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 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 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.
8. Beam Therapeutics Inc (NASDAQ:BEAM)
Stock Upside Potential: 69.01%
Number of Hedge Fund Holders: 31
Beam Therapeutics Inc (NASDAQ:BEAM) is one of the best gene therapy stocks to buy in 2026. The stock is up roughly 27% in the past six months and soared more than 60% in the past year. The Street expects BEAM stock to pop almost 70% in the next 12 months as therapy programs continue to advance.
On May 14, H.C. Wainwright reaffirmed its Buy rating and $80 price target on Beam Therapeutics stock. The firm based its decision on the progress of Beam’s BEAM-302 and risto-cel programs.
BEAM-302 is Beam’s lead genetic disease program. It’s aimed at treating alpha-1 antitrypsin deficiency (AATD) to address the underlying mutation that causes both liver and lung damage. Phase 1/2 trial of BEAM-302 is ongoing, and updated safety and efficacy data from that study has been positive. Beam Therapeutics is working toward initiating a pivotal Phase 1/2 trial of BEAM-302 in the second half of 2026. The company intends to seek an accelerated approval pathway for BEAM-302.
Meanwhile, Beam Therapeutics expects to file for marketing approval of Ristoglogene autogetemcel before the end of 2026. Ristoglogene autogetemcel, also called risto-cel, is a treatment for sickle cell disease that has produced impressive results in clinical trials. This therapy is currently in Phase 1/2 BEACON clinical trial, where it is being evaluated on sickle cell disease with severe vaso-occlusive crises.
Beam Therapeutics Inc is a biotech company in the genetic medicines space. The company is developing advanced gene therapies using a cutting-edge gene editing technique known as base editing. This technique enables Beam to chemically modify single letters without making double-stranded breaks in the DNA.
7. MeiraGTx Holdings PLC (NASDAQ:MGTX)
Stock Upside Potential: 116.22%
Number of Hedge Fund Holders: 23
MeiraGTx Holdings PLC (NASDAQ:MGTX) is one of the best gene therapy stocks to buy in 2026. The stock has gained around 25% year-to-date and soared more than 95% over the past year. Looking ahead, analysts believe the stock could explode more than 100% in the next 12 months as MeiraGTx advances its various therapy programs.
MeiraGTx reported its Q1 2026 financial and operational results on May 14. Regarding financials, the company expects its cash runway to extend into the second half of 2028. It said that it exited Q1 with $71.5 million in cash and expects to receive $14.7 million in tax incentives.
The company has also launched an equity offering to raise around $100 million. Additionally, MeiraGTx expects to receive $95 million in upfront payments from its deal Hologen, a joint venture partner.
Regarding its therapy programs, MeiraGTx said it is on pace to launch two therapies in the next two years. The company highlighted positive results from a Phase 1 study of AAV2-hAQP1, a potential therapy for patients with xerostomia. This program has recently received the FDA’s Breakthrough Therapy Designation, which MeiraGTx says supports potential accelerated development and regulatory pathway.
MeiraGTx also said that it acquired full rights to bota-vec from Johnson and Johnson. Bota-vec is a late-stage program being developed as a therapy for X-linked retinitis pigmentosa.
MeiraGTx Holdings PLC is a biotech company focused on genetic medicine. It develops gene therapies for both rare and common diseases. It targets ocular, neurodegenerative, and metabolic disorders.
6. Solid Biosciences Inc (NASDAQ:SLDB)
Stock Upside Potential: 119.69%
Number of Hedge Fund Holders: 27
Solid Biosciences Inc (NASDAQ:SLDB) is one of the best gene therapy stocks to buy in 2026. The stock has been a big win for investors, having gained roughly 75% over the past six months and surged more than 190% over the past 12 months. Analysts expect Solid Biosciences stock to keep going up, predicting that it could more than double in the next 12 months.
The stock has soared as Solid Biosciences advances its gene therapy development programs. On May 7, Solid Biosciences announced that it has begun a Phase 3 trial of its drug candidate SGT-003. The company plans to conduct this trial in multiple countries, including Australia, Canada, US, UK, and the EU zone. The first patient dosed in the Phase 3 study was in Australia.
