Is This Biotech Stock Your Best Shot at Building a Millionaire-Making Position?

Investors looking for stocks that can turn a modest portfolio into a multimillion-dollar nest egg often find what they're looking for in the biotech industry. For several years, CRISPR Therapeutics (NASDAQ: CRSP) has been attracting investment from Cathie Wood's Ark Innovation ETF. At the moment, it's the fund's second largest holding, at 6.6% of the overall portfolio.

You don't have to command billions like Wood does to see what makes CRISPR Therapeutics look like a millionaire-maker stock. It already has one approved therapy, Casgevy, and a well-heeled partner, Vertex Pharmaceuticals (NASDAQ: VRTX), to manufacture and commercialize it.

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Growing sales of Casgevy plus new candidates emerging from CRISPR Therapeutics' development pipeline could turn the company's recent losses into huge gains, but success is still a long way from guaranteed. Here's a look at some of the challenges CRISPR Therapeutics faces to gauge its chance at providing millionaire-making returns.

Why Casgevy sales could continue disappointing investors

CRISPR Therapeutics is entitled to a share of profits that remain after Vertex Pharmaceuticals is done paying for the manufacturing and commercialization of Casgevy. Despite earning approval in 2023, Vertex didn't generate any profit from Casgevy to share with CRISPR Therapeutics last year.

Vertex can't begin manufacturing Casgevy until it has stem cells collected from patients. Unfortunately, harvesting viable stem cells from sickle cell disease patients and using them to manufacture Casgevy has been extremely challenging. In 2024, 54 patients underwent their first stem cell collection, but just five were infused with the final product. There were 64 patients who received a Casgevy infusion in 2025, but a majority of patients who started the cell collection process did not receive the final product by the end of the year.

Vertex and CRISPR Therapeutics' manufacturing challenges spell trouble for investors because there's a competing therapy that also offers sickle cell disease patients permanent relief from blood transfusions. Genetix, formerly bluebird bio, is a privately held company that markets a cell-based therapy for sickle cell disease patients called Lyfgenia. Genetix reported over 100 Lyfgenia infusions last year, and the vast majority required just one stem cell collection procedure.

Betting on pipeline progress

CRISPR Therapeutics' operations lost a stunning $664.6 million last year. With Casgevy unlikely to generate significant revenue, CRISPR Therapeutics' investors are relying on the company's new drug candidates to pick up the slack.

In the second half of 2026, CRISPR Therapeutics expects to provide an update regarding CTX310, an mRNA-based treatment intended to switch off the ANGPTL3 gene. In a small phase 1 study, a single dose helped patients with severe dyslipidemia lower their cholesterol and triglycerides by about half.

A one-shot cure for patients with severe dyslipidemia could be worth billions in annual sales, but there's still a lot that we don't know about CTX310's long-term safety profile. It's probably best to wait until after we see another update before assuming this is your best chance at building a position worth millions.

Cory Renauer has no position in any of the stocks mentioned. The Motley Fool has positions in and recommends CRISPR Therapeutics and Vertex Pharmaceuticals. The Motley Fool has a disclosure policy.