RARE Ultragenyx Pharmaceutical Inc.

51.46
+3.16  (+7%)
Previous Close 48.3
Open 49.84
Price To Book 4.65
Market Cap 3,042,972,447
Shares 59,132,772
Volume 609,138
Short Ratio
Av. Daily Volume 535,150
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NewsSee all news

  1. Ultragenyx Enters into Strategic Partnership with Daiichi Sankyo for Gene Therapy Manufacturing Technology

    Daiichi Sankyo granted non-exclusive license to Ultragenyx HeLa manufacturing platform Ultragenyx to receive $200 million upfront, including $125 million in cash and $75 million via equity investment NOVATO, Calif.,

  2. REGENXBIO and Ultragenyx Announce New License Agreement for Use of NAV® Technology Platform for the Treatment of Rare Metabolic Disorder

    - REGENXBIO grants Ultragenyx exclusive, worldwide rights to NAV AAV8 and AAV9 Vectors for the development and commercialization of gene therapy treatments for a rare metabolic disorder- Agreement for use of NAV Vectors

  3. REGENXBIO and Ultragenyx Announce New License Agreement for Use of NAV® Technology Platform for the Treatment of Rare Metabolic Disorder

    ROCKVILLE, Md. and NOVATO, Calif., March 31, 2020 /PRNewswire/ -- REGENXBIO Inc. (NASDAQ:RGNX), a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy based

  4. GeneTx and Ultragenyx Announce First Patient Dosed in Phase 1/2 Clinical Trial of GTX-102 in Patients with Angelman Syndrome

    SARASOTA, Fla. and NOVATO, Calif., March 16, 2020 (GLOBE NEWSWIRE) -- GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and

  5. Ultragenyx to Present at Barclays Global Healthcare Conference

    NOVATO, Calif., March 09, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic

Drug Information

Drug catalyst information is displayed when you hover over / tap on the stage bar graph.

Phase 1/2 prophylactic steroid cohort data due 2H 2020.
DTX301
Ornithine Transcarbamylase (OTC) Deficiency
Approved under priority review November 15, 2017.
MEPSEVII (vestronidase alfa)
Mucopolysaccharidosis 7 (MPS 7)
Phase 3 data released October 26, 2018 - endpoints not met.
UX007 (triheptanoin)
Glut1 DS patients with the movement disorder phenotype
Phase 3 data released August 22, 2017 - endpoints not met.
Aceneuramic acid extended release (Ace-ER)
GNE Myopathy
Approval announced April 17, 2018.
Crysvita (Burosumab)
X-Linked Hypophosphatemia (XLH)
PDUFA date under priority review June 18, 2020.
Burosumab
Tumor-induced osteomalacia (TIO)
PDUFA date July 31, 2020.
UX007 (triheptanoin)
Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)
Phase 1/2 data from expansion cohort due 1H 2020.
DTX401
Glycogen Storage Disease Type Ia (GSDIa)
Phase 1/2 initiation of dosing announced March 16, 2020 with preliminary data from the first cohorts due in early 2021.
GTX-102
Angelman Syndrome

Latest News

  1. Ultragenyx Enters into Strategic Partnership with Daiichi Sankyo for Gene Therapy Manufacturing Technology

    Daiichi Sankyo granted non-exclusive license to Ultragenyx HeLa manufacturing platform Ultragenyx to receive $200 million upfront, including $125 million in cash and $75 million via equity investment NOVATO, Calif.,

  2. REGENXBIO and Ultragenyx Announce New License Agreement for Use of NAV® Technology Platform for the Treatment of Rare Metabolic Disorder

    - REGENXBIO grants Ultragenyx exclusive, worldwide rights to NAV AAV8 and AAV9 Vectors for the development and commercialization of gene therapy treatments for a rare metabolic disorder- Agreement for use of NAV Vectors

  3. REGENXBIO and Ultragenyx Announce New License Agreement for Use of NAV® Technology Platform for the Treatment of Rare Metabolic Disorder

    ROCKVILLE, Md. and NOVATO, Calif., March 31, 2020 /PRNewswire/ -- REGENXBIO Inc. (NASDAQ:RGNX), a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy based

  4. GeneTx and Ultragenyx Announce First Patient Dosed in Phase 1/2 Clinical Trial of GTX-102 in Patients with Angelman Syndrome

    SARASOTA, Fla. and NOVATO, Calif., March 16, 2020 (GLOBE NEWSWIRE) -- GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and

  5. Ultragenyx to Present at Barclays Global Healthcare Conference

    NOVATO, Calif., March 09, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic

  6. Ultragenyx Announces Planned Transition of Chief Financial Officer

    NOVATO, Calif., March 03, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare diseases, today

  7. Ultragenyx and Kyowa Kirin Announce FDA Acceptance and Priority Review Designation of Supplemental Biologics License Application for Crysvita® (burosumab) for Tumor-Induced Osteomalacia (TIO)

    NOVATO, Calif. and TOKYO, Feb. 27, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development and commercialization of novel products for rare and

