5 Best Gene Therapy Stocks to Buy in 2026

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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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