Tag: gene therapy

The Top Precision Medicine Stocks

If you are not aware of what precision medicine is, you should probably learn about it. Much information on precision medicine can be found on the site of the U. S. Government’s National Institute of Health National Library of Medicine.

According to the Precision Medicine Initiative, precision medicine is “an emerging approach for disease treatment and prevention that takes into account individual variability in genes, environment, and lifestyle for each person.” This approach will allow doctors and researchers to predict more accurately which treatment and prevention strategies for a particular disease will work in which groups of people. It is in contrast to a one-size-fits-all approach, in which disease treatment and prevention strategies are developed for the average person, with less consideration for the differences between individuals.

Although the term “precision medicine” is relatively new, the concept has been a part of healthcare for many years. For example, a person who needs a blood transfusion is not given blood from a randomly selected donor; instead, the donor’s blood type is matched to the recipient to reduce the risk of complications. Although examples can be found in several areas of medicine, the role of precision medicine in day-to-day healthcare is relatively limited. Researchers hope that this approach will expand to many areas of health and healthcare in coming years.

There is a lot of overlap between the terms “precision medicine” and “personalized medicine.” According to the National Research Council, “personalized medicine” is an older term with a meaning similar to “precision medicine.” However, there was concern that the word “personalized” could be misinterpreted to imply that treatments and preventions are being developed uniquely for each individual; in precision medicine, the focus is on identifying which approaches will be effective for which patients based on genetic, environmental, and lifestyle factors. The Council therefore preferred the term “precision medicine” to “personalized medicine.” However, some people still use the two terms interchangeably.

Pharmacogenomics is a part of precision medicine. Pharmacogenomics is the study of how genes affect a person’s response to particular drugs. This relatively new field combines pharmacology (the science of drugs) and genomics (the study of genes and their functions) to develop effective, safe medications and doses that are tailored to variations in a person’s genes.

Precision medicine holds promise for improving many aspects of health and healthcare. Some of these benefits will be apparent soon, as the All of Us Research Program continues and new tools and approaches for managing data are developed. Other benefits will result from long-term research in precision medicine and may not be realized for years.

Potential benefits of the Precision Medicine Initiative:

  • New approaches for protecting research participants, particularly patients’ privacy and the confidentiality of their data.
  • Design of new tools for building, analyzing, and sharing large sets of medical data.
  • Improvement of FDA oversight of tests, drugs, and other technologies to support innovation while ensuring that these products are safe and effective.
  • New partnerships of scientists in a wide range of specialties, as well as people from the patient advocacy community, universities, pharmaceutical companies, and others.
  • Opportunity for a million people to contribute to the advancement of scientific research.

Potential long-term benefits of research in precision medicine:

  • Wider ability of doctors to use patients’ genetic and other molecular information as part of routine medical care.
  • Improved ability to predict which treatments will work best for specific patients.
  • Better understanding of the underlying mechanisms by which various diseases occur.
  • Improved approaches to preventing, diagnosing, and treating a wide range of diseases.
  • Better integration of electronic health records (EHRs) in patient care, which will allow doctors and researchers to access medical data more easily.

For investors, there are a few stocks that participate in this industry.

Illumina (ILMN) makes and sells sequencing systems for for genomic analysis. The stock trades at 50 times trailing earnings and 46 times forward earnings. Earnings for the latest reported quarter were up 12%.

Vermillion (VRML) develops diagnostic tests, including genetics testing through its ASPiRA GenetiX platform for hereditary breast and ovarian cancer. The company is currently generating negative earnings.

Vertex Pharmaceuticals (VRTX) has developed gene therapy for treating cystic fibrosis. The stock has a price to earnings of 28 and a forward P/E ratio of 36.

Other companies involved in the precision medicine which are worth investigating are Biomarin Pharmaceutical (BMRN), IQVIA Holdings (IQV), and Loncar Cancer Immunotherapy ETF (CNCR).

Keep in mind that there are several industries that will be leaders in the future, and precision medicine will be one of them.

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

How to Invest in CRISPR

Did you happen to see 60 Minutes last weekend? There was a segment all about CRISPR. As a matter of fact, a while ago, I predicted that there would eventually be a feature  about CRISPR on 60 Minutes.

The acronym CRISPR means “clustered regularly interspaced short palindromic repeats”. What this means in very simple terms is that it is a technique for editing and splicing DNA much more quickly, simply, and less expensively than previously done in the past. It is a revolutionary technique that could potentially cure any genetic disease.

