CRISPR Therapeutics isn't the only CRISPR-focused biotech company with a big partner. Intellia Therapeutics (NTLA +4.43%) has teamed up with Regeneron Pharmaceuticals (REGN -2.04%).
The two companies are evaluating nexiguran ziclumeran (also known as NTLA-2001) in a phase 3 clinical study for the treatment of transthyretin amyloidosis (ATTR) with cardiomyopathy, a rare genetic disease. This experimental therapy has also advanced into another phase 3 study targeting hereditary ATTR amyloidosis with polyneuropathy.
Intellia also has a late-stage program that it fully owns. The company is evaluating lonvoguran ziclumeran (also known as NTLA-2002) in a phase 3 clinical study as a potential treatment for hereditary angioedema, a rare genetic disease characterized by severe inflammatory attacks in various organs and tissues.
Regeneron and Intellia are also working together to evaluate a CRISPR gene-editing therapy targeting hemophilia. In addition, Intellia is collaborating with multiple partners in researching the use of gene-editing therapies in treating other genetic diseases, autoimmune diseases, and cancer.
CRISPR companies forging the future
Several companies are using CRISPR to edit human genomes in an attempt to treat (and even cure) genetic diseases. Their therapies use either an ex vivo or an in vivo approach. With ex vivo therapies, genes are edited outside the body.
But CRISPR's use isn't limited to genetic diseases. Several gene-editing companies are also targeting various types of cancer. In particular, CRISPR is being used to develop chimeric antigen receptor (CAR) T-cell therapies, in which immune cells are genetically engineered to target specific tumors.
In addition, some companies are using CRISPR for screening (which shouldn't be confused with genetic testing). In CRISPR screening, genes are edited to render them inoperative. CRISPR screening enables companies to identify which genes perform specific functions, which is important for drug development.
Benefits and risks of investing in CRISPR companies
The benefits of investing in CRISPR companies include:
- High long-term growth potential.
- An opportunity to get in on a new and exciting medical technology.
- Your investment reflects a vision of a better world where more diseases can be treated and potentially even cured.
However, there are also several risks associated with investing in CRISPR companies, such as:
- High volatility.
- Significant uncertainty related to clinical trials and commercial success.
- Increased competition (including non-CRISPR companies).
How to invest in CRISPR stocks
Follow these steps to invest in CRISPR stocks:
- Open your brokerage app: Log in to your brokerage account where you handle your investments.
- Search for the stock: Enter the ticker or company name into the search bar to bring up the stock's trading page.
- Decide how many shares to buy: Consider your investment goals and how much of your portfolio you want to allocate to this stock.
- Select order type: Choose between a market order to buy at the current price or a limit order to specify the maximum price you're willing to pay.
- Submit your order: Confirm the details and submit your buy order.
- Review your purchase: Check your portfolio to ensure your order was filled as expected and adjust your investment strategy accordingly.
