The percentage of the population who suffer from genetic disorders is five percent or less. But recently, scientists have determined that as much as 20 percent of the population have disease-related genetic mutations. Given this, gene-editing techniques – notably, Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) – have garnered attention for potential treatment opportunities. And CRISPR Therapeutics AG, a leader in gene editing, appears to be paving the way for these precision medicine techniques.
While planned trials at CRISPR Therapeutics is exciting, gene editing has yet to be proven as an effective treatment. Likewise, this precision medicine approach to healthcare may pose some risks and challenges as human trials advance.
However, with over 6,000 different known genetic disorders, the potential for success is great. And companies like CRISPR Therapeutics are investing heavily on its promise as a treatment for the future.
CRISPR as a Gene Editing Treatment for Patients
The use of CRISPR for gene editing is not new. CRISPR is being used in agriculture, biofuels and other health areas to advance innovation. In this regard, CRISPR Therapeutics represents one of the original companies to explore this gene-editing technique in human healthcare. By using a viral defense mechanism found in bacteria, CRISPR can disrupt, correct or regulate disease-related genes. And by targeting specific DNA areas, CRISPR Therapeutics hopes to add this approach to the scope of precision medicine care.
At the current time, CRISPR Therapeutics is exploring ways to use gene editing in a number of human health conditions. Patients with genetic blood disorders like sickle cell disease and B-thalassemia are undergoing trials for Phase I/II. Additional areas where CRISPR Therapeutics plans to experiment with treatment include cancer, muscular dystrophy, cystic fibrosis, and others. Once there is an identification of specific gene targets, gene editing could manage the disease more effectively, if not cure it altogether.
CRISPR Therapeutics—A Company with Great Potential
CRISPR Therapeutics formed in late 2013. Since that time, the company has been extremely active in its gene editing pursuits. One of its three co-founders was Dr. Emmanuelle Charpentier, who is recognized as one of the original developers of the CRISPR technique. The development of gene-editing platforms helped form partnerships with key companies in the healthcare field.
Specifically, CRISPR Therapeutics entered a joint venture with Bayer Healthcare as well as with Vertex Pharmaceuticals. These and other collaborations have allowed CRISPR Therapeutics to significantly accelerate its investigational studies.
Most recently, CRISPR Therapeutics announced that it had treated its first patient with its flagship treatment, CTX001. The CTX001 human trials with Vertex are investigating treatments in patients with B-Thalassemia. A similar CTX001 trial is also being started with patients with sickle cell disease. Results may be ready by 2022, and if favorable, CRISPR Therapeutics could usher in a new era of gene editing therapeutics.
The Potential Impact (And Concerns) Over Gene Editing in Humans
At this point in time, CRISPR Therapeutics has yet to generate any significant revenue from gene-editing activities. This is primarily due to the fact that treatments need more proof to show that they are safe and effective. Potential concerns over gene editing using CRISPR as a therapeutics tool include gene editing missing its targets, as well as the possibility that the gene edits could trigger cancer formation. This factor is the reason why the current clinical trials are so important in predicting the future of gene editing in managing human diseases.
Though concerns exist, optimism far exceeds pessimism in this regard. In fact, stock prices of CRISPR Therapeutics are rising, and the company has a current market cap of $2.06 billion. It still may be several years before an actual gene-editing treatment may come to market. But the impact a successful treatment could yield has patients and investors alike very excited. Certainly, similar companies exist. However, CRISPR Therapeutics looks to be the most likely to introduce a gene-editing breakthrough in the healthcare field.