Gene editing stocks have gone from speculative and theoretical to commercially viable. In December 2023, the United States Food & Drug Administration (FDA) approved the first CRISPR therapies. That’s changed the conversation from “if” to “how fast.”
Table of Contents
How fast may take longer than some investors would like. However, it means there’s still an opportunity to invest in gene editing stocks while a long runway exists.
Right now, there are two distinct paths for investors. One is to invest in established pharmaceutical companies. These companies have the financial resources to integrate gene editing into their pipelines by acquiring smaller companies that have done the initial research and development.
The second path is to look for smaller innovators that are pioneering first-generation cures. Some of these companies may be takeover candidates for the future.
Here’s a look at four gene editing stocks ranging from blue-chip stability to high-risk, high-reward innovation.
Big Pharma’s Quiet Gene Editing Powerhouse
Eli Lilly & Co. (NYSE: LLY) has been one of the best stocks to own in the last three years regardless of the sector. LLY stock has delivered a total return of over 187% in that time, largely stemming from its leadership in GLP-1 drugs for treating type 2 diabetes and obesity.
However, Lilly is also using its robust balance sheet to fund key acquisitions and collaborations in the gene editing space. The company has deals in place with Beam Editing for base editing and Precision BioSciences for in vivo gene editing.
This positions the company in the “how” and the “where” of gene editing. Its current focus is on diseases driven by genetic mutations in the liver and neuromuscular system. The company’s candidates are in the early trial stage with completion not expected until 2028 to 2030.
That would be a concern for many smaller firms, but Lilly has the cash flow and global infrastructure to pull it off. Lilly isn’t the speculative moonshot that some investors crave, but it’s a blue-chip sector leader with yet another reason to believe it will climb even higher.
Commercializing CRSPR First
In the introduction, I mentioned that the FDA approved its first CRISPR therapy in 2023. Vertex Pharmaceuticals Inc. (NASDAQ: VRTX) is the large-cap biotech name behind that achievement. Vertex produced Casgevy via its partnership with CRISPR Therapeutics. Casgevy is a first-of-its-kind treatment for sickle cell disease and beta-thalassemia.
Vertex is also exploring in vivo delivery methods and expanding its genetic medicine pipeline beyond hematology to areas that include cystic fibrosis and Type 1 diabetes. That makes it a leader not just in gene editing, but in translational biotechnology, turning molecular innovation into patient outcomes and revenue.
However, investors haven’t seen much impact from gene editing on its stock yet. VRTX stock has delivered a total return of around 41% in the past three years. That’s not bad by any means, but certainly not what many growth investors seek.
One reason for that is that Casgevy is still in the early commercialization phase. Vertex expects around $100 million in revenue from Casgevy in its 2025 fiscal year, a fraction of the company’s total revenue which is expected to be approximately $12 billion.
The Pioneer Has Moved from Science to Revenue
CRISPR Therapeutics (NASDAQ: CRSP) helped invent the modern gene editing era, and now it’s starting to reap the rewards. Best known as Vertex’s partner on Casgevy, the company is transitioning from a development-stage biotech to a revenue-generating enterprise.
Beyond its approved therapy, CRISPR Therapeutics has a deep pipeline that includes CAR-T oncology programs, as well as in vivo gene editing candidates aimed at muscle and liver disorders.
The company’s expertise in CRISPR-Cas9 biology positions it at the forefront of next-generation therapeutics. This includes its investigational CRISPR/Cas9 in vivo gene editing therapy CTX310 that targets angiopoietin-related protein 3 (ANGPTL3) for cardiovascular and cardiometabolic disease.
Still, CRSP stock is a highly volatile moonshot of sorts. The company’s fortunes are closely tied to the speed and success of its early pipeline. For investors comfortable with risk, this is a pure-play on CRISPR technology with tangible commercial validation and a long runway for growth.
A Deep-Science Speculative Play
Editas Medicine Inc. (NASDAQ: EDIT) is the most speculative of the gene editing stocks on this list. The company is a pioneer in the CRSIPR field. However, Editas is still a clinical-stage company, which makes it a high-risk investment that may produce a high reward for investors.
The company’s lead program, EDIT-401, is a one-time gene editing therapy designed to cut LDL cholesterol. Another candidate, EDIT-301, targets sickle cell disease and beta thalassemia using a unique editing approach that differs from Casgevy’s. Early trial data have been promising, showing effective gene correction and robust hemoglobin production in treated patients.
While execution has been uneven in the past, recent management changes and solid clinical results have revived investor optimism. With a market cap under $1 billion, even modest clinical success could deliver outsized returns. For those willing to stomach volatility, Editas is the classic moonshot in genetic medicine.
Leave a Reply