The Rise of CRISPR Technology

CRISPR technology — otherwise known as clustered regularly interspaced short palindromic repeats — is a rapidly growing component of the overarching genetics industry, which has proven to be an essential part of human genome editing.

In fact, it was only in 2012 that CRISPR was initially discovered, according to a CB Insights report, and is now working to completely transform 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 said that in early 2013, Zhang lab revealed the first method to spearhead CRISPR to edit the genome in mouse and human cells.

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Similarly, it was only just a few years ago — in 2016, to be exact — that cells modified with CRISPR-Cas9 were finally 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 having DNA-cleaving functions.

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

As you can see, the rise of CRISPR technology is still in the very early stages of development, but with a variety of clinical trials currently underway, it’s clear that CRISPR technology and all of its components will have a significant impact in 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 to the rise of CRISPR technology, its market outlook, and what trials are currently in progress.

What is CRISPR technology?

While CRIPSR technology has certainly evolved in recent years, you might still be asking what, exactly, it is. Doudna explains that bacteria and archaea have adaptive immunity against foreign genetic elements using CRISPR-Cas systems. When infected, new foreign DNA sequences are “captured” and placed into the host CRISPR locus as a new spacer.

“The CRISPR locus is transcribed and processed to generate mature CRISPR RNAs, each encoding a unique spacer sequence,” Doudna writes.

AS CB Insights puts it, CRISPR is an integral feature of the bacterial genetic code and the immune system. It works as a defense system that bacteria uses to protect against virus attacks.

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CRISPR is, in other words, a series of short repeating DNA sequences with “spacers” in between. These genetic sequences are used by bacteria to know the specific viruses that attack them, which does this by fusing a virus’ DNA into a bacterial genome. According to CB Insights, this viral DNA then becomes the spacers in the CRISPR sequence.

When a virus attacks bacteria, human DNA is merged into a CRISPR sequence in the genome’s bacteria. What this means is when the 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 the CRISPR-Cas9 genome editing technology, which is a new DNA sequence that carries a “fixed” version of a gene. CRISPR-Cas9 is otherwise known as 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 specifies of the Cas9 gene.

Broad Institute wrote that because CRISPR-Cas9 systems can cut DNA strands, CRISPRs therefore 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 their DNA targets. Broad Institute further highlights that 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 CRISPR research, it has progressed beyond basic DNA testing, CB Insights said in its report. Case in point, it was only in December 2017 that the Salk Institute created a “handicapped version” of the CRISPR-Cas9 system, which can turn a targeted gene on or off without having to edit the genome.

The report says that this process has the potential to ease concerns regarding the “permanent nature of gene editing.”

CRISPR technology market outlook

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

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

Meanwhile, a Research and Markets report estimates that the CRISPR technology market will grow from US$ 562 million in 2018 to US$ 1.72 billion in 2023, growing at a compound annual growth rate of 25 percent during that period.

Driving that growth will be an increase in funding, private investments and, of course, a rise in 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 editing and epigenome editing services. According to Research and Markets, cell line engineering will grow the fastest out of these components.

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

Already, a number of gene therapy trials are in progress, which should certainly assist in pushing forward the adoption of CRISPR technology.

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Trials, approvals and collaborations

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, most of which are taking place at hospitals. Some Chinese universities are also gearing up for trials.

That said, there are a number of other ones taking place in North America and Europe to watch for. The University of Pennsylvania began a study on September 5, 2018, which is the first human trial in the US. The trial is geared to test CRISPR’s potential to attack four conditions, including: multiple myeloma, melanoma, synovial sarcoma and myxoid/round cell liposarcoma, which are different cancer diseases. According to ClinicalTrials.gov, an estimated 18 patients will be enrolled with the study expected to complete in January 2033.

Each patient will have blood cells removed and editing will then 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, which is 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, which is a blood disorder. The study, which began in September 14, 2018, will enroll an estimated 45 patients with a CTX001 therapy, which they will receive a single infusion through a central venous catheter. The Scientist describes the therapy as treating diseases which have a deficiency in the production of hemoglobin in adults. The study will remove blood cells from the patient, then edit and replace them. This study is expected to be completed in May 2022.

Vertex Pharmaceuticals and CRISPR Therapeutics are spearheading another trial for the CTX001 therapy in patients with severe sickle cell disease. The study, which began on November 27, 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 sells that are able to self-renew and differentiate into new lineages of the blood cell system.

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Meanwhile Editas Medicine (NASDAQ:EDIT), a genome editing company, received Investigational New Drug application from the US Food and Drug Administration for its EDIT-101 in November 2018. EDIT-101 is an experimental genome editing medicine that is being investigated to treat Leber Congenital Amaurosis type 10 (LCA10). Leber Congenital Amaurosis is a disease of the eye affecting the retina that detects light and color.

Thanks to the IND acceptance, Editas Medicine received US$ 25 million from Allergan to discover and develop experimental ocular medicines targeting vision-threatening diseases. With Allergan Pharmaceuticals, a wholly-owned subsidiary of Allergan (NYSE:AGN), 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 CRISPR-Cas9-based genome editing cell therapy in stem cell population. The collaboration aims to include the ex vivo development of innovative cell therapies using Ocular stem cells.

In short, 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.

Don’t forget to follow us @INN_LifeScience for real-time news updates.

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

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