CRISPR could revolutionize the way we treat thousands of diseases. Currently, there are about 7,000 caused by genetic disorders, which occur when a mutation affects your genes, or when you have the wrong amount of genetic material, as noted by the Cleveland Clinic. Fortunately, CRISPR, or clustered regularly interspaced short palindromic repeats, can potentially treat all of them.
So far, CRISPR has shown promise in treating cardiovascular diseases, cancer, and neurogenerative issues, such as Alzheimer’s, Parkinson’s, and even muscular dystrophy. It may even help with sickle cell anemia, lymphoblastic leukemia, lung cancer, multiple myeloma, Beta thalassemia, etc. I could go on for pages. The bottom line is CRISPR could usher in a new era of transformative medicine we’ve never seen before.
CRISPR Therapeutics (CRSP)
CRISPR Therapeutics (NASDAQ:CRSP) has seen better days. But don’t write it off just yet. There’s the big news ahead for CRISPR Therapeutics.
Along with Vertex Pharmaceuticals (NASDAQ:VRTX), the company’s exagamglogene autotemcel (exa-cel) could see approval for the treatment of sickle cell disease and transfusion-dependent beta-thalassemia (TDT) shortly. Currently, the companies are preparing to submit a Biologics License Application (BLA) for the treatment at the close of the first quarter.
“2022 marked a significant year of progress toward our goal of delivering innovative gene-edited therapies to patients. Exa-cel has the potential to be the first approved CRISPR-based therapy in the world, with regulatory submissions complete in Europe and underway in the United States,”
said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR Therapeutics.
Even better, analysts at Bernstein just initiated coverage of CRSP with a market perform rating, with a price target of $44.
Global X Genomics and Biotechnology ETF (GNOM)
Or, if you want to diversify at less cost, consider an ETF, such as the Global X Genomics and Biotechnology ETF (NASDAQ:GNOM). At $12.16 with an expense ratio of 0.50%, the ETF offers exposure to advances in gene editing, genomic sequencing, genetic medicine/therapy, computational genomics, and biotechnology.
Even better, the GNOM ETF offers exposure to 40 stocks, including Intellia Therapeutics (NASDAQ:NTLA), CRISPR Therapeutics, Myriad Genetics (NASDAQ:MYGN), Sarepta Therapeutics (NASDAQ:SRPT), Gilead Sciences (NASDAQ:GILD), Editas Medicine (NASDAQ:EDIT), and Sangamo Therapeutics (NASDAQ:SGMO) to name a few. What I like most about the ETF is the cost. If I wanted to buy 100 shares of the GNOM ETF, it would cost $1,216. If I wanted to buy 100 shares of each of the ETFs’ major holdings, it would cost me thousands.
Intellia Therapeutics (NTLA)
Intellia Therapeutics also hasn’t been anything to write home about. But don’t write this one off so fast, either. Just recently, the US FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to NTLA-2002 for treating hereditary angioedema ().
NTLA-2002 is an in vivo CRISPR-based investigational therapy designed to inactivate the target gene, kallikrein B1 (KLKB1), to potentially prevent life-threatening swelling attacks in people with HAE, according to the company. That RMAT designation is used to expedite promising candidates, such as this one from NTLA.
Analysts at Bernstein also started NTLA with an outperform rating, with a target of $54. BMO Capital also upgraded the stock to outperform with a target of $57. As noted by TheFly.com, “The firm believes recent NTLA-2002 IND approval removed a significant overhang for Intellia, assuaging investor concerns triggered by Verve’s IND hold and the FDA’s restrictive guidance around gene editing therapies.”
On the date of publication, Ian Cooper did not have (either directly or indirectly) any positions in the securities mentioned. The opinions expressed in this article are those of the writer, subject to the InvestorPlace.com Publishing Guidelines.