Gene editing stocks have received a lot of attention throughout 2023. The intense focus on gene editing therapies and CRISPR technology is likely only to increase moving forward: The U.S. Food and Drug Administration (FDA) just approved the world’s first medicine employing CRISPR technology.
The technology allows scientists to splice and edit the DNA in ways that were never possible before. CRISPR received the Nobel Prize in chemistry in 2020.
Since then many scientific and biopharmaceutical firms have continued to seek methods to commercialize the technology. It has the potential to cure a broad range of diseases. Given its recent approval by the FDA for use in treating sickle cell disease, it’s likely that more and more gene editing stocks are going to grow.
CRISPR Therapeutics (CRSP)
CRISPR Therapeutics (NASDAQ:CRSP) Is the big winner as the first gene editing therapies come to market. The company developed one of the two FDA-approved therapies along with Vertex Pharmaceuticals (NASDAQ:VRTX). The other approved therapy was developed by Bluebird Bio (NASDAQ:BLUE).
However, The label for bluebird bio’s treatment included a warning that some patients who had received the treatment developed cancer. The markets did not respond well to the information and its shares fell by 1/3.
CRISPR Therapeutics developed its therapeutic For the treatment of sickle cell disease under the trade name Casgevy. The treatment uses a patient’s cell and is developed inside of a lab rather than inside their bodies. While that eliminates certain risks, it also requires chemotherapy. That is a significant downside for patients along with a $2.2 million price tag.
It is expected that some insurers will deny coverage of the therapy, limiting its potential commercial success. Further, The chemotherapy regimen required is very intensive and results in infertility. Regardless, CRISPR Therapeutics is first to the market and the company continues to develop other therapies for related blood disorders including beta thalassemia.
Caribou Biosciences (CRBU)
Caribou Biosciences (NASDAQ:CRBU) continues to develop gene-edited therapies for the treatment of cancer. The firm’s pipeline of clinical trials spans three therapies in Stage 1.
The first is a study to try and find a treatment of refractory B cell Non-Hodgkin’s lymphoma. The other two programs are for myeloma and leukemia, respectively. The company anticipates that it will share FDA feedback regarding its lymphoma program by the end of the year. The company is currently enrolling patients in one of its other two programs and intends to begin enrollment of patients in the remaining program in 2024.
At the end of the third quarter of the company had $396.7 million in liquidity. The company expects that funding to keep it operational through the fourth quarter of 2025 at least. Caribou Biosciences’ shares currently trade for just below $6 and have the potential to multiply in value several times.
Intellia Therapeutics (NTLA)
Intellia Therapeutics (NASDAQ:NTLA) has developed a pipeline of in-vivo and ex-vivo therapies, many in partnership with Regeneron (NASDAQ:REGN). In-vivo therapies deliver cells directly into the patient’s body whereas ex-vivo therapies remove those cells, modify them and reintroduce them into the patient’s body. CRISPR’s Casgevy is an example of an ex-vivo therapy.
Intellia Therapeutics’ lead program is NTLA-2001. The program is being studied for its efficacy in treating transthyretin amyloidosis. The disease is characterized by a buildup of abnormal proteins called fibrils. Those proteins can concentrate in the nerves and the heart, weakening its pumping mechanism.
The disease affects thousands of patients worldwide with those afflicted having a life expectancy of 2 to 15 years from diagnosis. Intellia Therapeutics and its NTLA-2001 program employ gene editing to reduce circulating levels of the abnormal protein. That program is currently approaching late stage clinical trials and if successful promises to multiply the value of NTLA stock.
On the date of publication, Alex Sirois did not have (either directly or indirectly) any positions in the securities mentioned in this article. The opinions expressed in this article are those of the writer, subject to the InvestorPlace.com Publishing Guidelines.
