11 Best Gene Therapy Stocks to Buy According to Analysts

Page 9 of 9

1. Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE)

Price Target Upside: 95.96%

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) tops our list for being one of the best genomic stocks. It focuses on developing and commercializing treatments for genetic diseases. Its product portfolio includes Crysvita, Mepsevii, Dojolvi, and Evkeeza, with plans to expand in 2025 through several products in launch mode worldwide. The company exceeded expectations in 2024, reporting $560 million in total revenue and forecasting $640 million to $670 million in 2025. Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) is targeting full-year GAAP profitability by 2027, and analysts remain bullish, with Buy ratings and price targets ranging from $104.00 to $115.00, implying significant upside potential.

The company employs in vivo gene replacement therapy using adeno-associated viral (AAV) vectors to deliver therapeutic genes into specific cells. This one-time treatment aims to target the underlying cause of genetic diseases, potentially halting or slowing progression. The corporation’s gene therapy pipeline includes promising candidates such as UX111 (for Mucopolysaccharidosis Type IIIA), DTX301 (for Ornithine Transcarbamylase deficiency), DTX401 (for Glycogen Storage Disease Type Ia), and a gene therapy for Wilson disease.

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) has developed the Pinnacle PCL Platform, which is a proprietary manufacturing system designed to enhance scalability and reduce production costs. This platform uses engineered Producer Cell Lines (PCLs) to produce high yields of AAV vectors, ensuring safety and quality while enabling faster therapy delivery. The company is also advancing regulatory progress, which includes the submission of a Biologics License Application (BLA) for UX111, with a surrogate endpoint supporting long-term cognitive benefits.

Overall, Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) ranks first among the 11 best gene therapy stocks to buy according to analysts. While we acknowledge the potential of gene therapy companies, our conviction lies in the belief that AI stocks hold greater promise for delivering higher returns, and doing so within a shorter time frame. If you are looking for an AI stock that is more promising than RARE but that trades at less than 5 times its earnings, check out our report about the cheapest AI stock.

READ NEXT: 20 Best AI Stocks To Buy Now and 30 Best Stocks to Buy Now According to Billionaires.

Disclosure: None. Insider Monkey focuses on uncovering the best investment ideas of hedge funds and insiders. Please subscribe to our free daily e-newsletter to get the latest investment ideas from hedge funds’ investor letters by entering your email address below.

Page 9 of 9