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Ultragenyx Pharmaceutical ( (RARE) ) just unveiled an update.
On December 30, 2025, Ultragenyx Pharmaceutical announced it had completed the rolling submission of its Biologics License Application to the U.S. Food and Drug Administration for DTX401, an AAV gene therapy (pariglasgene brecaparvovec) for the treatment of Glycogen Storage Disease Type Ia. The application is supported by a clinical program involving 52 treated patients with up to six years of follow-up, including Phase 3 GlucoGene trial data showing significant and clinically meaningful reductions in patients’ daily cornstarch intake, improved glycemic control and fasting tolerance, and better patient-reported quality of life, alongside an acceptable safety profile. Completion of the BLA, including the final chemistry, manufacturing and controls module submitted in 2025, marks a key regulatory milestone that could advance Ultragenyx’s position in the rare disease gene therapy space and potentially bring a first-of-its-kind treatment option closer to patients with GSDIa, pending FDA review and decision.
The most recent analyst rating on (RARE) stock is a Buy with a $45.00 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’s stock score is primarily influenced by its strong earnings call, which highlighted robust revenue growth and strategic financial moves. However, significant financial performance challenges and valuation issues weigh down the overall score. Positive technical indicators provide some support, reflecting market optimism.
To see Spark’s full report on RARE stock, click here.
More about Ultragenyx Pharmaceutical
Ultragenyx Pharmaceutical Inc. is a biopharmaceutical company focused on developing therapies for rare and ultrarare genetic diseases, including gene therapies targeting serious metabolic disorders such as Glycogen Storage Disease Type Ia (GSDIa).
Average Trading Volume: 1,734,984
Technical Sentiment Signal: Sell
Current Market Cap: $1.9B
For a thorough assessment of RARE stock, go to TipRanks’ Stock Analysis page.