Almost a decade ago, the world was introduced to a technology that pretty much revolutionized the realm of biotechnology. The concept paved the way for some inimitable inventions and discoveries that literally blew the mind. The idea was so unthought-of before that it took around a decade to fit into the norms. Allow us to disclose the one-of-a-kind technology we are talking about currently. Yes, it is CRISPR technology.
Based on the several factors behind the prevailing hype of this cutting-edge technology, a recent report published by MarkNtel Advisors reveals that the Global CRISPR Gene Editing Market may register a massive upsurge with a CAGR of around 18.24% in the historical period of 2022-27. Before you feel puzzled by this abbreviation, allow us to decode for you, not just the term but also the other aspects of the CRISPR technique. So, stay till the end to disentangle the threads one by one.
What Is The CRISPR Technique, And How Has It Got Evolved?
CRISPR stands for Clustered Regularly Interspaced Short Palindromic Repeats, a family of DNA sequences found in the genomes of prokaryotic organisms such as bacteria and archaea. These sequences intend to detect and destroy similar DNAs, derived from DNA fragments of bacteriophages involved in infecting prokaryotes, whenever encountered again. Further simplifying, the technique imitates the naturally occurring genome editing technique adopted in the bacterial defense system.
Hence, when experiencing a viral infection, bacteria create CRISPR arrays into their DNA out of the sequence derived from the viruses’ DNA. It is to retain familiarity with such viruses so that in case of further attack, the bacteria produce RNA segments from these arrays. The bacteria then use Cas9 or a similar enzyme to cut the DNA apart, ultimately disabling the virus. It is how the defense system enables better immunity against such a virus. This naturally occurring defense mechanism forms the basis of genome editing technology referred to as CRISPR-Cas9, in genome engineering. Apart from -Cas9, the technology has a few other variants like -CPF1, etc., available in the market.
How Has CRISPR-CAS9 got the Upper Hand Over Earlier Gene Editing Technologies?
Unlike contemporaneous tools, exhibiting the need for separate cleaving enzymes, the CRISPR-Cas9 technique projects much broad utility. Thanks to its autonomous nature helpful in performing the elimination of DNA strands. The technology has proven to be more efficient and makes the perfect choice when considering customization, among others. A surplus of gRNA sequences has already been actualized for research purposes. What adds to the existing benefits of CRISPR-Cas9 is its compatibility with tailor-made “guide” RNA (gRNA) sequences engineered to lead them to its DNA targets. Another prominent factor catalyzing the need for such tools is their immense potential in targeting multiple genes simultaneously.
Talking about its history, ten years ago, Jennifer Doudna, and Emmanuelle Charpentier, Ph.D., co-proposed the adoption of CRISPR/Cas9 for gene editing. It was the first time gene-editing-based therapies were waved off and soon started to gain traction. However, the concept of CRISPR was formulated way before in 1987, when an unusual repetitive DNA sequence was discovered in the Escherichia coli genome during an analysis of genes involved in phosphate metabolism. Structural biology, comparative genomics, and advanced biochemistry have paved the way for today’s genome editing tools based on CRISPR-Cas9 and other class II CRISPR-Cas systems. Now, because of the much broader availability and transformation of the latest gene editing tools into viable therapies, the industry seems to witness a significant upsurge in the coming years.
Why Is CRISPR Gaining Traction These Days?
CRISPR gene editing has witnessed popularity over the years because of its comprehensive utility in treating hereditary disorders. CRISPR gene therapies have notable benefits when it comes to treating sickle cell anemia & beta-thalassemia, says New England Journal of Medicine, 2020. Also, the ongoing clinical trial for gene correction in humans is the most sought-after among all other research. Taking an instance –
- Gene Correction in Autologous CD34+ Hematopoietic Stem Cells (HbS to HbA) to Treat Severe Sickle Cell Disease (CEDAR) clinical trial was posted in the USA, as of 2021, which is considered a first-in-human, single-arm study. The study is currently in Phase1/11 & is expected to finish by 2026.
The utility of these tools has crossed the horizon and has now entered space. You heard it right! A successful attempt to demonstrate the use of CRISPR in space has been carried out in ISS (International Space Station) by the astronauts Nick Hague, Christina Koch, and David Saint-Jacques to modify the genomes of yeast cells. Such an accomplishment in microgravity paves the way for longer-duration missions beyond earth.
Who Are the Key Beneficiaries That Anticipate the Maximum Benefits of CRISPR?
- Academic Institutions & Research Centers
- Biotechnology Companies
- Contract Research Organizations (CRO)
- Pharmaceutical & Biopharmaceutical Companies
Pharmaceutical & Biopharmaceutical companies seem to reap the maximum benefits of such innovative technologies because of the growing requisite for immediate treatment & screening of genetic disorders. And to satiate these embryonic needs, the companies are looking forward to expanding their portfolios with numerous CRISPR gene editing-based products & services. For instance,
- In 2021, Vertex Pharmaceuticals & CRISPR Therapeutics altered their partnership agreement for inventing, manufacturing, and commercializing CTX001, an investigational CRISPR/Cas-9 therapy for sickle cell disease (SCD) & beta-thalassemia.
The CRISPR technique prompts the genome editing market to dive deep and explore the immense possibilities lying dormant by attracting substantial investment to carry out further research and development in Academic Institutions & Research Centers, Contract Research Organizations (CROs), and Biotechnology Companies. For instance,
- The biotechnology companies in Brazil are shifting their focus to cell therapies, tissue engineering, small molecules & biologics, regenerative medicines, and genetic & molecular testing.
- In 2021, Merck KgaA licensed its patented CRISPR-Cas9 technology to Cellecta to develop next-generation treatments, enabling researchers to advance treatments for inherited blood disorders, ophthalmologic, and oncological disorders, among others.
Amidst the undue promises, profound dilemmas, and prolonged controversy, genome editing has beautifully evolved to the extent that even ‘space’ is not the limit. Being able to alter the genes is no less than a miracle to biotechnology. However, dealing with genetics might pose life threats and needs to be tackled wisely. Nonetheless, the need for these innovative tools in treating genetic disorders accelerates, and so is the market for gene editing technology.