Ultragenyx Pharmaceutical (RARE) Resubmits Biologics License Application for Gene Therapy Treatment


Ultragenyx Pharmaceutical Inc (NASDAQ:rare) is one of the 13 Best Income Growth Stocks to Buy Right Now. On January 30, Ultragenyx Pharmaceutical Inc. (Nasdaq:RARE) resubmitted its Biologics License Application (BLA) to the US Food and Drug Administration, seeking accelerated approval for the UX111 AAV9 gene therapy as a treatment for patients with Sanfilippo syndrome type A (MPS IIIA).

The resubmitted BLA addresses the agency’s concerns about the drug. The company anticipates a review period of up to six months from the date of the new filing, which is expected in the third quarter. If approved, UX111 will be the first approved therapy for Sanfilippo syndrome type A. On the same day, Morgan Stanley analyst Maxwell Skor maintained a buy rating on Ultragenyx Pharmaceutical with a price target of $50. Last month, Leerink Partners gave Ultragenyx Pharmaceutical a buy rating, while Wedbush reiterated its hold rating on the stock. Ultragenyx Pharmaceutical Inc. is a biopharmaceutical company focused on developing therapies for rare and ultra-rare genetic diseases. Its products include Crysvita, Mepsevii, Dojolvi and Evkeeza. While we recognize RARE’s potential as an investment, we believe some AI stocks offer greater upside potential and less downside risk. If you’re looking for an extremely undervalued AI stock that will also benefit significantly from Trump-era tariffs and the onshoring trend, check out our free report on the best short term AI stock. READ THE FOLLOWING: 12 Best Software Infrastructure Stocks to Buy According to Hedge Funds i Cathie Wood’s Stock Portfolio: The 10 Best Stocks to Buy. Disclosure: no. This article is originally published in Monkey Insider.



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