In this article, we will discuss the 10 Best Gene Therapy Stocks to Buy in 2026.
Millions of people around the world live with rare genetic diseases that cause suffering and can lead to premature deaths. Most of these problems stem from defective genes.
Gene therapies aim to treat or prevent diseases by introducing new instructions in a patient’s cells to fix or replace the defective genes behind genetic disorders. These therapies promise ground-breaking solutions for hard-to-treat diseases. With the increasing prevalence of genetic disorders, regulatory regimes are adjusting to give patients faster access to promising treatments.
On February 23, Reuters reported that the FDA planned to speed up approval of gene therapies targeting rare and life-threatening diseases. The report said that the agency’s proposed guideline would allow drugmakers to seek marketing approval with smaller clinical datasets than would normally be required.
Beyond the relief for patients, the gene therapy field presents a strong growth opportunity for biotech and pharmaceutical companies. According to Mordor Intelligence, the gene therapy market is experiencing rapid expansion. This comes as real-world evidence continues to show that these therapies deliver durable benefits for patients and families. The gene therapy market is estimated at $10 billion in 2026 and is forecast to grow to roughly $26 billion by 2031.
That said, let’s explore some of the best gene therapy stocks to buy in 2026.
Our Methodology
To determine the 10 best gene therapy stocks to buy in 2026, we reviewed various ETF holdings, sifted through industry publications, and scanned financial media reports to identify companies in the genetics medicine field. From there, we selected pure-play gene therapy companies in commercial or clinical stages. We ended up with dozens of stocks in our initial list and applied additional filters to pick out the best. We looked for stocks with at least 10% upside potential and strong hedge fund support, drawing on Insider Monkey’s database as of Q4 2025. Finally, we ranked the stocks based on their upside potential.
Why are we interested in the stocks that hedge funds pile into? The reason is simple: our research shows we can outperform the market by imitating the top stock picks of the best hedge funds. Our quarterly newsletter’s strategy selects 14 small-cap and large-cap stocks every quarter and has returned 498.7% since May 2014, beating its benchmark by 303 percentage points (see more details here).
Best Gene Therapy Stocks to Buy in 2026
10. Sarepta Therapeutics Inc (NASDAQ:SRPT)
Stock Upside Potential: 14.48%
Number of Hedge Fund Holders: 36
Sarepta Therapeutics Inc (NASDAQ:SRPT) is one of the best gene therapy stocks to buy in 2026. Sarepta’s Q1 2026 report, released on May 6, showed that both earnings and revenue surpassed analysts’ expectations. Adjusted EPS of $3.16 smashed the expectation of $0.99, and revenue of $730.8 million exceeded the anticipated $474.3 million.
During the quarter, Sarepta’s flagship gene therapy drug, Elevidys, launched in Japan. The drug’s commercial launch in Japan earned Sarepta $40 million in milestone payments in Q1 under a deal with Roche.
However, Elevidys sales have come under pressure due to safety concerns that led Sarepta to update the drug’s label. In Q1, Elevidys revenue dropped to $102 million from $110 million in the previous quarter. The management attributed the revenue decline to lower Elevidys volume sales following the label update.
That said, Sarepta is rebuilding confidence in Elevidys and expects momentum to pick up gradually beginning in the back half of 2026 into 2027. Sarepta says that Elevidys is the only FDA-approved gene therapy for Duchenne muscular dystrophy (DMD), and the drug is backed by long-term data. According to the company, there remains a large eligible population that could use Elevidys, so it sees a substantial multi-year opportunity for the drug ahead.
Sarepta Therapeutics Inc (NASDAQ:SRPT) is a medical research and drug development company based in Massachusetts. Its specialty is precision genetic medicines for rare diseases. Sarepta develops its products using gene therapy, gene editing, and RNA technologies.
9. Uniqure NV (NASDAQ:QURE)
Stock Upside Potential: 41.19%
Number of Hedge Fund Holders: 66
Uniqure NV (NASDAQ:QURE) is one of the best gene therapy stocks to buy in 2026. Uniqure shares have gained 60% over the past month and more than doubled over the past year. The Street believes the stock’s rally isn’t done yet and anticipates at least a 40% gain over the next 12 months.
On April 30, Uniqure announced plans to apply for regulatory approval of its product candidate AMT-130 in the UK. AMT-130 is a gene therapy targeting Huntington’s disease. Uniqure plans to file for AMT-130’s approval with the UK regulator in Q3 2026.
The UK filing will be based on the data from the ongoing Phase 1/2 clinical trials of AMT-130 in the US and Europe. When trial data were analyzed at the three-year mark, the results showed that patients treated with the high dose of AMT-130 showed a 75% slowing of Huntington’s disease progression.
Uniqure has discussed the data package and manufacturing requirements with the UK regulator to support the AMT-130 application. Uniqure CEO Matt Kapusta said that they received constructive feedback from the UK regulator as they work toward securing marketing approval for AMT-130.
The company is also eying AMT-130 approval in the US. It plans to meet the FDA in Q2 2026 to discuss a potential Phase 3 trial of AMT-130, which could pave the way for approval of Huntington’s disease gene therapy in the US.
Uniqure NV (NASDAQ:QURE) is a Dutch biotech company founded in 1998 and based in Amsterdam, Netherlands. It develops gene therapies for rare and severe diseases. Uniqure is an industry leader, as its Glybera product became the first approved gene therapy in the Western world.
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