A year ago, CRISPR Therapeutics' (CRSP -1.35%) stock was trading for $46 per share. As of Tuesday afternoon, the biotech stock was at $64. It's easy for investors to look at that 39% rise and think they may have missed out on the boom, but that's hardly the case.

While other investors have begun to catch on to the upside of the company, it's still a clinical-stage biopharmaceutical company -- that is, one with no marketed therapies yet. There are no guarantees that CRISPR will get a marketed therapy soon, so that risk is keeping some investors on the sideline and the share price lower than it could be.

However, CRISPR is on the cusp of what could be a blockbuster, has a deep portfolio, and has plenty of cash to spend on research and development, all of which are why I'm saying it's a good time to buy the stock.

Why exa-cel is a big deal

Last month, CRISPR and Vertex Pharmaceuticals (VRTX -1.02%) completed their biologics license application to the Food and Drug Administration (FDA) regarding exa-cel to treat transfusion-dependent beta-thalassemia (TDT) and severe sickle cell disease (SCD). 

TDT and SCD are rare genetic blood disorders that, because they currently have no cure, can be quite expensive to treat over a person's lifetime as they require the need for regular transfusions. TDT patients don't produce enough hemoglobin, the iron-rich protein in red blood cells. SCD patients' red blood cells can become misshapen, causing painful incidents known as vaso-occlusive crises (VOCs).

Exa-cel is a CRISPR/Cas9 gene-edited therapy that edits a patient's stem cells to produce high levels of fetal hemoglobin, then transplants those stem cells back into a patient's body. In clinical trials, the therapy didn't just treat TDT or SCD; it effectively cured both diseases, with patients no longer needing transfusions. In clinical studies, 42 of 44 TDT patients stopped needing transfusions after receiving exa-cel, with two more patients having 75% to 89% reductions in the amount of transfusions. The 31 SCD patients were all VOC-free and no longer needed transfusions.

If approved, exa-cel could potentially cure more than 30,000 people in the U.S. and Europe with TDT or SCD.

Because the drug could end the need for transfusions and hospitalizations for SCD and TDT patients, the Clinical and Economic Review (ICER) said the therapy would be worth $1.9 million per treatment.

While CRISPR would only get 40% of that revenue (with Vertex receiving the rest), that's still a large slice of potential revenue, and the drug could be approved as early as this fall.

Exa-cel is only the beginning

CRISPR has a huge pipeline, and ultimately, exa-cel may not even be the therapy that is its biggest blockbuster. CTX130 is in early trials, but it has shown potential as a chimeric T-cell (CAR-T) therapy to treat a multitude of relapsed or refractory T- or B-cell cancers, including various T-cell lymphomas and kidney cancer. The therapy is made from healthy donor T-cells that are then edited and preserved for off-the-shelf use. The therapy's potential market is large, with kidney cancer alone affecting roughly 50,000 people in the U.S. and 45,000 people in the European Union.

The company is also high on CTX110 to treat B-cell lymphomas, including acute lymphoblastic leukemia (ALL) and chronic lymphocytic leukemia (CLL). CTX110 is also a CAR-T therapy, and it is also in early trials. CRISPR said it had achieved proof of concept with both therapies, with more than 100 people dosed across four trials.

The company is also building on CTX110 and CTX130 with two next-gen oncology therapies. Those therapies, CTX112 and CTX131, with just a few edits to improve their resistance to immunosuppression and tumor-killing ability, recently began clinical trials. It's important to note that most of CRISPR's therapies are wholly owned, so it won't have to share revenue, as it will with exa-cel.

CRISPR's cash will carry it

It's rare that a clinical-stage biotech would be as well-off in terms of cash reserves as CRISPR. At the end of the first quarter, the company said it had $1.89 billion in cash and marketable securities (or, near-term liquidity), compared to $1.86 billion at the end of Dec. 31. That kind of funding will allow the company to be patient in developing its therapies until they can become commercialized.

Though CRISPR doesn't have any product revenue, it received $100 million in collaboration revenue, mostly from upfront payments from Vertex, and a big increase from the $187,000 it received in the first quarter of 2022. The company also trimmed its losses in the quarter to $53.1 million, compared to $179.2 million in the same period last year.

A calculated risk

It's a good time to buy CRISPR stock because, I feel, the market hasn't fully priced in the company's potential. Due to the risks involved with groundbreaking science and the fact that CRISPR doesn't have any product revenue yet, plenty of people are taking a wait-and-see approach to the stock. The problem is, if the FDA does approve exa-cel, the stock will likely jump. Better to get in on a company with solid finances and a big pipeline now. The company has solid long-term potential.