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Ultragenyx Pharmaceutical (RARE) Garnering Bullish Sentiment With Forthcoming GTX-102 Phase 3 Angelman Trial

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) is one of the 10 best biotech stocks with highest upside potential.

On April 16, Morgan Stanley increased the price target for Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) from $50 to $67, resulting in an adjusted upside potential in excess of 178% at the prevailing level. The firm also reiterated its Outperform rating on the stock.

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Morgan Stanley revised the price target upward based on a strict new probability-of-success approach to the forthcoming GTX-102 Phase 3 Angelman trial. This major clinical readout is anticipated in the second half of 2026. The resulting risk profile is rather asymmetrical. The firm has projected a 50% to 70% upside, which significantly offsets a 20% downside risk of a clinical failure.

Back on March 30, Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) announced clearance from the US Food & Drug Administration for the Investigational New Drug Application for UX016. It is a prodrug of sialic acid under investigation for use as a substrate replacement therapy for GNE Myopathy. GNEM is a rare neuromuscular disease characterized by severe disability due to mutations in the GNE gene, which impairs sialic acid biosynthesis.

The UX016 program is supported externally via proof-of-concept clinical trials, which include a Phase 1/2 trial scheduled for initiation in the latter half of 2026. Commenting on the progress of UX016, CEO Emil Kakkis observed that moving the drug into the clinic is a significant step for the GNE myopathy community, and it demonstrates the company’s effort to create a novel method for improving sialic acid utilization by the muscles. The first-in-human study will involve around 24 patients between 18 and 55 years of age suffering from GNEM, who will be recruited in the US.

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) develops novel therapies, with a focus on identifying, acquiring, and commercializing products for rare and ultra-rare genetic diseases. It has a strong emphasis on gene therapy and covers various stages of clinical trials. The company is currently going through high cash burn with the aim of turning profitable from 2027 onwards.

While we acknowledge the risk and potential of RARE as an investment, our conviction lies in the belief that some 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 and that has 10,000% upside potential, check out our report about this cheapest AI stock.

READ NEXT: 33 Stocks That Should Double in 3 Years and 15 Stocks That Will Make You Rich in 10 Years.

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