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

newsfeedback@fool.com (Cory Renauer)
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⚡ Quantum Brief
CRISPR Therapeutics is now the second-largest holding (6.6%) in Cathie Wood’s flagship innovation ETF, signaling strong institutional confidence in its disruptive gene-editing potential. Its FDA-approved therapy Casgevy, developed with Vertex Pharmaceuticals, faces critical manufacturing hurdles: only 64 patients received infusions in 2025 despite 54+ stem cell collections in 2024, delaying profit-sharing. Competitor Genetix’s Lyfgenia outpaced Casgevy with 100+ infusions in 2025, leveraging a simpler single-collection process, threatening CRISPR’s market share in sickle cell disease treatments. The company’s $664.6M 2025 loss underscores reliance on pipeline candidates like CTX310, an mRNA-based cholesterol treatment showing 50% reductions in phase 1, with pivotal data expected mid-2026. Investor optimism hinges on upcoming trial results, but long-term safety risks and manufacturing inefficiencies leave CRISPR’s "millionaire-maker" potential speculative and high-risk.
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By Cory Renauer – Apr 7, 2026 at 4:43AM ESTKey PointsCRISPR Therapeutics is the second largest holding in Cathie Wood's Ark Innovation ETF.Incoming clinical trial data could send the stock soaring this year.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 (CRSP 0.73%) 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 (VRTX 1.00%), to manufacture and commercialize it. Image source: Getty Images. 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. ExpandNASDAQ: CRSPCRISPR TherapeuticsToday's Change(-0.73%) $-0.36Current Price$49.15Key Data PointsMarket Cap$4.7BDay's Range$49.05 - $50.7752wk Range$30.52 - $78.48Volume773Avg Vol1.9MGross Margin-653467.24% 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.Read NextApr 7, 2026 •By Adria CiminoYou'll Never Guess Which Healthcare Stock Delivered the Best First-Quarter Performance in the S&P 500.Apr 6, 2026 •By Eric VolkmanWhy ImmunityBio Stock Slumped on MondayApr 6, 2026 •By Eric VolkmanWhy Biogen Stock Got Mashed on MondayApr 6, 2026 •By Josh Kohn-LindquistStock Market Today, April 6: Soleno Therapeutics Climbs After $2.9 Billion All-Cash Buyout AgreementApr 6, 2026 •By Eric VolkmanWhy Soleno Therapeutics Stock Rocketed Higher on MondayApr 4, 2026 •By Parkev Tatevosian, CFAUnitedHealth Stock Analysis: Generational Buying Opportunity or a Falling Knife to Avoid?About the AuthorCory Renauer is a contributing Motley Fool healthcare analyst covering pharmaceuticals, biotechnology, and medical devices. Previously, Cory was a laboratory technician for the American Red Cross. He holds a bachelor’s degree in biology from Oakland University.TMFang4applesX@coryrenauerStocks MentionedVertex PharmaceuticalsNASDAQ: VRTX$434.30(-1.00%)-$4.41CRISPR TherapeuticsNASDAQ: CRSP$49.15(-0.73%)-$0.36Ark ETF Trust - Ark Innovation ETFNYSEMKT: ARKK$68.80(+0.35%)+$0.24*Average returns of all recommendations since inception. Cost basis and return based on previous market day close.

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