Ultragenyx Begins Dosing in Aurora Study for Angelman Syndrome

Ultragenyx Pharmaceutical ( (RARE) ) just unveiled an update.

On October 30, 2025, Ultragenyx Pharmaceutical announced the dosing of the first patient in the Aurora study, which evaluates the efficacy and safety of GTX-102 for Angelman syndrome across a broader patient population. The Aurora study aims to include approximately 60 participants with various genotypes and age ranges, expanding beyond the Phase 3 Aspire study’s focus on patients with full maternal UBE3A gene deletion. This development signifies a strategic expansion in Ultragenyx’s clinical research efforts, potentially enhancing its market position and offering new treatment options for Angelman syndrome patients.

The most recent analyst rating on (RARE) stock is a Hold with a $36.00 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 driven by financial performance challenges, including ongoing losses and negative cash flow. While technical indicators show positive momentum, the valuation is impacted by a negative P/E ratio. The earnings call provided optimistic guidance with strong revenue growth and clinical progress, but financial losses remain a concern.

To see Spark’s full report on RARE stock, click here.

More about Ultragenyx Pharmaceutical

Ultragenyx Pharmaceutical Inc. operates in the biotechnology industry, focusing on developing novel products for the treatment of rare and ultra-rare genetic diseases. The company specializes in creating therapies such as antisense oligonucleotides for conditions like Angelman syndrome.

Average Trading Volume: 1,554,148

Technical Sentiment Signal: Sell

Current Market Cap: $3.33B

For an in-depth examination of RARE stock, go to TipRanks’ Overview page.