Gene Editing: What is CRISPR and its Specialty Impact?

Specialty Pharmacy Times, January/February 2016, Volume 7, Issue 1

CRISPR technology may eventually hit the specialty landscape with promising treatments for HIV and cancer.

BIOPHARMA HAS BECOME A LARGE PART OF THE HEALTH CARE MARKET, following an explosion of new therapies to hit the market last year. However, nothing has been discussed recently as much as the rapid ascent of the technology called CRISPR, a new gene editing tool.

The purpose of this column is to describe the basics of gene editing in order to show specialty stakeholders why they should be aware of this growing trend. What is gene editing? It is the process of targeting DNA in the genome and changing a sequence in order to transform it.

More specifically, the gene is snipped like a reel of film and then spliced back together with a different protein to create a modified version that fights off a disease or creates immunity. Gene editing has been around in some form or another since the 1970s.

Advances in mapping the human genome have brought about more experimentation and targeting of certain genes. Two gene editing tools—zinc finger nucleases and transcription activator—like effector-based nucleases—have been in use for some time. The newest gene editing tool to be discovered is CRISPR-Cas9.

CRISPR stands for Clustered Regularly Interspaced Short Palindromic Repeats, and Cas9 is a protein that makes it all work. This new method is easier to use, cheaper, and more precise than its predecessors. CRISPR is found in bacteria and is used to fight off viruses.

In nature, bacteria use CRISPR sequences as a reference tool when confronted by a virus it has fought before. The bacteria then use CRISPR to inactivate the virus by naturally splicing it. The CRISPR-Cas9 system mimics this process, but then goes a step further by using the Cas9 protein to remake a gene.

Unlike other gene editing tools, CRISPR uses RNA, which makes the system easier to use and more precise. CRISPR has already been used to create cells immune to AIDS and has stopped cancer cells from multiplying in research labs. Soon, these new findings will move from the lab to the health care system in order to help find a cure for such diseases.

Currently, the technology is licensed to several companies and could possibly make the patent holders billions. Ongoing patent litigation between the University of California and The Broad Institute of Harvard and MIT, which may likely be decided this year, could have huge implications on the direction of CRIPSR.

Until this patent fight is complete, it remains unclear how the market will use CRISPR. The basis of the fight lies with 2 scientists, Jennifer Doudna, PhD, and Feng Zhang, PhD. Dr. Doudna published the first paper on CRISPR-Cas9 with her research partner, Emmanuelle Charpentier, PhD.

After the paper was published, the University of California applied for a patent for the gene editing tool for Dr. Doudna. Dr. Zhang subsequently published a different paper about the technique with 1 difference: he applied it to human cells. The Broad Institute then filed for a patent on behalf of Dr. Zhang and requested it be expedited.

The patent was granted to Dr. Zhang before Dr. Doudna, even though Zhang’s paper was published after Doudna’s paper. After seeing the patent, the University of California filed for a hearing with the US Patent and Trademark Office to decide who should have the patent. The hearing is expected to be heard this year.

Regardless of the patent fight, researchers are using the technology all over the world. When searching online for CRISPR, you will find dozens of ads for laboratories that will help you use the tool for only a few hundred dollars. With this much access to such a precise tool, there has been concern over the abuse of the technology.

After Chinese scientists used the technique on nonviable human embryos, the scientific community came together to discuss the ethical use of CRISPR-Cas9 at the December 2015 International Summit on Human Gene Editing in Washington, DC. The summit showed that the largest concern lies with the use of gene editing to affect the human genes of future generations.

Attending researchers did admit that the science has not yet developed to where we are creating “designer babies” or performing other questionable human engineering. At the end of the day, the scientists decided that there should be guidelines for the use of the technology and that it cannot be used to affect genes that are inherited.

At this point, because the science is clearly ahead of the law on this issue, it seems as though the scientists will continue to review and self-regulate. Currently, there is no legislation regarding the oversight of CRISPR’s use. Many researchers receive money from publicly funded sources and are wary of losing that funding.

Therefore, they want to do the most they can to stop the misuse of CRISPR and maintain their budgets. Many companies are using the same guidelines that exist for the use of stem cell research as the closest means of self-regulation. Also, there seems to be an invisible line between using embryos and nonreproductive cells.

Most of the research is being done on nonreproductive cells only. This gray area within the spirit of innovation demonstrates why it is important that the scientific community continues to monitor the evolution of gene editing use. Two major companies are using CRISPR to create new ventures in the health care market.

CRISPR Therapeutics, cofounded by Dr. Charpentier, has created several partnerships with other biotechnology firms in order to move forward with clinical trials and research for gene-based medicines. Editas Medicine, founded by Dr. Zhang, is also moving forward with the technology to treat diseases.

For now, the use of CRISPR-Cas9 as a gene editing tool looks like it is here to stay. This could mean an explosion of new drugs and therapies that could reach the market in the near future. Hence the rationale for specialty stakeholders to be aware of it.

There is no cure-all here just yet, but knowing what CRISPR-Cas9 is will help you understand these new therapies and medicines. Gene editing can sound scary, but having the knowledge in order to explain it to your patients who are on the Internet doing their own research can help alleviate anxiety.

With more patient advocacy and the coming of more right-to-try legislation, patients may come across this technique and have questions. Basically, CRISPR has already changed how gene therapy is viewed. It will be seen more and more over the years to come and will likely enter the toolkit of providers very soon. SPT

Ron Lanton III, ESQ is president of True North Political Solutions, LLC. He has over 20 combined years of government affairs and legal experience. This includes activities on the municipal, state, and federal levels of government. Most recently, he worked for a pharmaceutical wholesaler where he created and oversaw the company’s government affairs department, served as their exclusive lobbyist, and advocated for the company’s various health care customers. Prior to that, Ron worked at a government affairs consulting firm in Arlington, Virginia, where he focused on health care, energy, commerce, and transportation issues. He has also clerked for a federal magistrate, was appointed as a municipal commissioner on environmental issues, and has served as consultant to Wall Street firms on financial issues. He has been a featured industry speaker on issues such as pharmaceutical safety and health care cost containment. Ron earned his juris doctor from The Ohio State University Moritz College of Law and a bachelor of arts from Miami University of Ohio. He is also a “40 Under 40” award recipient. He is admitted to practice law in New York, Illinois, and the District of Columbia.