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

Core Viewpoint - Ultragenyx Pharmaceutical Inc. is recognized as one of the best revenue growth stocks, with a focus on developing therapies for rare genetic diseases, particularly through its resubmission of the Biologics License Application for UX111 AAV9 gene therapy targeting Sanfilippo syndrome type A [1][3]. Group 1: Company Developments - The company resubmitted its Biologics License Application (BLA) to the U.S. FDA on January 30, seeking accelerated approval for UX111 AAV9 gene therapy [1]. - The resubmitted BLA addresses previous concerns from the FDA, with an anticipated review period of up to six months, expected to conclude in the third quarter [3]. - If approved, UX111 will be the first therapy available for Sanfilippo syndrome type A [3]. Group 2: Analyst Ratings and Market Position - Morgan Stanley analyst Maxwell Skor maintained a Buy rating on Ultragenyx Pharmaceutical with a price target of $50 [3]. - In the past month, Leerink Partners also issued a Buy rating, while Wedbush reiterated a Hold rating on the stock [3]. - The company’s product portfolio includes therapies such as Crysvita, Mepsevii, Dojolvi, and Evkeeza, emphasizing its commitment to rare and ultra-rare genetic diseases [3].