What is CRISPR and Why Should Your Portfolio Care?

 by Fred Fuld III

What is CRISPR?

CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) is a revolutionary gene-editing technology that allows scientists to make precise changes to an organism’s DNA. It is based on a natural system that bacteria use to defend against viral infections. The CRISPR-Cas9 system, in particular, has gained significant attention and is widely used in scientific research.

The CRISPR-Cas9 technology consists of two main components: the Cas9 enzyme and a guide RNA (gRNA). The gRNA is designed to target a specific sequence of DNA, and the Cas9 enzyme acts as molecular scissors to cut the DNA at that location. Once the DNA is cut, the cell’s natural repair mechanisms come into play, which can be harnessed to introduce desired genetic changes, such as adding, removing, or modifying specific genes.

CRISPR technology has revolutionized the field of genetic engineering and holds tremendous potential for various applications, including medical research, agriculture, and biotechnology. It has opened up new possibilities for understanding genetic diseases, developing new therapies, creating genetically modified organisms, and enhancing crop characteristics, among other things. However, it is still an active area of research and there are ongoing discussions and considerations regarding its ethical, legal, and social implications.

There are a few publicly traded companies that are in the CRISPR field. The CRISPR technology industry is at its very early stages, so there are risks involved with some of the smaller companies. Here are some of the pure plays, all of which are generative negative earnings.

Intellia Therapeutics (NTLA) is a biotechnology company that was founded in 2014 with the goal of developing CRISPR-Cas9-based therapies for the treatment of genetic diseases. The company was co-founded by Jennifer Doudna, one of the pioneers of CRISPR technology, along with Rodger Novak, Luciano Marraffini, and Nessan Bermingham.

The history of Intellia Therapeutics is closely tied to the breakthrough discovery of CRISPR-Cas9 as a powerful gene-editing tool. Jennifer Doudna’s research group, along with Emmanuelle Charpentier, published a seminal paper in 2012 that demonstrated the potential of CRISPR-Cas9 for precise DNA editing. This discovery laid the foundation for the development of Intellia’s therapeutic approach.

In its early years, Intellia Therapeutics focused on establishing its intellectual property and securing licenses to key CRISPR-Cas9 patents. The company also built a strong team of scientists and researchers with expertise in gene editing, molecular biology, and drug development.

Since its founding, Intellia Therapeutics has made significant progress in advancing its pipeline of potential therapies. The company has collaborated with various partners, including Novartis, Regeneron Pharmaceuticals, and Ospedale San Raffaele, to develop and commercialize CRISPR-based treatments for a wide range of genetic diseases.

Intellia’s research and development efforts have focused on several therapeutic areas, including genetic disorders, liver diseases, and cancer. The company has been involved in preclinical and clinical studies to evaluate the safety and efficacy of its CRISPR-based therapies.

The stock has a market cap of $3.75 billion, cash per share of $10.44, and has no long term debt. The latest quarterly revenue growth year-over-year was 11.5%.

CRISPR Therapeutics (CRSP) is a biotechnology company that was founded in 2013 with the aim of harnessing the potential of CRISPR-Cas9 gene editing technology for the development of novel therapies. The company was co-founded by Emmanuelle Charpentier, Jennifer Doudna, and Rodger Novak.

The history of CRISPR Therapeutics is intertwined with the groundbreaking discovery of CRISPR-Cas9 as a powerful tool for precise gene editing. In 2012, Emmanuelle Charpentier and Jennifer Doudna published a seminal paper demonstrating the feasibility of using CRISPR-Cas9 for targeted genome editing. This discovery opened up new possibilities for gene therapy and genetic engineering.

CRISPR Therapeutics was established shortly after this pivotal discovery. The company focused on translating the potential of CRISPR technology into therapeutic applications for the treatment of genetic diseases. CRISPR Therapeutics obtained licenses to key CRISPR-Cas9 patents and assembled a team of scientists and experts in the field.

Since its founding, CRISPR Therapeutics has made significant progress in its research and development efforts. The company has collaborated with various partners, including Vertex Pharmaceuticals, to advance its pipeline of potential therapies. CRISPR Therapeutics has focused on several therapeutic areas, such as blood disorders, genetic diseases, and immuno-oncology.

The company has conducted preclinical and clinical studies to evaluate the safety and efficacy of its CRISPR-based treatments. Notably, in 2019, CRISPR Therapeutics initiated the first clinical trial in the United States for CRISPR-based gene editing in humans. The trial aimed to evaluate the use of CRISPR-Cas9 to treat patients with transfusion-dependent beta-thalassemia.

CRISPR Therapeutics continues to be at the forefront of CRISPR-based therapeutics development, leveraging the potential of gene editing technology to address unmet medical needs.

It has a market cap of $4.59 billion, is debt free and has cash per share of $23.38. Quarterly sales growth year-over-year skyrocketed by over 10,000%. This is due to the fact that sales were extremely low a year ago.

Editas Medicine (EDIT) is a biotechnology company founded in 2013 with a focus on developing transformative genome editing technologies, particularly using CRISPR-Cas9, to treat genetic diseases. The company was co-founded by Jennifer Doudna, Feng Zhang, George Church, Keith Joung, and J. Keith Joung, all prominent scientists who have made significant contributions to the development of CRISPR technology.

The history of Editas Medicine can be traced back to the breakthrough discovery of CRISPR-Cas9 as a versatile gene-editing tool. In 2012, Jennifer Doudna and Emmanuelle Charpentier published a seminal paper demonstrating the feasibility of using CRISPR-Cas9 for precise DNA editing. Feng Zhang’s lab at the Broad Institute of MIT and Harvard also independently developed the CRISPR-Cas9 system for gene editing around the same time.

In 2013, Editas Medicine was established with significant investments from venture capital firms and scientific institutions. The company aimed to leverage the power of CRISPR technology to develop novel therapeutics for a range of genetic disorders. Editas Medicine holds licenses to key CRISPR-Cas9 patents and collaborates with academic and industry partners to advance its research and development efforts.

Since its inception, Editas Medicine has made progress in developing therapies for various genetic diseases. The company has focused on specific conditions such as inherited retinal diseases, which can cause vision loss, and has initiated clinical trials to evaluate the safety and efficacy of its CRISPR-based treatments.

The company sports a market cap of a bit over $582 million, and is debt free with $4.39 in cash per share. The latest quarterly revenue growth year-over-year was 45.6%.

CRISPR, initially considered a niche within the biotechnology industry, has the potential to transcend boundaries and emerge as the preeminent and rapidly expanding force among all biotech enterprises, poised to redefine the landscape of scientific advancements and medical breakthroughs.

Disclosure: Author didn’t own any of the above at the time the article was written.