Listing The 10 Stocks For The Gene Therapy Revolution

Introduction

Even though most gene-editing stock prices have experienced large declines over the past few months, it is still anticipated that the genome-editing industry will expand at a compound annual growth rate of 53.1% from 2018 to 2027, reaching a total of $59.4 billion. Following is a list containing the top seven stocks in the gene-editing business that Bank of America has determined to be among the most valuable.

Modern biotechnology is cutting edge in cutting-edge topics like gene therapy and gene editing. Gene Therapy Revolution is a method of treating genetic problems that involve introducing genetic material at the cellular level. A functioning copy of a gene is often added to a patient's genome through gene therapy. The therapeutic purpose of gene editing is to modify an organism's DNA sequence.

Gene therapy is one of the topics that are quickly increasing at the forefront of modern medical research. In this therapy, defective genes that cause genetic or inherited disorders are either fixed or replaced. This particular subsector of the biotechnology industry contains a diverse range of company sizes, which provides investors with opportunities in three primary categories:

• Companies that are desirable in and of themselves.
• Companies that are likely to be acquired by another business.
• Companies that are expanding through mergers and acquisitions.

Intellia Therapeutics

Jennifer Doudna is one of the co-founders of Intellia, a company established in 2014. Recent clinical data that was published by Intellia and its partner, Regeneron Pharmaceuticals (NASDAQ: REGN), demonstrated the safety and efficacy of NTLA-2001 CRISPR-Cas9 gene editing in vivo (in the body) for the treatment of ATTR amyloidosis, a "protein misfolding disorder" that can be debilitating and frequently fatal. This treatment may also be successful in treating disorders like Alzheimer's, Parkinson's, and Huntington's; all brought on by improper folding of proteins.

CRISPR Therapeutics

Emmanuelle Charpentier co-founded CRISPR in 2013, and she did so with the intention of "creating transformational gene-based therapies for critical human diseases." As partners with Vertex Pharmaceuticals (NASDAQ: VRTX), CRISPR can develop life-saving drugs expecting to be the first to market with the groundbreaking CTX001 medicine by using CRISPR-Cas9 gene editing ex vivo (out of the body) Sickle cell anemia treatment cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT).

These blood illnesses are perfect candidates for treatment with CRISPR procedures due to the ease with which blood stem cells may be isolated, altered, and reintroduced into patients. Vertex paid CRISPR $900 million in the second quarter of 2018 based on the outcomes of the most recent clinical trials, with the possibility of an additional $200 million awaiting regulatory approval. As a result of this trade, Vertex's share of future CTX001 profits increased from 50% to 60%.

Editas Medicine

Editas Medicine is at the forefront of the industry in converting the potential of CRISPR gene editing into developing a solid pipeline of medications. At this time, the company's primary focus is on eye care, namely the creation of gene-editing medicines to eradicate inherited retinal illnesses such as blindness. The price per share of the company's stock has climbed by more than forty per cent since the first of June.

Illumina

Cancer and other complicated disorders are the subjects of genetic research carried out by Illumina. Illumina is without peers regarding NGS (also known as next-generation sequencing). ILMN stock is intriguing for several reasons, one being that industry analysts foresee a rapid expansion of the market for DNA sequencing. Scientists will better understand transmission dynamics, mutation rates, and the process of creating vaccines thanks to NGS. Whether it is COVID-19 or cancer, our company possesses the knowledge necessary to decode their genetic make-up.

Pacific Biosciences of California

Another company that is doing intriguing things in the DNA sequencing market is Pacific Biosciences of California, based in California (PacBio). The company's flagship product is known as Sequel, a nucleic acid sequencing technology based on single-molecule real-time sequencing (SMRT). In contrast to the process used by Illumina, which requires cutting and reassembling DNA, PacBio's approach, also referred to as long-read sequencing, includes the use of lasers to read a sequence multiple times in order to guarantee its accuracy.

Significance For Investors

According to Barron, around 5,000 uncommon diseases can be traced back to a single mutation or fault in an individual's genes. In theory, treatment is possible for each disorder with a targeted medicine. Because of the high prices, even though many of these diseases only affect relatively tiny populations, a successful therapy might still bring in over one billion dollars annually, even if it soles to a limited number of people.

Even though gene therapy has been the subject of research for many years, the field is still very much in the experimental stage, and widespread commercialization of the practice is still quite a ways off. Spark Therapeutics possesses the only gene-replacement therapy that has been given the go-ahead by the Food and Drug Administration (FDA). This therapy treats a rare eye ailment that can lead to blindness. It was only able to make sales of $27 million in 2018, which was its first full year on the market.