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The latest announcement is out from Ultragenyx Pharmaceutical ( (RARE) ).
Ultragenyx Pharmaceutical announced positive long-term results from its Phase 3 study of DTX401 AAV gene therapy for glycogen storage disease type Ia, presented at the International Congress of Inborn Errors of Metabolism 2025 in Kyoto, Japan. The study showed significant reductions in cornstarch intake and improved glycemic control, with participants reporting enhanced quality of life and the therapy demonstrating an acceptable safety profile. These findings underscore the potential of DTX401 to transform treatment for GSDIa, reinforcing Ultragenyx’s position in the rare disease market and offering promising implications for patients and stakeholders.
The most recent analyst rating on (RARE) stock is a Buy with a $105.00 price target. To see the full list of analyst forecasts on Ultragenyx Pharmaceutical stock, see the RARE Stock Forecast page.
Spark’s Take on RARE Stock
According to Spark, TipRanks’ AI Analyst, RARE is a Neutral.
Ultragenyx Pharmaceutical’s overall stock score is primarily influenced by its financial performance challenges, including ongoing losses and negative cash flow. While technical indicators suggest a bearish trend, recent earnings call and corporate events provide some optimism with strong revenue growth and clinical advancements. However, valuation concerns due to unprofitability and regulatory setbacks temper the outlook.
To see Spark’s full report on RARE stock, click here.
More about Ultragenyx Pharmaceutical
Ultragenyx Pharmaceutical Inc. operates in the biopharmaceutical industry, focusing on developing novel products for the treatment of rare and ultra-rare genetic diseases. The company specializes in gene therapy and other innovative therapeutic approaches to address unmet medical needs in this niche market.
Average Trading Volume: 1,972,148
Technical Sentiment Signal: Sell
Current Market Cap: $3.07B
Learn more about RARE stock on TipRanks’ Stock Analysis page.