Ultragenyx Pharmaceutical Inc., a biopharmaceutical company, focuses on the identification, acquisition, development, and commercialization of novel products for the treatment of rare and ultra-rare genetic diseases in the United States. Its biologic products include Crysvita (burosumab), an antibody targeting fibroblast growth factor 23 for the treatment of X-linked hypophosphatemia, as well as tumor-induced osteomalacia; Mepsevii, an enzyme replacement therapy for the treatment of children and adults with Mucopolysaccharidosis VII; and Dojolvi for treating long-chain fatty acid oxidation disorders. The company is also developing DTX401, an adeno-associated virus 8 (AAV8) gene therapy clinical candidate for the treatment of patients with glycogen storage disease type Ia; DTX301, an AAV8 gene therapy product candidate for the treatment of patients with ornithine transcarbamylase; UX143, a human monoclonal antibody for the treatment of osteogenesis imperfecta; GTX-102, an antisense oligonucleotide for the treatment of Angelman syndrome; UX701, for the treatment of Wilson disease; and UX053 for the treatment of glycogen storage disease type III. Ultragenyx Pharmaceutical Inc. has collaboration and license agreement with Kyowa Kirin Co., Ltd.; Saint Louis University; Baylor Research Institute; REGENXBIO Inc.; Bayer; GeneTx; Mereo; University of Pennsylvania; Arcturus Therapeutics Holdings Inc., Solid Biosciences Inc.; and Daiichi Sankyo Co., Ltd. Ultragenyx Pharmaceutical Inc. was incorporated in 2010 and is headquartered in Novato, California.
IPO Year: 2014
Exchange: NASDAQ
Website: ultragenyx.com
| Date | Price Target | Rating | Analyst |
|---|---|---|---|
| 3/24/2026 | $25.00 | Buy → Neutral | Goldman |
| 10/20/2025 | $65.00 | Overweight | Wells Fargo |
| 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 |
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NOVATO, Calif., June 03, 2026 (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 announced that Emil Kakkis, M.D., Ph.D., the company's president and chief executive officer will participate in a fireside chat at the Goldman Sachs 47th Annual Global Healthcare Conference on Tuesday, June 9, 2026, at 4:00 PM ET. The live and archived webcast of the panel will be accessible from the company's website at https://ir.ultragenyx.com/events-presentations. About Ultragenyx Pharmaceutical Inc. Ultragenyx is a biopharmaceutical comp
NOVATO, Calif., May 22, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultra-rare diseases, today reported the grant of 17,567 restricted stock units of the company's common stock to 11 newly hired non-executive officers of the company. The awards were approved by the compensation committee of the company's board of directors and granted under the Ultragenyx Employment Inducement Plan, with a grant date of May 16, 2026, as an inducement material to the new employees entering into employment with Ultragenyx in accordance with Nasdaq Listing Rule 5635(c)(4). T
NOVATO, Calif., May 06, 2026 (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 announced that Howard Horn, the company's Chief Financial Officer and Executive Vice President and Joshua Higa, Chief of Staff and Vice President of investor relations, will participate in a fireside at Bank of America's 2026 Healthcare Conference on Tuesday, May 12, 2026, at 2:20 PM PT. The live and archived webcast of the panel will be accessible from the company's website at https://ir.ultragenyx.com/events-presentations. About Ultragenyx Pha
First quarter total revenue of $136 million, Crysvita® revenue of $93 million and Dojolvi® revenue of $18 million Reaffirm 2026 financial guidance, including total revenue of $730 million to $760 million and combined R&D and SG&A expenses to be flat to slightly down versus 2025; remain on path to profitability in 2027 GTX-102 for Angelman syndrome: New longer-term data from Phase 1/2 clinical study support durable and improving effects across multiple domains; Phase 3 data expected in the second half of 2026 NOVATO, Calif., May 05, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel
NOVATO, Calif., April 29, 2026 (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, May 5, 2026, to discuss its financial results and corporate update for the quarter ending March 31, 2026. 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 biopharmaceutica
NOVATO, Calif., April 24, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultra-rare diseases, today reported the grant of 36,447 restricted stock units of the company's common stock to 12 newly hired non-executive officers of the company. The awards were approved by the compensation committee of the company's board of directors and granted under the Ultragenyx Employment Inducement Plan, with a grant date of April 16, 2026, as an inducement material to the new employees entering into employment with Ultragenyx in accordance with Nasdaq Listing Rule 5635(c)(4)
If approved, UX111 will be the first approved therapy for the treatment of Sanfilippo syndrome Type A, a rare disease affecting young children that leads to progressive, irreversible neurodegeneration and early death PDUFA action date set for September 19, 2026 NOVATO, Calif., April 02, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) today announced the U.S. Food and Drug Administration (FDA or the Agency) has accepted for review the resubmitted Biologics License Application (BLA) seeking accelerated approval for UX111 (rebisufligene etisparvovec) AAV9 gene therapy as a treatment for patients with Sanfilippo syndrome Type A (MPS IIIA). The FDA set a Prescription Dru
NOVATO, Calif., March 30, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) today announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for UX016, an investigational small molecule prodrug of sialic acid (SA) being evaluated as a substrate replacement therapy for GNE myopathy (GNEM). GNEM is a rare, severely debilitating, inherited neuromuscular disorder caused by mutations in the GNE gene that lead to deficient SA production. The UX016 program is externally funded by a patient group through clinical proof-of-concept, including a Phase 1/2 study expected to begin in the second half of 2026. "People living
NOVATO, Calif., March 20, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultra-rare diseases, today reported the grant of 10,839 restricted stock units of the company's common stock to two newly hired non-executive officers of the company. The awards were approved by the compensation committee of the company's board of directors and granted under the Ultragenyx Employment Inducement Plan, with a grant date of March 16, 2026, as an inducement material to the new employees entering into employment with Ultragenyx in accordance with Nasdaq Listing Rule 5635(c)(4
