In this article, we will list the 5 Best Gene Therapy Stocks to Buy in 2026. Please visit 10 Best Gene Therapy Stocks to Buy in 2026 if you would like to see the extended list and the methodology behind it.
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 (NASDAQ:RCKT) 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 (NASDAQ:FDMT) 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 (NASDAQ:PASG) 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 (NASDAQ:VYGR) 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 (NASDAQ:RGNX) 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.
While we acknowledge the potential of RGNX to grow, our conviction lies in the belief that some AI stocks hold greater promise for delivering higher returns and have limited downside risk. If you are looking for an AI stock that is more promising than RGNX and that has 100x upside potential, check out our report about the cheapest AI stock.
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