10 Best Gene Therapy Stocks to Buy Right Now

2) Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT)

Number of Hedge Fund Holders: 31

Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) operates as a late-stage biotechnology company that focuses on developing gene therapies for rare and devastating diseases.

Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT)’s lead program, RP-A501 for Danon disease, received significant attention from the medical community. Danon disease is a rare, X-linked cardiomyopathy caused by a deficiency of LAMP2 protein, that often leads to severe cardiac issues and early mortality. RP-A501 is being developed as a potential one-time gene therapy and the results of the long-term Phase 1 study demonstrate the promise of gene therapy across cardiac diseases, including PKP2-ACM, BAG3-DCM, and others.

Long-term follow-up data from the Phase 1 trial of RP-A501 demonstrated sustained improvement in LAMP2 expression and left ventricular mass index (LVMI), together with a favorable tolerability profile. The pivotal Phase 2 trial is fully enrolled and ongoing, with primary endpoints consisting of improved LAMP2B expression and reduced LVMI.

Wall Street analysts believe that medicine possesses a high 65-75% chance to succeed in a pivotal Phase II study next year and can be a $1 billion opportunity for Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT). Despite being a rare disease, the severity of Danon disease and the lack of effective treatments ensure strong demand.

Baron Funds, an investment management company, released its Q2 2024 investor letter. Here is what the fund said:

“Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) specializes in the development of gene therapies for rare genetic diseases outside of oncology. Currently these include Danon disease, Fanconi anemia, lysosomal acid lipase deficiency, and pyruvate kinase deficiency. The first three drug treatments are slated for commercial launch by 2025, which should generate substantial revenue. Shares detracted from performance after the FDA extended the priority review period by three months for the Kresladi gene therapy for leukocyte adhesion deficiency, potentially influenced by sluggish competitive gene therapy launches from bluebird bio in sickle cell disease and BioMarin in hemophilia B. Given the lifesaving nature of Rocket’s therapies and the high unmet need for each of these life ending diseases, we retain conviction in our investment.”