According to the free list of CRISPR stocks here at WSTNN.com, there are several publicly traded companies that have jumped on the CRISPR bandwagon, both big and small. Obviously, the CRISPR technology industry is at its very early stages, so there are risks involved with some of the purer plays, none of which are currently generating earnings. Here are some of the pure plays.






Editas Medicine (EDIT) is a Cambridge, Massachusetts based genome editing company, focusing on treating patients with genetically defined diseases through the development of a proprietary genome editing platform based on CRISPR/Cas9 technology. The company, which sports a market cap of a bit over $1.48 billion, and has $146 million in cash, but carries a significant amount of debt.

Intellia Therapeutics (NTLA) is another pure play CRISPR stock, and is currently collaborating with Novartis (NVS). Intellia is developing in vivo projects which target liver diseases, including transthyretin amyloidosis, alpha-1 antitrypsin deficiency, hepatitis B virus, and inborn errors of metabolism; and ex vivo relating to chimeric antigen receptor T cell and hematopoietic stem cell product candidates. The stock has a market cap of $937 million, cash of $341 million, and has no long term debt.

CRISPR Therapeutics (CRSP) is a Swiss company which is developing transformative gene-based medicines for the treatment of serious human diseases using its regularly interspaced short palindromic repeats associated protein-9 (CRISPR/Cas9). It has a market cap of $2.28 billion, cash of $239 million, and is debt free.

CRISPR is a narrow niche of the biotechnology industry, but it may become the fastest growing and most significant of all the biotech businesses. To see a list of over a dozen CRISPR companies, go to the WSTNN.com link here.

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

I Want to Live Forever Stocks

The Growth of the Anti-Aging Industry

The anti-aging industry is experiencing tremendous growth. Now valued at over $380 Billion, the industry is projected by economists to exceed $1 trillion by 2025.

Biotechnology companies are helping people to live longer than ever. This is due in part to efforts in the field of life extension science, which focuses on developing cutting-edge technologies for extending human life. Three technologies of importance in this field are DNA tests to aid in disease prevention, stem cell research, and gene therapy.

Stem cells allow fetuses to grow complete organs from a single cell. They are used in research to grow transplantable organs. In recent years, scientists have been able to grow replacement body parts and functioning organs. In certain situations, these organs have been successfully transplanted into human patients. Scientists expect that, as this technology continues to advance, the survival rate of patients needing organ transplants will increase dramatically.

One of the challenges in the efforts toward human life extension includes genetic defects that cause fatal diseases. Current research for combating these diseases includes modifying the structure of human DNA through gene therapy. Using gene therapy, scientists can repair damaged DNA by implanting genetic components into human cells.

Some people can inherit a much greater chance of contracting fatal illnesses. With DNA testing, scientists can pinpoint individual genes that cause a high likelihood of developing a specific disease. When locating these harmful genes, fatal diseases can be prevented or treated early enough to prevent death.

Industry experts predict that, at the current rate of advancement, the anti-aging industry may soon surpass that of traditional disease-treating healthcare.

One way for investors to get in on the ground floor is through stocks that are involved in stem cell research and gene therapy. We have a list of over a dozen stem cell stocks.

One example is Alexion Pharmaceuticals (ALXN), a Connecticut based company, which is involved in the development of biologic therapeutic products for the treatment of hematologic and cardiovascular disorders, autoimmune diseases, and cancer. They are collaborating with the University of Connecticut on stem cell research to treat and cure rare diseases. This $27 billion stock trades at 23.2 times forward earnings.

Cellgene (CELG) is a New Jersey company that is involved in the discovery, production, and marketing of therapies designed to treat cancer and immune-inflammatory-related diseases. The company owns patent on placental stem cell recovery. Cellgene has a market cap of $94.4 billion and has a forward price to earnings ratio of 17.3.

One other related narrow industry in this area is CRISPR. If you are not familiar with what this is, you can check out our CRISPR article.

Just be aware that many of the stocks involved in gene therapy and stem cells are very low cap companies and therefore, very speculative.

Hopefully, one or more of these stocks will provide you with great returns over a long and healthy life. Who knows? You may not live forever, but at least to the age to 200 or 300.

Disclosure : Author didn’t own any of the above at the time the article was written. No recommendations are expressed or implied.