William Blair initiated coverage on Ultragenyx Pharma
$RARE
Biotechnology: Pharmaceutical Preparations
Health Care
William Blair initiated coverage of Ultragenyx Pharma with a rating of Outperform
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| Date | Price Target | Rating | Analyst |
|---|---|---|---|
| 7/28/2025 | $80.00 | Buy | H.C. Wainwright |
| 5/28/2025 | Outperform | William Blair | |
| 6/6/2024 | $56.00 → $67.00 | Neutral → Buy | Goldman |
| 4/22/2024 | $77.00 | Outperform | RBC Capital Mkts |
| 12/8/2023 | $72.00 | Overweight | Wells Fargo |
| 6/14/2023 | $96.00 | Outperform | Credit Suisse |
| 6/6/2023 | $60.00 → $80.00 | In-line → Outperform | Evercore ISI |
| 4/26/2023 | $114.00 | Overweight | Cantor Fitzgerald |
NOVATO, Calif., Aug. 18, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) today announced the initiation of a rolling submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) seeking approval for DTX401 AAV gene therapy as a treatment for Glycogen Storage Disease Type Ia (GSDIa). The company has submitted the non-clinical and clinical modules to the FDA and plans to complete the full BLA including submission of the chemistry, manufacturing and controls (CMC) module in the fourth quarter of 2025. "Initiating the BLA for DTX401, for the potential treatment of GSDIa, is an important milestone for this much needed treatment option
Second quarter total revenue of $166 million,Crysvita® revenue of $120 million and Dojolvi® revenue of $23 million Reaffirm 2025 Revenue Guidance: Total revenue between $640 million to $670 million, Crysvita revenue of $460 million to $480 million, and Dojolvi revenue of $90 million to $100 million UX143 for osteogenesis imperfecta Phase 3 data from Orbit and Cosmic studies expected around the end of the year GTX-102 for Angelman syndrome received Breakthrough Therapy Designation from FDA;Phase 3 Aspire study fully enrolled NOVATO, Calif., Aug. 05, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercia
Company expects to complete Phase 3 Aspire study in the second half of 2026 Aurora study of GTX-102 in additional ages and genotypes on track to initiate in the second half of 2025 NOVATO, Calif., July 31, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) today announced that the Phase 3 Aspire study evaluating GTX-102 (apazunersen) as a treatment for Angelman Syndrome is fully enrolled, with approximately 129 participants ages four to 17 with a genetically confirmed diagnosis of full maternal UBE3A gene deletion. "The accelerated enrollment of the Phase 3 Aspire study underscores the urgent need and strong desire for an effective treatment for these patients. Suppor
4 - Ultragenyx Pharmaceutical Inc. (0001515673) (Issuer)
4 - Ultragenyx Pharmaceutical Inc. (0001515673) (Issuer)
4 - Ultragenyx Pharmaceutical Inc. (0001515673) (Issuer)
H.C. Wainwright resumed coverage of Ultragenyx Pharma with a rating of Buy and set a new price target of $80.00
William Blair initiated coverage of Ultragenyx Pharma with a rating of Outperform
Goldman upgraded Ultragenyx Pharma from Neutral to Buy and set a new price target of $67.00 from $56.00 previously
8-K - Ultragenyx Pharmaceutical Inc. (0001515673) (Filer)
8-K - Ultragenyx Pharmaceutical Inc. (0001515673) (Filer)
8-K - Ultragenyx Pharmaceutical Inc. (0001515673) (Filer)
NOVATO, Calif., July 12, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultrarare diseases, today announced that it has appointed Howard Horn as executive vice president and chief financial officer (CFO) effective October 16, 2023. Mr. Horn will be responsible for leading the finance, accounting, corporate strategy and investor relations functions. "Howard is joining Ultragenyx and its leadership team during a pivotal period as our robust late-stage pipeline is maturing and we are preparing for the next phase of company growth," said Emil D. Kakkis, M.D.,
NOVATO, Calif., March 14, 2023 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) announced that Eric Crombez, M.D., has been promoted to chief medical officer and executive vice president effective May 1, 2023, succeeding Camille Bedrosian, M.D., who will remain with the company in a full-time strategic advisory role. Dr. Crombez currently serves as Ultragenyx's chief medical officer for gene therapy and inborn errors of metabolism. "Eric has extensive expertise in the development and execution of clinical development programs for rare genetic disorders and has been a driving force for our entire gene therapy pipeline," said Emil D. Kakkis, M.D., Ph.D., chief executive of
NOVATO, Calif., April 22, 2022 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, today announced the appointment of Amrit Ray, M.D., M.B.A. to the company's Board of Directors. Dr. Ray will serve on the Board's Research and Development Committee. "Dr. Ray brings to our Board of Directors decades of global experience as a physician researcher, biopharmaceutical executive, and champion for patient access to innovative treatments," said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. "His expertise
Second quarter total revenue of $166 million,Crysvita® revenue of $120 million and Dojolvi® revenue of $23 million Reaffirm 2025 Revenue Guidance: Total revenue between $640 million to $670 million, Crysvita revenue of $460 million to $480 million, and Dojolvi revenue of $90 million to $100 million UX143 for osteogenesis imperfecta Phase 3 data from Orbit and Cosmic studies expected around the end of the year GTX-102 for Angelman syndrome received Breakthrough Therapy Designation from FDA;Phase 3 Aspire study fully enrolled NOVATO, Calif., Aug. 05, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercia
NOVATO, Calif., July 30, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, today announced that it will host a conference call at 5:00 p.m. ET on Tuesday, August 5, 2025, to discuss its financial results and corporate update for the quarter ending June 30, 2025. The live and replayed webcast of the call will be available through the company's website at https://ir.ultragenyx.com/events-presentations. The replay of the call will be available for three months. About Ultragenyx Pharmaceutical Inc.Ultragenyx is a biopharmace
First quarter total revenue of $139 million, Crysvita® revenue of $103 million and Dojolvi® revenue of $17 million Reaffirm 2025 Financial Guidance: Total revenue between $640 million to $670 million, Crysvita revenue of $460 million to $480 million, and Dojolvi revenue of $90 million to $100 million UX111, an investigational treatment for Sanfilippo syndrome, biologics license application (BLA) on track for PDUFA action date of August 18, 2025 NOVATO, Calif., May 06, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultra-rare genetic diseases, today
SC 13G/A - Ultragenyx Pharmaceutical Inc. (0001515673) (Subject)
SC 13G/A - Ultragenyx Pharmaceutical Inc. (0001515673) (Subject)
SC 13G/A - Ultragenyx Pharmaceutical Inc. (0001515673) (Subject)