  8. Ultragenyx to Present at SVB Leerink Global Healthcare Conference

    NOVATO, Calif., Feb. 21, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases,

  9. Ultragenyx Reports Fourth Quarter and Full Year 2019 Financial Results and Corporate Update

    2019 total revenue is $103.7 million; 2019 Crysvita® (burosumab) revenue to Ultragenyx is $87.3 million 2020 Crysvita revenue in Ultragenyx territories guidance of $125 million to $140 million reaffirmed NOVATO,

  10. Ultragenyx to Host Conference Call for Fourth Quarter and Full-Year 2019 Financial Results and Corporate Update

    NOVATO, Calif., Feb. 06, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases,

  11. GeneTx and Ultragenyx Announce Investigational New Drug (IND) Application Active for GTX-102 in Patients with Angelman Syndrome

    SARASOTA, Fla. and NOVATO, Calif., Jan. 15, 2020 (GLOBE NEWSWIRE) -- GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products

  12. Ultragenyx Reports Preliminary 2019 Revenue and Provides 2020 Crysvita Revenue Guidance

    Preliminary 2019 total revenue is approximately $102 million to $104 million Preliminary 2019 Crysvita revenue to Ultragenyx of approximately $86 million to $88 million 2020 Crysvita revenue in Ultragenyx

  13. Ultragenyx and Kyowa Kirin Announce Submission of Supplemental Biologics License Application to U.S. FDA for Crysvita® (burosumab) for Tumor-Induced Osteomalacia (TIO)

    NOVATO, Calif. and TOKYO, Jan. 13, 2020 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases,

  14. Ultragenyx Announces Positive Topline Cohort 3 Results and Improved Longer-Term Cohort 2 Results from Phase 1/2 Study of DTX301 Gene Therapy in Ornithine Transcarbamylase (OTC) Deficiency

    More uniform response in Cohort 3 with two confirmed responders and one potential responder New female responder in Cohort 2 for a total of three confirmed female responders across all cohorts Up to six responders

  15. Ultragenyx to Present at 38ᵗʰ Annual J.P. Morgan Healthcare Conference

    NOVATO, Calif., Dec. 19, 2019 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases,

  16. Ultragenyx Announces Sale of Future European Royalties on Crysvita® (burosumab) for $320 Million to Royalty Pharma

    NOVATO, Calif. and NEW YORK, Dec. 18, 2019 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases,

  17. Ultragenyx to Present at Upcoming Investor Conferences

    NOVATO, Calif., Nov. 08, 2019 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases,

  18. Ultragenyx Reports Third Quarter 2019 Financial Results and Corporate Update

    Strong Crysvita® (burosumab) Launch Continues with Approximately 1,130 Patients on Reimbursed Commercial Therapy in the United States as of the end of the Third Quarter UX007 (triheptanoin) NDA for Treatment of

  19. Ultragenyx to Host Conference Call for Third Quarter 2019 Financial Results and Corporate Update

    NOVATO, Calif., Oct. 30, 2019 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases,

  20. Ultragenyx Announces FDA Accepts New Drug Application for UX007 (triheptanoin) for Treatment of Long-chain Fatty Acid Oxidation Disorders

    NOVATO, Calif., Oct. 14, 2019 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare diseases, today

  21. Ultragenyx to Present at Jefferies Gene Therapy/Editing Summit

    NOVATO, Calif., Oct. 02, 2019 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases,

  22. Ultragenyx and Kyowa Kirin Announce U.S. FDA Approves Label Update for Crysvita® (burosumab) for the Treatment of X-Linked Hypophosphatemia (XLH)

    Expanded Label Includes Additional Clinical Data Highlighting Superiority Over Conventional Therapy for Pediatric Patients and Extends Current U.S. Indication to Patients Aged Six Months and Older Expanded Label

  23. Ultragenyx and Kyowa Kirin Announce Intent to Submit Supplemental Biologics License Application to U.S. FDA for Crysvita® (burosumab) in Tumor-Induced Osteomalacia (TIO)

    NOVATO, Calif. and TOKYO, Sept. 10, 2019 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, and

  24. Ultragenyx Announces Positive Data from Phase 1/2 Study of DTX401 Gene Therapy in Glycogen Storage Disease Type Ia

    Increased Time to Hypoglycemia and Reduction in Cornstarch Use in Cohorts 1 and 2; Improvement of Additional Key Metabolic Measures Observed Cohort 1 Patients Continue to Demonstrate Long-Term, Durable Responses

  25. GeneTx and Ultragenyx Announce Orphan Drug Designation and Rare Pediatric Disease Designation for GTX-102

    SARASOTA, Fla. and NOVATO, Calif., Sept. 03, 2019 (GLOBE NEWSWIRE) -- GeneTx Biotherapeutics LLC and Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel

  26. Ultragenyx to Present at Upcoming Investor Conferences

    NOVATO, Calif., Aug. 30, 2019 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE), a biopharmaceutical company focused on the development of novel products for serious rare and ultra-rare genetic diseases,