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

Ultragenyx Pharmaceutical Inc (NASDAQ:RARE) is one of the 13 Best Revenue Growth Stocks to Buy Right Now. On January 30, Ultragenyx Pharmaceutical Inc. (Nasdaq:RARE) resubmitted its Biologics License Application (BLA) to the U.S. Food and Drug Administration, seeking accelerated approval for UX111 AAV9 gene therapy as a treatment for patients with Sanfilippo syndrome type A (MPS IIIA). The resubmitted BLA addresses the agency’s concerns regarding the drug. The company anticipates up to a six-month review period from the date of resubmission, 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. In the past month, Leerink Partners gave a Buy rating to Ultragenyx Pharmaceutical, 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 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. READ NEXT: 12 Best Software Infrastructure Stocks to Buy According to Hedge Funds and Cathie Wood’s Stock Portfolio: Top 10 Stocks to Buy. Disclosure: None. This article is originally published at Insider Monkey.