There’s a new industry that is rising, that is promising to be as disruptive as AI and blockchain, that industry is genomics. With the benefit of CRISPR, affordable human genome sequencing, and advances in data science, a handful of companies are positioning themselves to take advantage of this new advent of healthcare. We will explore what is genomics, what companies are positioning themselves for this future, and why it matters.
What is Genomics?
Genomics is the study of a person’s genes (the genome), the field reviews the interactions of those genes with each other and with the person’s environment. The genome is an organism’s complete set of DNA which programs the individual with different physical traits. Virtually every single cell in the body contains a complete copy of the approximately 3 billion DNA base pairs, or letters, that make up the human genome. With the power of data science it is now possible to study how genes interact, and in some cases edit genes in order to modify a single DNA letter, or base, in a gene.
Sequencing Human Genomes
There are multiple exponential technologies that have merged to enable the easy and cost-effective sequencing of the complete human genome. Sequencing the first human genome cost 2.7 Billion and took 13 years. Due to Moore’s Law and other exponential technologies the cost has now been reduced to $1000, and is projected to be further reduced to $100.
This means we are entering an era where anyone can have their complete human genome sequenced, in search of errors, or predispositions to certain illnesses or health issues.
Multiple companies are clamoring for a marketplace where initially consumers will demand to have their genome sequenced, and eventually, medical professionals will demand it. This is driving the force behind many of the genomic companies in the marketplace, the second driving force is the transformative power of CRISPR technology.
What is CRISPR?
To familiarize oneself with CRISPR, one must first understand that there are multiple use cases for the terminology. In biology, CRISPR refers to DNA sequences naturally found in the genomes of bacteria and other microorganisms. CRISPRs are the sequences of a crucial component of the immune system that protect bacteria from viruses. The CRISPR immune system defends itself from viral attack by destroying the genome of the invading virus. This is where CRISPR technology then enters the scene.
For popular usage and in how we will explain it, “CRISPR” is shorthand for “CRISPR-Cas9.” CRISPR in combination with a protein called Cas9 (or “CRISPR-associated”) is an enzyme that acts like a pair of molecular scissors, capable of cutting strands of DNA. While the idea of gene editing has been around since the 1950s, CRISPR has enabled a cost-effective level of gene editing with a precision that has never before been available.
Companies that are Involved in Genomics
There are currently dozens of companies that are taking advantage of genomics, we will review five of the leading companies that are publicly traded.
CRISPR Therapeutics AG (Symbol CRSP)
What sets CRSPR Therapeutics apart is the all-star team of founders, this includes Dr. Emmanuelle Charpentier whose seminal research unveiled the key mechanisms of the CRISPR-Cas9 technology, laying the foundation for the use of CRISPR-Cas9 as a versatile and precise gene-editing tool. Numerous awards have recognized her work, including the Breakthrough Prize in Life Science.
CRISPR Therapeutics is developing an efficient and versatile CRISPR/Cas9 gene-editing platform into therapies to treat hemoglobinopathies, cancer, diabetes, and other diseases.
The first therapy that they are advancing is targeting the blood diseases β-thalassemia and sickle cell disease, this has now entered clinical testing, as has the first allogeneic CAR-T program targeting B-cell malignancies. While sickle cell is a disease with an arguable small market, once the technology is mature they can advance to targeting other disease vectors.
Invitae Corporation (Symbol NVTA)
Invitae Corporation is primed to take advantage of the rapidly decreasing cost of sequencing a human genome. This is a genetic information company that specializes in providing information for genetic diagnostics, preimplantation, and carrier screening for inherited disorders, miscarriage analysis, and hereditary cancer. Basically, any big data that can be provided from sequencing the human genome, Invitae Corporation offers.
Recently, Invitae Corporation partnered with ArcherDX to bring comprehensive cancer genetics and precision oncology to patients worldwide.
For anyone searching to take advantage of a company that is sequencing human genomes, they are a market leader.
Pacific Biosciences of California, Inc (Symbol: PACB )
Pacific Biosciences of California, Inc. is a biotechnology company founded in 2004 that develops and manufactures systems for gene sequencing and some novel real-time biological observation. They’ve developed a single molecule, real-time (SMRT) Sequencing technology that delivers high consensus accuracy with unprecedented read lengths. This a new way to study the synthesis and regulation of DNA, RNA, and proteins by harnessing advances in biochemistry, optics, nanofabrication, and more.
Some of the current applications they are working on include detecting epigenetic changes during sequencing to open the door to easier exploration of DNA modification to connect genotype with phenotype. This will enable understanding the effects of epigenetics on a broad range of biological processes, including gene expression, host-pathogen interactions, environmental response, DNA damage, and DNA repair. Furthermore, it will uncover the role of epigenetics in the inheritance of traits from one generation to the next.
Twist Biosciences Corp (Symbol: TWST)
Twist Biosciences Corp manufactures synthetic DNA for clients in the biotechnology industry. They’ve designed a DNA synthesis platform to address the limitations of throughput, scalability, and cost inherent in legacy DNA synthesis methods. This allows them to achieve precision manufacturing of DNA at scale.
The high-throughput silicon platform enables Twist Biosciences to miniaturize the chemistry necessary for DNA synthesis. This miniaturization allows the company to reduce the reaction volumes by a factor of 1,000,000 while increasing throughput by a factor of 1,000, enabling the synthesis of 9,600 genes on a single silicon chip at full scale.
This is essentially an easy way for other companies to order DNA as needed.
Illumina, Inc (Symbol: ILMN)
Illumina, Inc develops, manufactures, and markets integrated systems for the analysis of genetic variation and biological function. The company provides a line of products and services that serve the sequencing, genotyping and gene expression, and proteomics markets.
The technology is currently being used to assist with COVID-19, they enable companies working around the clock to track transmission, conduct surveillance, develop therapies and vaccines for any type of viral infection.
Some of their current offerings include access to fast, high-quality, sample-to-data next-generation sequencing (NGS) services such as RNA and whole-genome sequencing services.
While we have listed some of the primary companies that are taking advantage of low-cost human genome sequencing, and gene editing, there are many other companies that are promising. The healthcare industry is ripe for disruption; investors that strategically become shareholders in some of these market leaders will be best positioned for the advent of a new industry. Investors should ensure that they become familiar with genomics, with a special emphasis on CRISPR.