Solid Biosciences is developing SGT-003 as a treatment for Duchenne muscular dystrophy. SGT-003 has received fast-track, orphan drug, and rare pediatric designations from the FDA.
According to Solid Biosciences, the Phase 3 study is designed to play a major role in supporting various regulatory pathways for gene therapy in the US and internationally. Notably, SGT-003’s Phase 3 study joins the ongoing Phase 1/2 trial of the drug candidate.
Solid Biosciences Inc is a life sciences company based in Massachusetts. It is focused on developing gene therapies for cardiac and neuromuscular diseases. It targets rare diseases with unmet medical needs.
5. Rocket Pharmaceuticals Inc (NASDAQ:RCKT)
Stock Upside Potential: 180.60%
Number of Hedge Fund Holders: 39
Rocket Pharmaceuticals Inc (NASDAQ:RCKT) is one of the best gene therapy stocks to buy in 2026. The Street expects Rocket Pharmaceuticals’ stock to explode 180% over the next 12 months.
On April 28, Leerink Partners boosted its price target on Rocket Pharmaceuticals shares to $12 from $11 while keeping a Market Perform rating on the stock. The firm cited Rocket’s recent sale of FDA priority review vouchers as the reason for the price target upgrade.
Rocket received the vouchers as part of the FDA approval of its gene therapy product Kresladi, which targets a rare pediatric disease. These vouchers are transferable and allow the holder to get accelerated FDA review of future drug applications. Rocket decided to sell its vouchers to raise funds to bankroll its programs. It sold the vouchers for $180 million.
According to Leerink, the voucher sale provides Rocket with non-dilutive capital and extends the company’s cash runway. Therefore, this transaction eases balance sheet concerns as Rocket works through its various clinical programs. In light of this, Leerink decided to raise its price target on Rocket Pharmaceuticals stock.
According to Rocket CEO Gaurav Shah, the voucher sale extends the company’s cash runway into Q2 2028 and strengthens its ability to advance the cardiovascular gene therapy pipeline.
Rocket Pharmaceuticals Inc is an American biotech company focused on developing gene therapies for rare and devastating diseases. It has programs targeting cardiovascular, immunology, and hematology conditions.
4. 4D Molecular Therapeutics Inc (NASDAQ:FDMT)
Stock Upside Potential: 195.62%
Number of Hedge Fund Holders: 36
4D Molecular Therapeutics Inc (NASDAQ:FDMT) is one of the best gene therapy stocks to buy in 2026. The stock has gained nearly 30% year-to-date and surged more than 190% over the past year. Looking ahead, analysts expect FDMT stock to almost triple in the next 12 months as the company continues to advance its clinical programs.
On May 7, 4D Molecular Therapeutics reported its Q1 2026 results and issued operational updates. On the financial side, the company said that it expects to have enough cash to fund its operations at least into the second half of 2028. It closed Q1 with $458 million in cash, cash equivalents, and marketable securities. The company also expects to receive some payments under its arrangement with Otsuka.
Regarding its clinical programs, 4D Molecular Therapeutics said it completed enrollment in the 4FRONT-1 Phase 3 trial of 4D-150 ahead of schedule. It said this demonstrates strong investigator and patient enthusiasm for the study. It expects topline data from this study in the first half of 2027. The company expects to complete enrollment in the 4FRONT-2 Phase 3 trial of 4D-150 in the back half of 2026 and expects topline data readout in the second half of 2027.
4D Molecular is developing 4D-150 as a therapy for blinding retinal vascular diseases. The lead indication is wet age-related macular degeneration, which is the subject of the 4FRONT Phase 3 studies. Another indication is diabetic macular edema. 4D Molecular is also advancing programs for geographic atrophy and cystic fibrosis therapies.
California-based 4D Molecular Therapeutics Inc is a biotech company focused on genetic medicines. The company is developing advanced gene therapies that target rare and common diseases in areas like ophthalmology and pulmonology.
