10 Best Gene Therapy Stocks to Buy Right Now

1) Sarepta Therapeutics, Inc. (NASDAQ:SRPT)

Number of Hedge Fund Holders: 50

Sarepta Therapeutics, Inc. (NASDAQ:SRPT) is a commercial-stage biopharmaceutical company, which focuses on the discovery and development of RNA-targeted therapeutics, gene therapies, and other genetic therapeutic modalities for the treatment of rare diseases. The company’s primary focus is on developing genetic-based treatments for rare diseases, particularly those caused by genetic mutations.

The cornerstone of Sarepta Therapeutics, Inc. (NASDAQ:SRPT)’s recent success has been ELEVIDYS, a gene therapy approved by the FDA in June 2023, and later expanded in 2024 to treat ambulatory and non-ambulatory Duchenne muscular dystrophy (DMD) patients aged 4 and above. DMD is a rare genetic disorder characterized by progressive muscle degeneration and weakness. It mainly affects boys and is caused by mutations in the DMD gene, which encodes the protein dystrophin. Dystrophin is important for maintaining the structural integrity of muscle cells.

ELEVIDYS focuses on addressing a broader patient population using a truncated dystrophin gene delivered via an AAV vector.  The one-time treatment demonstrates a meaningful advance. It provides improved functional outcomes in clinical trials. The therapy’s premium pricing, which stands at $3.2 million per patient, places it among the most expensive treatments in the U.S. Despite this, it comes out to be cost-effective over time as compared to recurring exon-skipping therapies, which cost up to $1.5 million on an annual basis.

As per Wall Street, ELEVIDYS approval demonstrates a pivotal shift in Sarepta Therapeutics, Inc. (NASDAQ:SRPT)’s business model, pivoting from a predominantly recurring revenue structure to a business model that relies more on high-value, one-time therapies. This strategic shift enables Sarepta Therapeutics, Inc. (NASDAQ:SRPT) to generate substantial cash flow to fuel innovation while maintaining a balanced pipeline.

While we acknowledge the potential of SRPT as an investment, our conviction lies in the belief that some deeply undervalued AI stocks hold greater promise for delivering higher returns, and doing so within a shorter timeframe. If you are looking for a deeply undervalued AI stock that is more promising than SRPT but that trades at less than 5 times its earnings, check out our report about the cheapest AI stock.

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