Picking a growth stock that becomes a big winner over a decade or more can transform any investor’s returns. I know this from my own portfolio, where the flowers happily outshine the occasional giant weed.

Here, I’ll outline a high-risk, high-reward stock that’s recently piqued my interest.

An exciting gene-editing platform

The share is CRISPR Therapeutics (NASDAQ: CRSP). This biotech firm is focused on developing gene-editing therapies using CRISPR technology to treat genetic diseases, such as sickle cell disease and cancer.

Its breakthrough medicine, Casgevy, has now been approved in multiple countries. Developed in collaboration with Vertex Pharmaceuticals, this therapy treats sickle cell disease and transfusion-dependent beta thalassemia. Both are rare, debilitating blood disorders.

The treatment works by removing stem cells from a patient’s bone marrow, editing the faulty gene in a lab, then re-infusing the modified cells into the patient. It’s no exaggeration to call this transformative.

In August, the NHS announced it will begin rolling out Casgevy for beta thalassemia. There are about 460 patients in England potentially eligible for the treatment. It will cost about £1.65m ($2.09m) per patient.

Limited competition

CRISPR and Vertex see an addressable market of 35,000 patients in Europe and the US, with another 23,000 in Saudi Arabia and Bahrain.

Given the treatment costs $2m-$2.2m in Europe and the US, that’s a significant revenue opportunity. Vertex, with whom the firm will share product revenue, is predicting a “potential multi-billion opportunity”.

Better still, the therapy currently has no competition in Europe and the Middle East. In the US, it has a rival in the shape of Bluebird Bio, which has an approved sickle cell disease treatment. So that’s worth noting.

However, the market doesn’t seem to rate Bluebird’s prospects, given the stock is down 84% in one year and is priced at just $0.50. It has limited funds and its treatment is uncompetitively priced at $3.1m.

As of mid-July, Vertex had activated more than 35 treatment centres globally to collect patients’ cells. But since revenue is only recognized once patients receive their treatment, commercialisation is still in its early stages.

So while sales are expected to motor higher from 2025, potential delays could always arise.

Indistinguishable from magic

CRISPR stock skyrocketed to $199 in 2021, but has since fallen 74% to its current price of $50. This gives the firm a $4.3bn market cap.

For comparison, Vertex Pharmaceuticals is now a $123bn company after its share price surged around 4,350% over the past two decades. This shows what’s possible if a small biotech gets it right.

CRISPR ended Q2 with $2bn in cash and equivalents, so remains well-capitalised to advance its five other gene-editing medicines in clinical trials. Naturally though, there’s a risk some or all of these could fail.

Also, since these treatments are designed to be one-time cures, they’re unlikely to generate recurring sales like medications that require continuous use. Investors obviously favour recurring revenue.

Still, the high upfront price and likely expansion into other areas could drive significant financial success. Therefore, I think the stock could rocket in the years ahead and I’m considering buying it.

British science fiction writer Arthur C. Clarke once said: “Any sufficiently advanced technology is indistinguishable from magic.” This revolutionary gene-editing technology is akin to magic, in my opinion.