Statistically significant improvements in primary endpoint of ammonia control compared with placebo at 36 weeks Clinically important changes observed in patient global impression scale for overall OTC symptoms, OTC deficiency symptoms, and OTC impact on daily living DTX301 was well tolerated with an acceptable safety profile NOVATO, Calif., March 12, 2026 (GLOBE NEWSWIRE) -- Today Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) announced positive results from its Phase 3 Enh3ance study of DTX301, an investigational AAV8 gene therapy for the treatment of ornithine transcarbamylase (OTC) deficiency. At Week 36 in the randomized, double-blind placebo-controlled period of the trial, DTX301-tre
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Goldman downgraded Ultragenyx Pharma from Buy to Neutral and set a new price target of $25.00
Wells Fargo initiated coverage of Ultragenyx Pharma with a rating of Overweight and set a new price target of $65.00
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
RBC Capital Mkts initiated coverage of Ultragenyx Pharma with a rating of Outperform and set a new price target of $77.00
Wells Fargo initiated coverage of Ultragenyx Pharma with a rating of Overweight and set a new price target of $72.00
Credit Suisse resumed coverage of Ultragenyx Pharma with a rating of Outperform and set a new price target of $96.00
Evercore ISI upgraded Ultragenyx Pharma from In-line to Outperform and set a new price target of $80.00 from $60.00 previously
Cantor Fitzgerald initiated coverage of Ultragenyx Pharma with a rating of Overweight and set a new price target of $114.00
Live Leadership Updates
NOVATO, Calif., Sept. 30, 2025 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) today announced the appointment of Eric Olson as chief business officer (CBO) and executive vice president effective September 22, 2025, following the planned retirement of Thomas Kassberg. Mr. Olson will be responsible for leading the company's business development, corporate development and alliance management functions. "We thank Tom for his 14 years of impactful contributions to Ultragenyx, including transformative business deals that helped build the largest clinical pipeline in rare disease; expanded our reach to patients with fatty acid oxidation diseases, X-linked hypophosphatemia, o
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
Accomplished biotechnology executive brings over 40 years of commercial and leadership experience across multinational pharmaceutical and biotechnology companies to ShouTi ShouTi Inc., a clinical-stage biopharmaceutical company replacing biologics with world-class small molecule medicines using advanced computational and structure-based technology, today announced that Daniel Welch has been named Chairman of the company's board of directors. "It is a true privilege to welcome Dan to ShouTi as Chairman of our board," said Raymond Stevens, Ph.D., Chief Executive Officer, ShouTi. "As an industry veteran with an outstanding track record in guiding companies across all stages of development an
Dr. Suliman brings over 25 years of drug development, deal-making and company building expertise as ReCode continues advancing its pipeline of novel genetic medicines using its powerful selective organ targeting (SORT) LNP delivery platform ReCode Therapeutics (the "Company"), a biopharmaceutical company pioneering disease-modifying genetic medicines using its proprietary LNP delivery platform, today announced the appointment of Shehnaaz Suliman, M.D., MBA, M.Phil., as chief executive officer and as a member of the board of directors effective today. Former CEO, David Lockhart, Ph.D., will transition to the role of chief scientific officer and will remain president and a member of the boar
NOVATO, Calif., June 30, 2021 (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 announced the appointment of Corsee Sanders, Ph.D., to the Board of Directors, effective June 29, 2021. Dr. Sanders, an experienced veteran of the biopharma industry, will serve as an independent director. "Dr. Sanders' established experience in global clinical development and especially with new technologies will be instrumental as we further advance our ongoing clinical trials and continue to expand our rare disease pipeline of novel therapeut
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Live finance-specific insights
First quarter total revenue of $136 million, Crysvita® revenue of $93 million and Dojolvi® revenue of $18 million Reaffirm 2026 financial guidance, including total revenue of $730 million to $760 million and combined R&D and SG&A expenses to be flat to slightly down versus 2025; remain on path to profitability in 2027 GTX-102 for Angelman syndrome: New longer-term data from Phase 1/2 clinical study support durable and improving effects across multiple domains; Phase 3 data expected in the second half of 2026 NOVATO, Calif., May 05, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel
NOVATO, Calif., April 29, 2026 (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, May 5, 2026, to discuss its financial results and corporate update for the quarter ending March 31, 2026. 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 biopharmaceutica
2025 total revenue of $673 million,Crysvita® revenue of $481 million and Dojolvi® revenue of $96 million 2026 total revenue from current products expected to be between $730 million to $760 million Initiated a strategic restructuring plan to significantly reduce and focus expenses and headcount, reiterate path to profitability in 2027 2026 catalysts include two potential approvals and expected pivotal Phase 3 data from the GTX-102 Phase 3 Aspire study for Angelman syndrome NOVATO, Calif., Feb. 12, 2026 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and
NOVATO, Calif., Feb. 05, 2026 (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 Thursday, February 12, 2026, to discuss its financial results and corporate update for the quarter and the year ending December 31, 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
Third quarter total revenue of $160 million, Crysvita® revenue of $112 million and Dojolvi® revenue of $24 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 Bolstered balance sheet with $400 million proceeds from sale of portion of Crysvita royalty interest NOVATO, Calif., Nov. 04, 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 reported its financial results for the quarte
NOVATO, Calif., Oct. 28, 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, November 4, 2025, to discuss its financial results and corporate update for the quarter ending September 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 biophar
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
NOVATO, Calif., April 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, May 6, 2025, to discuss its financial results and corporate update for the quarter ending March 31, 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 biopharmaceutica