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Ever since scientists began mapping the human genome, gene editing has always been the end goal. The idea once seemed like something out of a science fiction novel, but it’s here today and Crispr Therapeutics AG (NASDAQ:CRSP) is leading the way.
In late 2023, the company had a breakthrough when it received approval for CASGEVY. This is the first gene editing treatment of its kind to be approved. CASGEVY addresses sickle cell disease (SCD) and beta-thalassemia.
That said, CRSP stock is trading at about the same level it was in 2018 and investors who bought the stock in 2020 and 2021 are holding a heavy bag if they’re still involved in it. Many medical stocks carry risk, and that risk is more pronounced when investing in biopharmaceutical companies.
However, CASGEVY is a powerful proof of concept that will likely lead to future gene editing approvals. That means the cash-burning stage may be coming to an end, and the company’s and CRSP stock’s growth may be ready to start.
Keeping an Eye on a Larger Target
Although exact numbers are unknown, SCD impacts millions worldwide, including approximately 100,000 in the United States. The World Health Organization (WHO) and the United Nations recognize that SCD is a global health issue.
In its most recent corporate presentation, the company announced that CASGEVY is approved in eight jurisdictions and that it has 65 authorized treatment centers (ATC) activated globally. It expects new patient initiations to grow significantly in 2025 and estimates the total addressable market for CASGEVY to be approximately 60,000.
However, the addressable market for SCD would pale in comparison to diseases such as cancer, diabetes, and other autoimmune diseases. Gene editing is focused on these long-term goals, and the company is currently involved in five clinical trials and 10 pre-clinical trials.
That’s also why CRSPR views this as a transitional year. By 2026, it expects to generate enough revenue from CASGEVY to deliver sustainable growth for current and future clinical trials. As support for that, CRSPR points out that as of March 31, 2025, it had approximately $1.86 billion of cash on its balance sheet.
There’s Still Time to Buy CRSP Stock
The promise of gene editing is real. A prudent investment in companies like CRSPR Therapeutics has multi-bagger potential. But that day is not today.
One obstacle is the cost of these treatments. Currently, it costs about $2 million per dose of CASGEVY. Doctors may find the price tag too hefty compared to other effective SCD treatments.
In fact, since CRISPR received approval for CRISPR-Cas9 in late 2023, the stock has been down nearly 50% and trading near five-year lows. The company is not yet profitable and generates little revenue, which has made the stock unattractive during higher-for-longer interest rates. Short interest remains high at around 25%.
However, over 69% of CRSP stock is owned by institutional investors, and buying has outpaced selling in each of the last nine quarters. A significant number of analysts also cover CRSP stock. The analyst forecasts on MarketBeat have a Moderate Buy rating on the stock with a $71.75 price target that is over 100% above the CRSP stock price on May 15, 2025.
That tells you that CRSPR isn’t a meme stock. If you believe in the long-term potential of gene editing, a prudent investment in CRSP stock, with money you can afford to use for speculation, could lead to big gains in the next 10 years.