Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) is one of the 12 best buy-the-dip stocks to invest in now.
As of June 5 closing, the consensus sentiment around Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) remained strongly bullish. Based on a median one-year target price of $51.87, the stock offers more than 122% upside.
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Back on May 21, Kristen Kluska from Cantor Fitzgerald increased the target price for the stock from $84 to $96, resulting in a revised upside potential of almost 312%. The analyst reiterated her Overweight rating on the stock, based on a greater statistical success probability of GTX-102 to cure Angelman syndrome. This stems from the Phase 3 design, enabling the threshold for statistical significance to be compartmentalized into 2 endpoints, i.e., MDRI and Bayley-4 cognitive raw score.
More recently, the U.S. Food and Drug Administration issued draft guidance designed to streamline the development and regulatory submission process for promising human gene therapies. After it is finalized, the framework will describe how developers can use current scientific data, public information, and established platform expertise. These include manufacturing, chemistry, and control data, as well as nonclinical and clinical outcomes.
Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), being a key player in this space, is expected to benefit from this development. This regulatory effectiveness focuses on gene therapy products that use genome editing in human somatic cells.
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 potential of RARE as an investment, we believe certain AI stocks offer greater upside potential and carry less downside risk. If you’re looking for an extremely undervalued AI stock that also stands to benefit significantly from Trump-era tariffs and the onshoring trend, see our free report on the best short-term AI stock.
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