sequence, which is a specific protospacer-adjacent motif. The PAM sequence is dependent on the bacterial specifics of the Cas9 gene.

Broad Institute states that because CRISPR-Cas9 systems can cut DNA strands, they don’t need to be paired with a separate cleaving enzyme. However, they can easily be paired with a guide RNA (gRNA) sequence that “leads” to DNA targets.

The institute further highlights that CRISPR-Cas9 can “identify and modify ‘typos’ in the three-billion letter sequence of the human genome in an effort to treat genetic disease.”

Thanks to advancements in research, CRISPR has progressed beyond basic DNA testing. Case in point: It was only in December 2017 that the Salk Institute created a “handicapped version” of the CRISPR-Cas9 system. It can turn a targeted gene on or off without having to edit the genome.

This process has the potential to ease concerns regarding the “permanent nature of gene editing.”

CRISPR technology market outlook

With CRISPR technology still in its early days, there is no sure way of knowing just how significant its impact will be. What is clear, however, is that it’s undoubtedly promising.

A report from Kalorama Information projects that the CRISPR-Cas9 technology market will increase 33.7 percent between the forecast period of 2017 to 2023, growing from US$779 million to US$5.2 billion.

North America and Europe will account for 70 percent of revenues, but other areas will showcase faster revenue growth for longer periods of time.

Similarly, a Research and Markets report estimates that the CRISPR technology market will grow from the US$562 million it reached in 2018 to US$1.72 billion in 2023, increasing at a compound annual growth rate of 25 percent during that period. Driving its growth will be higher levels of funding, private investment and, of course, a rise in the adoption of CRISPR technology.

The fastest-growing component during the forecast period will be the CRISPR services segment, which includes gRNA design and vector construction, cell line engineering, screening services and mediated transcriptome and epigenome editing services. Cell line engineering will grow the fastest of all.

In terms of market share, the biomedical applications segment will be the largest over that growth period; it includes gene therapy, drug discovery and diagnostics.

CRISPR technology trials and approvals

According to ClinicalTrials.gov, there are currently nine trials that are in the recruiting or not yet recruiting stage. Six of them are based in China and most are taking place at hospitals. Some Chinese universities are also gearing up for trials.

There are a number of trials taking place in North America and Europe as well. The University of Pennsylvania began the first human trial in the US in September 2018.

The trial is geared at testing CRISPR’s potential to attack four cancer diseases: multiple myeloma, melanoma, synovial sarcoma and myxoid/round cell liposarcoma. According to ClinicalTrials.gov, an estimated 18 patients will be enrolled, with the study expected to complete in January 2033.

Patients will have blood cells removed, and editing will delete genes in the T cells. These edits will remove gene codes for PD-1 protein and provide T cells a receptor for NY-ESO-1, a protein found on tumors.

The second notable trial is a collaboration between Vertex Pharmaceuticals (NASDAQ:VRTX) and CRISPR Therapeutics (NASDAQ:CRSP). This Phase 1/2 study is to treat thalassemia, a blood disorder.

The study, which began in September 2018, enrolled approximately 45 patients with a CTX001 therapy; they will receive a single infusion through a central venous catheter. It will remove blood cells from patients, then edit and replace them. It is expected to be completed in May 2022.

Vertex Pharmaceuticals and CRISPR Therapeutics spearheaded another trial for the CTX001 therapy in patients with severe sickle cell disease. The study, which began in November 2018, will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic and progenitor cells (hHSPCs) using the therapy.

hHSPCs are comprised of a rare population of tissue-specific cells that are able to self-renew and differentiate into new lineages of the blood cell system. Primary completion is expected for early next year, while the entire trial is forecast to come to a close and provide results in May 2022.

Editas Medicine (NASDAQ:EDIT), a genome-editing company, had its investigational new drug (IND) application for EDIT-101 accepted by the US Food and Drug Administration in November 2018. EDIT-101 is an experimental genome-editing medicine that is being investigated to treat Leber Congenital Amaurosis type 10, an eye disease.

Thanks to the IND acceptance, Editas Medicine received US$25 million from Allergan (NYSE:AGN) to discover and develop experimental ocular medicines targeting vision-threatening diseases. With Allergan Pharmaceuticals, a wholly owned subsidiary of Allergan, 10 to 20 patients will enroll in a Phase 1/2 open-label study to evaluate EDIT-101.

EDIT-101 is given through a subretinal injection to reach and deliver the gene-editing tool directly to photoreceptor cells. While the company was given IND in November, there has been no word on when the company expects to begin enrolling patients.

Finally, Intellia Therapeutics (NASDAQ:NTLA) and Novartis revealed a collaboration in December 2018 to pursue a genome-editing cell therapy based on CRISPR-Cas9. The collaboration aims to include the ex vivo development of innovative cell therapies using ocular stem cells.

The CRISPR technology market is still very much in its infancy. However, with clinical trials already in development and with more on the way, the opportunity for investors is present in this growing market.

This is an updated version of an article first published by the Investing News Network in 2019.

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Securities Disclosure: I, Nicole Rashotte, hold no direct investment interest in any company mentioned in this article.