5 Best Gene Therapy Stocks to Buy in 2026

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.