3. Passage Bio Inc (NASDAQ:PASG)
Stock Upside Potential: 125.73%
Number of Hedge Fund Holders: 7
Passage Bio Inc (NASDAQ:PASG) is one of the best gene therapy stocks to buy in 2026. While the stock is down around 50% year-to-date, analysts see it surging 125% over the next 12 months.
On April 20, Passage Bio said it was reviewing its strategic alternatives to maximize shareholder value. It has tapped Wedbush PacGrow as a financial advisor to help with this task.
Passage Bio’s lead product candidate is PBFT02. The company is developing PBFT02 as a potential treatment for frontotemporal dementia. A Phase 1/2 trial of this gene therapy is ongoing.
Interim data from that trial suggested that PBFT02 may slow disease progression in patients with frontotemporal dementia. The management cited improvements in both brain atrophy and plasma neurofilament levels, and noted that these two are well-established biomarkers of disease progression.
However, when the company met with the FDA, the regulator indicated that it would require a randomized controlled trial for registrational purposes. Passage Bio’s management said that in light of this outcome as well as taking into account logistical and financial challenges, they are viewing the next steps for the PBFT02 program as and the company.
Pennsylvania-based Passage Bio Inc is a genetic medicines company. Passage Bio is developing gene therapies for central nervous system disorders. Its programs target hard-to-treat, devastating diseases.
2. Voyager Therapeutics Inc (NASDAQ:VYGR)
Stock Upside Potential: 252.57%
Number of Hedge Fund Holders: 17
Voyager Therapeutics Inc (NASDAQ:VYGR) is one of the best gene therapy stocks to buy in 2026. While the stock has risen only a modest 30% over the past year, analysts believe it can explode more than 250% over the next 12 months as the company advances its drug programs.
On May 13, Voyager Therapeutics announced that it presented preclinical data for its Alzheimer’s disease gene therapy, VY1706, at the American Society of Gene & Cell Therapy’s 2026 Annual Meeting in Boston.
Voyager’s VY1706 is a tau-silencing gene therapy. In testing in non-human primates, the therapy reduced tau protein in key brain regions by up to 64%. The treatment was also well-tolerated with no adverse events up to the highest dose.
With these solid results on hand, Voyager says it is on track to request FDA authorization for clinical testing of VY1706 in humans in Q2 2026. If the request is granted, the company projects first-in-human dosing with VY1706 in patients living with Alzheimer’s disease to occur in the back half of 2026.
Voyager is also advancing its VY7523 (anti-tau antibody) program and expects efficacy data from an ongoing trial in the back half of 2026. The company exited Q1 2026 with $172 million in cash and expects this to be enough to fund its programs into 2028.
Massachusetts-based Voyager Therapeutics Inc is a biotechnology company developing gene therapies for diseases with unmet medical needs. It focuses on treatments for neurological diseases such as Alzheimer’s disease and Parkinson’s disease.
1. Regenxbio Inc (NASDAQ:RGNX)
Stock Upside Potential: 324.77%
Number of Hedge Fund Holders: 28
Regenxbio Inc (NASDAQ:RGNX) is one of the best gene therapy stocks to buy in 2026. On May 14, Regenxbio reported its Q1 2026 financial results and provided operational updates. The company exited the quarter with $150.5 million in cash. The management expects the cash balance to be enough to fund operations into early 2027.
Regenxbio has multiple drug development programs underway. Its RGX-202 gene therapy product candidate returned positive topline results in a Phase 3 study for the treatment of Duchenne. The therapy demonstrated efficacy and was well-tolerated with a favorable safety profile. The company said the topline results support plans for potential accelerated approval of RGX-202 in 2027.
Regenxbio also said that it expects to receive $100 million in milestone payments from AbbVie in connection with their partnered program. Regenxbio and AbbVie are working together to develop ABBV-RGX-314 as a treatment for various retinal conditions. They are setting up for Phase 2b/3 trials of ABBV-RGX-314, with dosing expected to begin in Q2 2026. The milestone payment will be triggered once the first patient is dosed in the Phase 2b study.
Regenxbio Inc is an American biotech company based in Maryland. The company is focused on developing gene therapies for a variety of rare and retinal diseases. It has several potential therapies in the pipeline, including one that targets Duchenne treatment.
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